Position:  Medical Director, Vaccines at [Beardsworth Consulting Group, Inc.]  /   Editor in Chief at Journal of Immune Based Therapies and Vaccines  /   Consultant at Chesapeake PERL  /   Consultant, Vaccines and Biologics at RWMaloneMD.com (Self-employed)  /   President at RW Malone MD LLC

Location :   Greater Atlanta Area

Work:

• [Beardsworth Consulting Group, Inc.] since Mar 2010  /   Medical Director, Vaccines
• Journal of Immune Based Therapies and Vaccines since Feb 2009  /   Editor in Chief
• Chesapeake PERL since 2008   /   Consultant
• RWMaloneMD.com (Self-employed) since Oct 2007  /   Consultant, Vaccines and Biologics [, and President]
• Accelovance Oct 2008 - Oct 2009  /   Medical Director, Vaccines
• PATH 2008 - 2009  /   consultant
• EpiVax Dec 2007 - Oct 2008  /   Senior Director, Vaccines and Therapeutics
• Solvay Pharmaceuticals, Inc. Nov 2006 - Dec 2007  /   Director, Clinical Development/Medical Affairs, Influenza
• Summit Drug Development Services Oct 2005 - Oct 2006  /   Senior Medical Director
• Gene Delivery Alliance, Inc. Apr 2001 - Oct 2005  /   President and founder.
• Dynport Vaccine Company, LLC Oct 2002 - Oct 2003  /   Associate Director, Clinical Research
• University of Maryland, Baltimore School of Medicine, Dept. of Jan 1997 - Aug 2000  /   Assistant Professor
• University of California, Davis Department of Medical Pathology, Oct 1993 - Jan 1997  /   Assistant Professor , Pathology
• UC Davis Medical Center Jul 1992 - Sep 1993  /   Research Fellow
• Vical 1989 - 1989  /   Scientist
• Salk Institute 1986 - 1989  /   Graduate Student
• Michael Charmichael Construction Oct 1978 - Jan 1980  /   Foreman, Shingling crew

Education:

• University of California, Davis - School of Medicine 1991 - 1992  /   Medical Internship, Pathology
• Northwestern University - The Feinberg School of Medicine 1984 - 1991
• University of California, San Diego 1986 - 1988  /   M.S., Biology
• University of California, Davis 1982 - 1984  /   B.S, Biochemistry

Interests:

• Vaccine development, Biotechnology, Clinical Development, Polynucleotide delivery, Gene therapy, DNA vaccines, Electroporative delivery, Federal Grants and Contracts, Proposal Development, Regulatory Affairs

Honor & Awards:

• • Trainee Investigator Award, American Federation for Clinical Research (4/93), • Bank of America-Giannini Foundation Medical Research. Fellow (6/92 to 6/93), • Henry Christian Award for Excellence in Research, American Federation for Clinical Res. (5/92), • UCDMC Medical Scholars Grant (7/92 to 7/93), • First Place, Northwestern AOA Research Symposium competition for Medical Students (1989) , • USPHS Pre-Doctoral Fellowship (1986-1988), • San Diego Supercomputer Grant for RNA structure modeling (1988), • Northwestern University MD../Ph.D. Scholarship (1984-1986), • Dean's List, UC. Davis (1982-1984), • President's Undergraduate Fellowship Grant for investigation of oncogene expression in breast tumor tissue. UC. Davis (9/83-6/84), • Edmonson Summer Fellowship, Department of Pathology, UC. Davis Medical School (1984)

" Robert W. Malone, M.D., M.Sci.  CEO, CSO,  co-founder and board of manager member of Atheric Pharmaceutical, LLC. Dr. Malone has extensive research and development experience in the areas of clinical trials, vaccines, gene therapy, bio-defense, and immunology.  He has over twenty years of management and leadership experience in the academia, pharmaceutical and biotechnology industries.  His FDA, HHS, and DoD agency knowledge is extensive.  Dr. Malone is an internationally recognized scientist and is known as one of the original inventors of “DNA Vaccination.”  Dr. Malone holds numerous fundamental domestic and foreign patents in the fields of gene delivery, delivery formulations, and vaccines.  He has over fifty peer-reviewed publications, has been an invited speaker at over thirty-five conferences, has chaired numerous conferences and he has sat on numerous study sections.  Dr. Malone is in the Harvard Medical School Global Clinical Scholars Program in 2015-2016. In August 2014, colleagues at the Department of Defense/Defense Threat Reduction Agency asked Dr. Malone to step in and help NewLink manage the Ebola project and develop the contracts necessary to move the "orphan" PHAC/rVSV ZEBOV vaccine forward quickly.  Dr. Malone got the project on track, recruited our client Focus Clinical Trials to team with USAMRIID/WRAIR to develop the immunoassays, put WHO leadership in touch with Pentagon leadership to expedite the initial WRAIR clinical trials, recruited the government of Norway to help fund the clinical research, used social media (LinkedIn) to recruit Merck Vaccines to join the project, recruited a management team, and lead the development of the BARDA and DTRA contracts - yielding over 200M$ in resources.  Those were frightening times, but now we have a remarkably effective vaccine, developed in record time. Dr. Malone was CEO and co-founder of RW Malone MD, LLC, a very successful consulting and development firm focused on clinical trials and USG medical countermeasure proposal capture and management.  As a capture and proposal manager, Dr. Malone has an extraordinary funding record.  In the past five years, he has been involved in ten billion dollars of successful government contracts and awards."

PROFESSIONAL EXPERIENCE

Dr. Malone is internationally recognized as one of the original inventors of “DNA Vaccination.” He holds numerous fundamental domestic and foreign patents in the fields of gene delivery, delivery formulations, and vaccines, has over fifty publications, has served as an invited speaker at over thirty conferences, has chaired numerous conferences, and has sat on numerous US Federal study sections.

Dr. Malone has extensive research and clinical development experience in the areas of clinical trials design and implementation, vaccines, gene therapy, biodefense, and immunology. He has over twenty years of management and leadership experience in academia, pharmaceuticals and biotechnology. His NGO, HHS, NIH, and DoD contract and grant knowledge is extensive, and he has helped many groups and companies to capture and manage multi-million dollar awards with these sponsors, including almost 10 billion dollars won in the last five years. Dr. Malone has superior leadership skills, and has often brought diverse teams together to tackle complex problems, and develop innovative solutions.

In 2014, Dr. Malone built and led the initial team, under NewLink Genetics, that took the Canadian rVSVZEBOV-G Ebola vaccine from an abandoned vaccine candidate to funding in the 100 million USD range. He led the team that implemented effective stockpiling strategies, project planning and clinical trials development for dosing strategies. He also set up the initial acquisition talks between Merck Vaccine and Newlink, which led to the sale, research collaboration, and successes of the development of the rVSVZEBOV-G Ebola vaccine.

In 2016, Dr. Malone started a new company: Atheric Pharmaceutical, LLC. Atheric™ Pharmaceutical LLC is a biopharmaceutical company focused on the rapid development and commercialization of repurposed drugs to prevent and treat Zika and other Flavivirus disease. Atheric's lead drug products are reformulated broad spectrum antiviral drugs that inhibit autophagy-dependent viral replication as well as other virus-cell interactions. Atheric is committed to providing broad-spectrum medical countermeasures for Zika and other neglected tropical diseases. Provisional patents for these indications have been filed with the USPTO. The lead drug candidates are approved or allowed by FDA/EMA for use during pregnancy, and cross the placenta enabling clinically significant pharmacodistribution to both mother and fetus. While CEO of Atheric, Dr. Malone has led research teams that have conducted an extensive analysis of drug compounds –working closely with USAMRIID, published two papers in PLoS NTD, have three more papers on Zika in preparation, has filed nine patents with due diligence performed for field of use, conducted in-vitro screening of compounds, and has identified lead drug candidates for prophylactic and therapeutic indications.

Dr. Malone is licensed to practice medicine in the state of Maryland, USA and holds a Doctorate of Medicine from Northwestern University Medical School.

He graduated from Harvard Medical School, Global Clinical Scholars Research Training Program in 2016. His pathology internship was completed at UC Davis. Dr. Malone has served as Adjunct Associate Professor of Biotechnology at Kennesaw State University, as faculty (Associate Professor) at the Uniformed Services University of the Health Sciences, as faculty at the University of Maryland, Medical School and at UC Davis. He holds a MS from UC San Diego in Biology and a BS in Biochemistry from UC Davis.

SUMMARY OF ACCOMPLISHMENTS / SKILLS

• A senior executive and scientist with a highly successful track record of leading bench and discovery research through FDA Phase I, II, and III clinical trials, protocol development and submission, and related regulatory submissions including pIND and IND.
• Significant expertise in drug development and delivery.
• Experienced capturing and managing large federal contracts (including BARDA) with over 9 billion in ID/IQ awards and 562 million government contracts won and/or managed in the last five years.
• Domestically trained, Maryland Licensed Physician/Scientist.
• Expertise in pathology, infectious disease, pandemic clinical trials, influenza, regulatory affairs, project management, biodefense, HIV and Ebola.
• Significant expertise with federal contracting, grants, international NGO health related research and development coupled with professional relationships at CDC, DoD, HHS (BARDA, CDC, FDA and NIAID).
• Prior and current service on many federal study sections and oversight boards involving infectious disease, vaccine, and biodefense.
• Experienced Business Development Professional, project manager, capture/proposal manager, color team reviewer and editor for projects valued from 10M$ up to 1B$ US, with experience managing processes and teams in a wide variety of non-profit and for-profit corporate cultures including both matrix and traditional environments.
• Highly skilled in fostering a culture of innovative problem solving within project teams.
• DoD Secret Clearance authorized.
• Graduated from the Harvard Medical School Global Clinical Scholars Research Training Program, a year-long program focused on international clinical research. This program combines on-site (London & Boston) as well as distance learning, with an average of 15h per week lecture and practicum exercises. The 2016 class included approximately 150 other MD and PhD-level participants from around the world.

WORK EXPERIENCE

• Atheric Pharmaceutical, LLC.
  • • CEO, and Co-founder.
      • • Feb 2016-present. Atheric™ Pharmaceutical LLC is a biopharmaceutical company focused on the rapid development and commercialization of re-purposed drugs to prevent and treat Zika and other Flavivirus disease.
• RW Malone MD, LLC
  • • CEO and Consultant 2001-Present
    • Proposal development, strategic leadership, partnership development, Consulting services in Government Contracting Consulting services in Commercial Intelligence and due diligence, FDA Phase I, II, and III clinical trials, protocol development and submission, and related regulatory submissions including pIND and IND. Provided business development, proposal management, clinical development and medical affairs support for vaccines-related business operations
    • Projects include:
      • • Ebola vaccine project for NewLink/[BioProtection Systems Inc.] (rVSVdG ZEBOV vaccine project), resulting in well over 100M USD non-dilutive capital to NL/BPS. This also included working with the World Health Organization as well as initial set up of the licensing deal to Merck Vaccines of the rVSVdG ZEBOV vaccine.
        • Served as Medical Director, [Beardsworth Consulting Group, Inc.], half time position on retainer (2010 – 2013)
        • Service on federal biotechnology/vaccines proposal study sections
        • Served as Editor-In-Chief of Journal of Immune Based Therapies and Vaccines 2007-2012
        • Service on Safety Monitoring Committee, Phase 1 safety/immunogenicity of novel Influenza vaccine
        • Consulting support for multiple vaccine-focused clinical sites in US and Latin America
        • Served as Medical Director, Vaccines with Accelovance, Inc. (2008 – 2009)
        • Served as medical monitor for multiple seasonal and pandemic (H1N1) studies
        • Reviewed and edited clinical protocols
        • Examples of multi-year contract clients include Accelovance, Avancer, [Beardsworth Consulting Group, Inc.], Chesapeake Perl, Corium, ITS, ITT-Exelis, [EpiVax], Jean Brown Research, Quest Diagnostics (Focus), PaxVax, SAI, Soligenix, TASC, Univ of MA.
        • Commercial intelligence work for two of the largest pharmaceutical companies in the world (subcontractor).
        • Partnering with Galloway and Associates ([Dr. Darrell Ray Galloway (born 1946)]) 2012-2014
        • Acting as Managing Director, Clinical Development and Government Affairs for the Avancer Group. ( April 2012 – 2016.)
        • Proposal development (patch-based vaccine delivery, Tularemia vaccine, CDC contract for clinical trials site development, international government and NGO contract and grant solicitations) – Aeras Global TB Vaccine Foundation 2003-2005
        • Proposal development (plague vaccine- HHS), Technical diligence – VaxGen Corporation
        • Consulting services for [EpiVax], 2005-present [which is 2017] (member, Scientific Advisory Board)
        • Consulting services for [Aldevron]   2001-2005 (operating as Gene Delivery Alliance) 
        • Business and proposal development in the areas of Bioinformatics and Life Sciences (including telemedicine) and research at the University of Bern, Switzerland
        • Consulting services for Molecular Histology, Inc. with the title of Medical Director
        • Consulting services for MSD, Inc. for business/ technology development planning
• Kennesaw State University
  • • Adjunct Associate Professor 2009-2013
• [Beardsworth Consulting Group, Inc.]
  • • Medical Director, Vaccines (RW Malone MD, LLC under contract to Beardsworth)
    • 2010-2013, Dr. Malone functioned as the in-house medical vaccine expert for medical monitoring and Scientific Liaison
      • • Medical liaison to investigator sites including oversight of clinical monitoring
        • Provided medical monitoring input including CRF review, 24x7 accessibility to site personnel, assess enrollment waiver requests, SAE review, etc.
        • Safety Officer and Medical Representative on project teams
        • Medical consultant to clients
        • Business development/proposal writing/government contracting
• [Solvay pharmaceuticals unit] (currently Abbvie)
  • • Director, Clinical Development/Medical Affairs, Influenza 2006-2007
      • • Led an extended clinical team (both internal and CRO components), providing project and clinical trials management oversight, serving as primary author on clinical protocols, strategic documents including clinical development plans, DSMB/SMC charters, and all clinical documents required to support IND filing
        • Support and review of outcomes including safety data assessment
        • Generated and managed cost projections and budgetary oversight, providing strategic management and serving as a communication hub for clinical aspects of a $300 million USD federal contract to develop and license a cell-based influenza vaccine
        • Solvay’s US Government contract for cell-based influenza vaccine was terminated around the end of 2007. At which point the cell-based influenza vaccine project was dissolved.
• Summit Drug Development Services
  • • Senior Medical Director 2005-2006
      • • Directed due diligence assessments and strategic drug development planning and prepared regulatory submissions and implemented, monitored, and analyzed clinical trials for clients (oncology, vaccines, biologicals, cell/stem cell therapies)
        • Primary author of three pIND, two IND, an Appendix M submission
        • Served as proposal manager and primary author for a 129M USD federal contract submission focused on pandemic influenza.
• [AERAS Global TB Vaccine Foundation]
  • • Director, Business Development and Program Management 2004-2005
      • • Initially serving as consultant, provided leadership primarily focused on tuberculosis vaccine development and proposal development to NGO (B&M Gates), USG (CDC, NIH, DoD).
• [DynPort Vaccine Company, LLC]
  • • Associate Director, Clinical Research 2002-2003
      • • Served as liaison between product development teams and clinical research support groups
        • Prepared planning documents and product development plans
        • Participated in and supported safety review and assessment of smallpox vaccine product.
        • Identified new technologies relevant to product development teams, facilitating integration of same in product development plans
        • Created documents for clinical trials including investigator brochures. Prepared proposal solicitations, technical review of subcontractor proposals. Performed technical review of potential subcontractors, new technologies
        • Assisted business development group in strategic evaluation and planning concerning new business opportunities and managed in-house Publication
• [Intradigm Corporation]
  • • Co-Founder (one of three co-founders), CSO, Board of Director Member 2000-2001
      • • Helped to secure $2.3 million in V.C. funding, including monies from the Novartis Venture Fund, ETP Venture Capital Fund and the State of Maryland
        • Performed facilities set-up, infrastructure set-up and Intellectual Property Development
        • Business and technology development planning, including in-depth business and scientific plan
• USUHS
  • • Dept of Surgery, Clinical Breast Care Program (CBCP) through the Henry M. Jackson Foundation
    • Chief of Laboratory Science and Director of Tissue Banking 2000-2001
      • • Worked closely with architect firm to design space, set-up laboratory facilities for the Clinical Breast Care Project, including new facilities design (tissue banking facilities, laboratory, animal rooms, animal surgical suite, office suites) at USUHS and Windber Medical Center, PA
        • Hired faculty, technicians, staff for CBCP at both sites, including writing and initiating job descriptions, job interviews, hiring decisions, set-up for re-locations
        • Laboratory Supervisor: Tissue banking immunology, cell culture, gene transfer, genetic vaccination research, animal research
        • Set-up equipment and laboratory purchases, including vendor price quotes, equipment specs
        • Wrote initial budgets and supervised equipment purchases; wrote animal protocols and grants
• University of Maryland, Baltimore School of Medicine, Dept. of Pathology
  • • Assistant Professor 1997-2000
      • • Set-up and ran successful research laboratory in immunology (genetic vaccination) and gene transfer.
• University of California, Davis Department of Medical Pathology
  • • Assistant Professor 1993-1997
      • • Director and Founder, Gene Therapy Program (pulmonary, dermal, heart, liver, mucosal and parenteral vaccines)
• Medical Pathology, UC Davis Medical
  • • Research Fellow, Pathology Resident 1991-1993
• [Vical Incorporated]
  • • Research Scientist 1989
      • • Set up Vical’s molecular biology laboratory
        • Initiated and carried out research in non-viral gene therapy and DNA vaccination
        • Inventor of “naked DNA” gene therapy. (see issued patents for details)
        • Inventor of DNA vaccination (see issued patents for details)

TEACHING EXPERIENCE

PROFESSIONAL OFFICES AND MEMBERSHIPS

• American Society of Tropical Medicine and Hygiene Member (ASTMH): 2016-present
• Harvard Medical School Alumni
• Virginia Bio: 2016-present
• IEEE Genomics and Bioinformatics Working Group Member: 2002
• Northern Virginia Technology Council BioMedTech Committee: Co-chair: 2002 – 2003
• Intradigm, Corp. – a new start-up from Novartis, Inc.: Scientific Advisory Board: 2000 – 2001
• Novartis, Inc. (GTI/Systemix & Pharmacokinetics): Scientific Advisory Board and External Portfolio Reviewer: 1999 – 2001
• University of Maryland, Medical School: Pathology Education Policy Committee: 1999 – 2000
• UC Davis:
  • • Education Policy Committee Graduate Group in Comparative Pathology: 1996 – 1/1997
    • Member, Biochemistry and Molecular Biology Graduate Group: 1993 – 1/1997
    • Member, Comparative Pathology Graduate Group: 1995 – 1/1997
    • Boehringer Mannheim: Scientific Advisory Board: 1992 – 1993

EDUCATION

• HARVARD MEDICAL SCHOOL Global Clinical Scholars Research Training Program
  • • A year-long comprehensive program that combines on-site (London, Boston) and distance learning, with an average of 15h per week lecture and practicum exercises. 2015-2016.
• UC DAVIS, RESEARCH FELLOWSHIP, funded by Bank of America- Giannini Foundation Medical
  • • Research: 1992 – 1993
    • Postgraduate Fellowship Award
• UNIVERSITY OF CALIFORNIA, DAVIS, MEDICAL CENTER: 1992
  • • Clinical Pathology Internship
• NORTHWESTERN UNIVERSITY MEDICAL SCHOOL: 1991
  • • Doctor of Medicine
• UNIVERSITY OF CALIFORNIA, SAN DIEGO: 1988
  • • Master of Science, Biology
• UNIVERSITY OF CALIFORNIA, DAVIS: 1984
  • • Bachelor of Science, Biochemistry
• LICENSURE / CERTIFICATIONS
  • • Physician and Surgeon, State of Maryland License 1997-present. #DOO55466

SPECIALTY POSTGRADUATE COURSEWORK

• Walter Reed Healthcare System, Department of Clinical Investigation: Research Course: 2000
• USUHS: Rodent Handling and Techniques Laboratory: 2000
• USUHS: Animal Protocol Writing Workshop: 2000
• University of Maryland: CIPP 909 Research Ethics: 1999
• University of Maryland: Primate Handling Course: 1999
• University of Maryland: Biohazard US Training Course: 1999
• University of Maryland: Chemical Training Course: 1999
• University of Maryland: Radiation Training Course: 1999
• Anne Arundel Community College: Computer Programming in C: 1999
• Bowdoin College: Annual Course in Flow Cytometry: 1998
• University of Maryland Office of Human Resource Services: The Magic of Conflict” Conflict Management Workshop: 1998
• University of California, Davis: Laboratory Animal Handling Course: 1994 – 1997
• University of California, Davis: Biohazard US Training Courses: 1994 – 1997
• University of California, Davis: Chemical Training Courses: 1994 – 1997
• University of California, Davis: Radiation Training Courses: 1994 – 1997

EDITORIAL BOARDS

• Chairperson and scientific reviewer for Department of Defense, U.S. Army Medical Research and Materiel Command, for “Congressionally Directed Medical Research Programs (DMRDP). 2012
• Editor-In-Chief, Journal of Immune Based Therapies and Vaccines. 2009 – 2012, Editor: 2012-present
• Committee member and reviewer for NIH/NAIAD Committee for Development of Technologies that Accelerate the Immune Response to BioDefense Vaccines. 2011
• Chair and reviewer for NIH/NAIAD: Partnerships in Biodefense Immunotherapeutics. 2011
• NIH/NAIAD Committee member and reviewer for Development of Technologies to Facilitate the Use of, and Response to Biodefense Vaccines,” Special Emphasis panel. 2010
• Gene Therapy/Molecular Biology International Society. 1997 – 2014

Reviewer for:

• Numerous peer-reviewed journals on infectious disease, public health 2016
• Nucleic Acids Research: 2001 – 2002
• Molecular Therapy: 1999 – 2001
• NIH Study Section K01 Breast Cancer Study Section: July 1997
• NIDDK Special Emphasis Panel Review Committee for Competing Continuation Program Project:  April 1999 and April 1998
• NIAID Study Section “Innovative Grant Program for Approaches in HIV Vaccine Research”: June  1998

ACADEMIC HONORS

• DNA Vaccine Recognizes Robert W. Malone, MD, MS, 2013
• Trainee Investigator Award, American Federation for Clinical Research: 1993
• Bank of America – Giannini Foundation Medical Research Fellow: 1992 – 1993
• Henry Christian Award for Excellence in Research, American Federation for Clinical Research: 1992
• UCDMC Medical Scholars Grant: 1992 – 1993
• First Place, Northwestern AOA Research Symposium Competition for Medical Students: 1989
• USPHS Pre-Doctoral Fellowship: 1986 – 1988
• San Diego Supercomputer Grant for RNA Structure Modeling: 1988  [ I think this is it ... https://www.newspapers.com/image/390622176 ]
• Northwestern University MD/ PhD Scholarship: 1984 – 1986
• Dean's List, UC Davis: 1982 – 1984
• President's Undergraduate Fellowship Grant for Investigation of Oncogene Expression in Breast Tumor Tissue: 1983 – 1984
• Edmonson Summer Fellowship, Department of Pathology, UC Davis Medical School: 1984

PATENTS SUBMITTED:

• Application No.   /     Filing   Date   /    Title    /   MLB Docket No.
• 62/292,296   /   2/6/16   /   “Use of Anti-Malarial Compounds for prevention or Treatment of ZIKV Infection”   /   11985-5004
• 62/294,355   /   2/12/16   /   “Methods for Preventing Guillain-Barre Syndrome and/or Microcephaly”   /   115985-5001
• 62/301,147   /   2/29/16   /   “Methods for Preventing Diseases of the Central Nervous System”   /   115985-5002
• 62/304,211   /   3/5/16   /   “Methods for Preventing Diseases of the Central Nervous System”   /   11985-5005
• 62/304,214   /   3/5/16   /   “Methods for Preventing Diseases of the Central Nervous System”   /   11985-5006
• 62/330,142   /   4/30/16   /   “Compositions of Matter and Methods for Preventing Infection and Disease Caused by Arbovirus”   /   11985-5007
• 62/357,923   /   7/1/16   /   “Methods of Use of Piperaquine Metabolites”   /   115985-5009

PATENTS ISSUED:

1. Lipid-mediated polynucleotide administration to deliver a biologically active peptide and to induce a cellular immune response. [Dr. Philip Louis Felgner (born 1950)], [Dr. Jon Asher Wolff (born 1956)], Rhodes GH, Malone RW, [Dr. Dennis A. Carson (born 1936)]. US Pat. Ser. No. 7,250,404. issued 7/31/07
2. Lipid-mediated polynucleotide administration to reduce likelihood of subject's becoming infected. [Dr. Philip Louis Felgner (born 1950)], [Dr. Jon Asher Wolff (born 1956)], Rhodes, Malone RW, [Dr. Dennis A. Carson (born 1936)]. US Pat. Ser. No. 6,867,195. issued 3/15/05
3. Generation of an immune response to a pathogen. [Dr. Philip Louis Felgner (born 1950)], [Dr. Jon Asher Wolff (born 1956)], Rhodes GH, Malone RW, [Dr. Dennis A. Carson (born 1936)]. US Pat. Ser. No. 6,710,035. issued 3/23/04
4. Expression of exogenous polynucleotide sequences in a vertebrate, mammal, fish, bird or human [Dr. Philip Louis Felgner (born 1950)], [Dr. Jon Asher Wolff (born 1956)], Rhodes GH, Malone RW, [Dr. Dennis A. Carson (born 1936)]. US Pat. Ser. No. 6,673,776. issued 1/6/04
5. Methods of delivering a physiologically active polypeptide to a mammal. [Dr. Philip Louis Felgner (born 1950)], [Dr. Jon Asher Wolff (born 1956)], Rhodes GH, Malone RW, [Dr. Dennis A. Carson (born 1936)]. US Pat. Ser. No. 6.413.942 issued 7/2/02
6. Induction of a protective immune response in a mammal by injecting a DNA sequence. [Dr. Philip Louis Felgner (born 1950)], [Dr. Jon Asher Wolff (born 1956)], Rhodes GH, Malone RW, [Dr. Dennis A. Carson (born 1936)]. US Pat. Ser. No. 6,214,804 issued 4/10/01
7. DNA vaccines for eliciting a mucosal immune response. Malone, RW. and Malone, JG US Pat. Ser. No. 6,110,898 issued 8/29/00
8. Formulations and methods for generating active cytofectin: polynucleotide transfection complexes Nantz M, Bennett M, Balasubramaniam RP, Aberle AM, Malone RW. US Pat. Ser. No. 5,925,623  7/20/99
9. Cationic Transport Reagents. Bennett M, Nantz M, and Malone RW. US Pat. Ser. No. 5,892,071 issued 4/06/99
10. Polyfunctional cationic cytofectins, formulations and methods for generating active cytofectin: polynucleotide transfection complexes. Nantz M, Bennett M, Balasubramaniam RP, Aberle AM, Malone RW. US Pat. Ser. No. 5,824,812 issued 10/20/98
11. Cationic Transport Reagents. Bennett M, Nantz M, and Malone RW. US Pat. Ser. No. 5,744,625  issued 4/28/98
12. Generation of antibodies through lipid mediated DNA delivery. [Dr. Philip Louis Felgner (born 1950)], [Dr. Jon Asher Wolff (born 1956)], Rhodes GH, Malone RW, [Dr. Dennis A. Carson (born 1936)]. US Pat. Ser. No. 5,703,055. issued 12/30/97
13. Induction of a protective immune response in a mammal by injecting a DNA sequence. [Dr. Philip Louis Felgner (born 1950)], [Dr. Jon Asher Wolff (born 1956)], Rhodes GH, Malone RW, [Dr. Dennis A. Carson (born 1936)]. US Pat. Ser. No. 5,589,466. issued 12/31/96
14. Delivery of exogenous DNA sequences in a mammal . [Dr. Philip Louis Felgner (born 1950)], [Dr. Jon Asher Wolff (born 1956)], Rhodes GH, Malone RW, [Dr. Dennis A. Carson (born 1936)]. US Pat. Ser. No. 5,580,859. issued 12/3/96
15. Cationic Transport Reagents. Bennett M, Nantz M, and Malone RW. US Pat. Ser. No. 5,527,928. issued 6/18/96

PUBLICATIONS

1. Malone RW, Soloveva V, Bulitta JB, Jiao Y, Glasspool-Malone J, Dean N, et al. Accelerated Discovery and Development of Re-purposed Licensed Drugs for Zika Virus Prophylaxis and Therapy

2. Schneider AB, Malone RW, Guo J, Homan J, Linchangco G, Witter Z, et al. Molecular evolution of Zika virus as it crossed the Pacific to the Americas. Cladistics. 2016; 12: 10.1111/cla.12178
3. Malone RW, Homan J, Callahan MV, Glasspool-Malone J, Damodaran L, Schneider Ade B, et al. Zika Virus: Medical Countermeasure Development Challenges. PLoS Negl Trop Dis. 2016;10(3):e0004530.
4. Klase ZA, Khakhina S, Schneider Ade B, Callahan MV, Glasspool-Malone J, Malone R. Zika Fetal Neuropathogenesis: Etiology of a Viral Syndrome. PLoS Negl Trop Dis. 2016;10(8):e0004877.
5. Homan J, Malone RW, Darnell SJ, Bremel RD. Antibody mediated epitope mimicry in the pathogenesis of Zika virus related disease. PLoS Negl Trop Dis, submitted to (preprint in bioRxiv). 2016.
6. De Groot AS, Einck L, Moise L, Chambers M, Ballantyne J, Malone RW, et al. Making vaccines "on demand": a potential solution for emerging pathogens and biodefense? Hum Vaccin Immunother.  2013;9(9):1877-84.
7. Byrnes CK, Malone RW, Akhter N, Nass PH, Wetterwald A, Cecchini MG, et al. Electroporation enhances transfection efficiency in murine cutaneous wounds. Wound Repair Regen. 2004;12(4):397-403.
8. Glasspool-Malone J, Steenland PR, McDonald RJ, Sanchez RA, Watts TL, Zabner J, et al. DNA transfection of macaque and murine respiratory tissue is greatly enhanced by use of a nuclease inhibitor. J Gene Med. 2002;4(3):323-2.
9. Glasspool-Malone J, Steenland P, McDonald RJ, Sanchez RA, Watts TL, Zabner J, et al. Marked enhancement of macaque respiratory tissue transfection by aurintricarboxylic acid. J Gene Med.  2002;4(3):323-2.
10. Glasspool-Malone J, Malone RW. Enhancing direct in vivo transfection with nuclease inhibitors and pulsed electrical fields. Methods Enzymol. 2002;346:72-91.
11. Drabick JJ, Glasspool-Malone J, King A, Malone RW. Cutaneous transfection and immune responses to intradermal nucleic acid vaccination are significantly enhanced by in vivo electropermeabilization. Mol Ther. 2001;3(2):249-55.
12. Somiari S, Glasspool-Malone J, Drabick JJ, Gilbert RA, Heller R, Jaroszeski MJ, et al. Theory and in vivo application of electroporative gene delivery. Mol Ther. 2000;2(3):178-87.
13. Glasspool-Malone J, Somiari S, Drabick JJ, Malone RW. Efficient nonviral cutaneous transfection. Mol Ther. 2000;2(2):140-6.
14. Colosimo A, Goncz KK, Holmes AR, Kunzelmann K, Novelli G, Malone RW, et al. Transfer and expression of foreign genes in mammalian cells. Biotechniques. 2000;29(2):314-8, 20-2, 24 passim.
15. Kisich KO, Malone RW, Feldstein PA, Erickson KL. Specific inhibition of macrophage TNF-alpha expression by in vivo ribozyme treatment. J Immunol. 1999;163(4):2008-16.
16. Glasspool-Malone J, Malone RW. Marked enhancement of direct respiratory tissue transfection by aurintricarboxylic acid. Hum Gene Ther. 1999;10(10):1703-13.
17. Berlyn KA, Ponniah S, Stass SA, Malone JG, Hamlin-Green G, Lim JK, et al. Developing dendritic cell polynucleotide vaccination for prostate cancer immunotherapy. J Biotechnol. 1999;73(2-3):155-79.
18. Ahearn A, Malone RW. Models of Cationic Liposome Mediated Transfection. Gene Therapy and Molecular Biology (Dec., 1999) Vol 4. Gene Therapy and Molecular Biology 1999;4.
19. Malone JG, Bergland PJ, Liljestrom P, Rhodes GH, Malone RW. Mucosal immune responses associated with polynucleotide vaccination. Behring Inst Mitt. 1997(98):63-72.
20. Bennett MJ, Aberle AM, Balasubramaniam RP, Malone JG, Malone RW, Nantz MH. Cationic lipid-mediated gene delivery to murine lung: correlation of lipid hydration with in vivo transfection activity. J Med Chem. 1997;40(25):4069-78.
21. Montbriand PM, Malone RW. Improved method for the removal of endotoxin from DNA. J  Biotechnol. 1996;44(1-3):43-6.
22. Freedland SJ, Malone RW, Borchers HM, Zadourian Z, Malone JG, Bennett MJ, et al. Toxicity of cationic lipid-ribozyme complexes in human prostate tumor cells can mimic ribozyme activity. Biochem Mol Med. 1996;59(2):144-53.
23. Bennett MJ, Aberle AM, Balasubramaniam R, P., Malone JG, Nantz MH, Malone RW.  Considerations for the design of improved cationic amphiphile-based transfection reagents. . Journal of Liposome Research 1996;6(3):545-65.
24. Balasubramaniam RP, Bennett MJ, Aberle AM, Malone JG, Nantz MH, Malone RW. Structural and functional analysis of cationic transfection lipids: the hydrophobic domain. Gene Ther. 1996;3(2):163-72.
25. Aberle AM, Bennett MJ, Malone RW, Nantz MH. The counterion influence on cationic lipidmediated transfection of plasmid DNA. Biochim Biophys Acta. 1996;1299(3):281-3.
26. Schenborn E, Oler J, Goiffon V, Balasubraniam R, Bennett M, Aberle A, et al. Tfx-50 Reagent, a new transfection reagent for eukaryotic cells. 1995.
27. Hickman MA, Malone RW, Sihc TR, Akitad GY, Carlsonc DM, Powelle JS. Hepatic gene expression after direct DNA injection. Advanced Drug Delivery Reviews. 1995;17(3):265-71.
28. Bennett† MJ, Malone RW, Nantz MH. A flexible approach to synthetic lipid ammonium salts for polynucleotide transfection. Tetrahedron Letters. 1995;36(13):2207-10.
29. Bennett MJ, Nantz MH, Balasubramaniam RP, Gruenert DC, Malone RW. Cholesterol enhances  cationic liposome-mediated DNA transfection of human respiratory epithelial cells. Biosci Rep.   1995;15(1):47-53.
30. Malone RW, Hickman MA, Lehmann-Bruinsma K, Sih TR, Walzem R, Carlson DM, et al.  Dexamethasone enhancement of gene expression after direct hepatic DNA injection. J Biol Chem.  1994;269(47):29903-7.
31. Hickman MA, Malone RW, Lehmann-Bruinsma K, Sih TR, Knoell D, Szoka FC, et al. Gene expression following direct injection of DNA into liver. Hum Gene Ther. 1994;5(12):1477-83.
32. Dwarki VJ, Malone RW, Verma IM. Cationic liposome-mediated RNA transfection. Methods Enzymol. 1993;217:644-54.
33. [Dr. Jon Asher Wolff (born 1956)], Malone RW, Williams P, Chong W, Acsadi G, Jani A, et al. Direct gene transfer into mouse muscle in vivo. Science. 1990;247(4949 Pt 1):1465-8.
34. Malone RW, [Dr. Philip Louis Felgner (born 1950)], Verma IM. Cationic liposome-mediated RNA transfection. Proc Natl Acad Sci U S A. 1989;86(16):6077-81. [SEE BELOW IN EVIDENCE TIMELINE]
35. Malone RW. mRNA Transfection of cultured eukaryotic cells and embryos using cationic liposomes. Focus. 1989;11:61-8.
36. [Dr. Murray Briggs Gardner (born 1945), Malone RW, Morris DW, Young LJ, Strange R, Cardiff RD, et al. Mammary tumors in feral mice lacking MuMTV DNA. J Exp Pathol. 1985;2(2):93-8.
37. Faulkin LJ, Mitchell DJ, Young LJ, Morris DW, Malone RW, Cardiff RD, et al. Hyperplastic and neoplastic changes in the mammary glands of feral mice free of endogenous mouse mammary tumor virus provirus. J Natl Cancer Inst. 1984;73(4):971-82.

PUBLISHED ABSTRACTS: Over 40 published  ( CHAIRPERSON/ORAL PRESENTATIONS BY INVITATION: Over 40 Invitations ) 

• Chairperson, Repurposing drugs. International Conference on Zika Virus. Washington, DC Feb 22- 25, 2017.
• Accelerated Discovery and Development of re-purposed licensed drugs for Zika virus outbreak antiviral rophylaxis and therapy. International Conference on Zika Virus. Washington, DC Feb 22- 25, 2017.
• Bridging the Sciences: Zika Virus. Speaker. Zika Virus: Accelerating Development of Medical Countermeasures by Re-purposing Licensed Drugs, Emery, Atlanta, GA 1-3 May, 2016
• World Vaccine Conference. Speaker/Round table- Zika virus: Challenges for Medical Countermeasure Development Washington, DC. 29-31 March, 2016
• The World Health Organization (WHO) Consultation for Zika Virus: Research and Development. Presentation of Drug Development TPP. Geneva, Switzerland. 12-14 March, 2016
• Vaccines R&D, Keynote Speaker: Ebola Vaccine in 12 months, Global Village, and the Need for Speed. Baltimore, MD. 2-4 November, 2015
• World Vaccine Conference Speaker. Current USG contracting Opportunities and Initiatives from the point of View of Vaccine Developers. Washington, DC. 24-26 March, 2014
• World Vaccine Conference Session Chair, Washington, DC. 24-26 March, 2014 Preclinical and Clinical Vaccine Research.
• Conference Organizer and Coordinator: MODELING WORKSHOP, 2013
• The World Health Organization (WHO) Global Action Plan for Influenza Vaccines. Robert W Malone, MD, MS "Vaccine Production Strategies: Ensuring Alignment and Sustainability" Geneva, Switzerland. 12-14 July 2011 Invited speaker [ downloaded here : https://www.who.int/influenza_vaccines_plan/resources/malone.pdf   2011-07-world-health-organization-influenza-vaccines-malone-production-alignment-sustainability.pdf  .....  video recording is here https://vimeo.com/30252721  ] 

RECENT STUDY SECTIONS:

• Chairperson and scientific reviewer for Department of Defense, U.S. Army Medical Research and Materiel Command, for “Congressionally Directed Medical Research Programs (CDMRP), Defense Medical Research And Development Program (DMRDP), 2012
• Chairperson and reviewer for NIH/NAIAD Committee on Partnerships in Biodefense Immunotherapeutics, Fall 2011.
• Committee member and reviewer for NIH/NAIAD Committee for Development of Technologies that Accelerate the Immune Response to BioDefense Vaccines, Fall 2011.
• NIH/NAIAD Committee member and reviewer for Development of Technologies to Facilitate the Use of, and Response to Biodefense Vaccines,” Special Emphasis panel, 2010

BOOK CHAPTERS

• Malone RW. "Present and Future Status of Gene Therapy.' Intro Chapter in Advanced Gene Delivery: From Concepts to Pharmaceutical Products. Editor: Allain Rolland. Harwood Academic Pub. 1998.
• Malone RW. Toxicology of non-viral gene transfer. Editor, Walsh B. In: Non Viral Therapeutics:  Advances, Challenges and Applications for Self-Assembling Systems. Boston: IBC’s Biomedical Library Series. (1996) 4.1

Five Year Performance: Grants and Contracts   ( Grants and Contracts   /   Role   /   Year   /   Awarded amount  ) 

• Awarded to What a Smoke: Development of a Standardized Electronic Cigarette for Clinical Research
  • • Capture manager, Proposal manager and Technical Proposal lead author
    • 2016   /   In Negotiation
• IDIQ Award to TASC by U.S. Army for Medical Product Research and Development. (W81XWH-15-D-0042)
  • • Capture manager, lead author.
    • 2016   /   $5 Billion
• Awarded to [BioProtection Systems Inc.] (New Link Genetics): DTRA Contract for Ebola Medical Countermeasure.
  • • Capture manager, Proposal manager lead author.
    • 2015   /   $8 Million base, $5.2 M option
• Awarded to What a Smoke: Development of a Standardized Electronic Cigarette for Clinical Research
  • • Proposal manager and Technical Proposal lead author.
    • 2015   /   $350,000
• Award to [BioProtection Systems Inc.] (Newlink): contract to support the development and manufacturing of its VSV-EBOV (Ebola) vaccine candidate, including a new 330-subject Phase 1b study. Options for base grant.
  • • Capture Manager, Proposal manager and Technical Proposal lead author.
    • 2015    /   $18 Million
• Award to [BioProtection Systems Inc.] (Newlink): contract to support the development and manufacturing of its VSV-EBOV (Ebola) vaccine candidate, including a new 330-subject Phase 1b study.
  • • Capture Manager, Proposal manager and Technical Proposal lead author.
    • 2014   /   $30 Million
• Award to Soligenix: NIAID contract for "Development of Vaccine Formulations Effective against NIAID Priority Pathogens." (HHSN272201400039C)
  • • Proposal manager and Technical Proposal lead author.
    • 2014   /   $24.7 Million
• Awarded to Kai Research and Benchmark Research: contract for Support Annual Influenza Vaccine Serology Testing - for the CDC.
  • • Developed the plan, assembled the team, formulated the contract and helped produce the final product.
    • 2013   /   $2.8 million base period, 5 total renewable years
• IDIQ Award to Excelis: Combating Weapons of Mass Destruction Research and Technology Development from Defense Threat Reduction Agency. (HDTRA1-14-D-0005)
  • • Capture manager, lead author for CBRN section.
    • 2013   /   $4 Billion
• Awarded to Nanotherapeutics: Defense Department contract for Medical Countermeasures Advanced Development.
  • • Original thought leader, Pre-Proposal manager and Technical Proposal lead author.
    • 2013   /   $427 million
• Awarded to Soligenix: BARDA Contract (Advanced Development of OrbeShield in GI ARS)
  • • Tech Watch: proposal development. Proposal manager and Technical Proposal lead author.
    • 2013   /   $26 million
• Awarded to Vaxin: Contract by BARDA to develop next generation Anthrax Vaccine.
  • • Proposal manager and principal scientific author.
    • 2011   /   $21 million
• TOTAL Flexible IDIQ Awards 2011-2016 9 Billion
• TOTAL Award Amounts (excluding IDIQ awards) 2011-2016 562 Million

PROFESSIONAL EXPERIENCE

An original inventor of core mRNA and DNA vaccination technology (1989), Dr. Malone is a specialist in clinical research, medical affairs, regulatory affairs, project management, proposal management (large grants and contracts), vaccines and biodefense. This includes writing, developing, reviewing and managing vaccine, bio-threat and biologics clinical trials and clinical development strategies. He has been involved in developing, designing, and providing oversight of approximately forty phase 1 clinical trials and twenty phase 2 clinical trials, as well as five phase 3 clinical trials. He has served as medical director/medical monitor on both phase 1, phase 2 and phase 3 clinical trials, including those run at a well known vaccine-focused Clinical Contract Research Organizations. He has served as principal investigator on some of these. Examples of his infectious disease pathogen advanced (clinical phase) development oversight experience include HIV, Influenza (seasonal and pandemic), Plague, Anthrax, VEE/EEE/WEE, Tularemia, Tuberculosis, Ebola, Zika, Ricin toxin, Botulinum toxin, and Engineered pathogens. In many cases, this experience has included vaccine product development, manufacturing, regulatory compliance, and testing (manufacturing release and clinical) aspects. In most cases, his oversight responsibilities have included clinical trial design, regulatory and ethical compliance, and laboratory assay strategy, design, testing and performance.

Dr. Malone has a history of assembling and managing expert teams that focus on solving complicated biodefense challenges to meet US Government requirements. He was instrumental in enabling the PHAC/rVSV ZEBOV (“Merck Ebola”) vaccine to move forward quickly towards BLA and (now recently granted) licensure. Dr. Malone got the project on track in support of DoD/DTRA and NewLink Genetics, recruited organizations to team with USAMRIID/WRAIR to develop the immunoassays, put WHO and Norwegian government philanthropic leadership in touch with Pentagon leadership to expedite the initial WRAIR clinical and ring vaccination trials, recruited a management team, recruited Merck vaccines to purchase the product candidate from NewLink, helped write and edit the clinical trials developed by the World Health Organization and lead the development of the BARDA and DTRA contracts - yielding over 200M$ in resources. Dr. Malone’s early involvement in this project allowed for the Merck vaccine to be developed very rapidly.

Currently, Dr. Malone is leading a large team since January 10, 2020, focused on clinical research design, drug development, computer modeling and mechanisms of action of repurposed drugs for COVID-19 treatment. This work has included multiple manuscripts summarizing most recent findings relating to famotidine and overall insights into the mechanism of COVID-19 disease, and others focused on celecoxib and famotidine are being reviewed for publication. He has developed and wrote the initial clinical trial design: A Single Center, Randomized, Double Blinded Controlled Crossover Observational Outpatient Trial of the Safety and Efficacy of Oral Famotidine for the Treatment of COVID-19 in Non-Hospitalized Symptomatic Adults. Another project he has been involved with is a DTRA/DOMANE-funded development and performance of a virtual outpatient clinical trial designed to test new monitoring and data capture technology while using COVID19 as a live-fire example. He has helped open two IND fo famotidine and celecoxib use for treatment and prevention of COVID19 disease including an associated drug master file, and has enabled teaming/pharmaceutical supply arrangements with two major pharmaceutical firms.

Dr. Malone is an internationally recognized scientist and is the original inventor of mRNA Vaccination, DNA Vaccination, and multiple non-viral DNA and RNA/mRNA delivery technologies. Dr. Malone holds numerous fundamental domestic and foreign patents in the fields of gene delivery, delivery formulations, and vaccines: including DNA and RNA/mRNA vaccines. His expertise includes virology, immunology, molecular biology, pathology and pharmacology.

Scientifically trained at UC Davis, UC San Diego, and at the Salk Institute Molecular Biology and Virology laboratories, Dr. Malone received his medical training at Northwestern University (MD) and Harvard University (Clinical Research Post Graduate Fellowship) medical schools, and in Pathology at UC Davis. Dr. Malone is currently finishing up his board certification in medical affairs (BCMAS).

He has extensive research and development experience (bench to bedside) in the areas of pre-clinical discovery research, clinical trials, vaccines, gene therapy, bio-defense, repurposing drugs for infectious diseases, high throughput screening and immunology. He has over twenty years of management and leadership experience in academia, pharmaceutical and biotechnology industries, as well as in governmental and non-governmental organizations. He often serves as study section chairperson for NIAID contract study sections relating to biodefense medical product development. He is currently a topic editor for the journal Frontiers in Pharmacology, in the area of “Treating COVID-19 With Currently Available Drugs.”

Dr. Malone has approximately 100 peer-reviewed publications and published abstracts and has about 12,000 citations of his peer reviewed publications, as verified by Google Scholar. His google scholar ranking is “outstanding” for impact factors. He has been an invited speaker at over 50 conferences, has chaired numerous conferences and he has sat on or served as chairperson on numerous NIAID and DoD study sections.

SUMMARY OF ACCOMPLISHMENTS / SKILLS

• Inventor of mRNA and DNA vaccination.
• Inventor of lipid mediated and naked mRNA delivery (transfection).
• Inventor of in-vivo electroporation (particularly for skin delivery).
• A senior executive and scientist with a highly successful track record of leading bench and discovery research through FDA Phase I, II, and III clinical trials, protocol development and submission, and related regulatory submissions including pIND and IND.
• Significant expertise in drug development and delivery.
• Specialist in Medical Affairs.
• Special in Regulatory Affairs.
• Domestically trained, Maryland Licensed Physician/Scientist.
• Experienced capturing and managing large federal contracts (including BARDA) with over 9 billion in ID/IQ awards and almost a billion USD in government contracts won and/or managed in the last decade.
• Expertise in pathology, infectious disease, pandemic clinical trials, influenza, regulatory affairs, project management, biodefense, HIV and Ebola. A verified list of capture is available upon request.
• Significant expertise with federal contracting, grants, international NGO health related research and development coupled with professional relationships at CDC, DoD, HHS (BARDA, CDC, FDA and NIAID).
• Prior and current service on many federal study sections and oversight boards involving infectious disease, vaccine, and biodefense.
• Experienced and formally trained as a Business Development Professional, project manager, capture/proposal manager, color team reviewer and editor for projects valued from 10M$ up to 1B$ US, with experience managing processes and teams in a wide variety of non-profit and for-profit corporate cultures including both matrix and traditional environments.
• Highly skilled in fostering a culture of innovative problem solving within project teams.
• DoD Secret Clearance authorized.
• Expert witness experience, with extensive training from some of the top attorneys/law firms in the USA.
• Rated outstanding for impact factors, by Google scholar.
• Graduated from the Harvard Medical School Global Clinical Scholars Research Training Program with distinction, a year-long program focused on international clinical research. This program combines onsite

• Dr. Malone is currently working on becoming board certified in medical affairs (BCMAS). The BCMAS program is the certification program developed by the Accreditation Council for Medical Affairs (ACMA).

• Led a large team since January 10, 2020, focused on drug development, computer modeling and mechanisms of action for COVID-19 and is now preparing a manuscript summarizing most recent findings relating to famotidine and overall insights into the mechanism of COVID-19 disease.
• Accelerated COVID-19 Therapeutic Interventions and Vaccines: ACTIV Therapeutics Clinical Working Group, NIH. Invited Participant. June, 2020-present.
• Clinical trials protocol development: Developed and wrote initial clinical trial design: A Single Center, Randomized, Double Blinded Controlled Crossover Observational Outpatient Trial of the Safety and Efficacy of Oral Famotidine for the Treatment of COVID-19 in Non-Hospitalized Symptomatic Adults.
• Proposed is a DOMANE/WRAIR joint development and performance of outpatient clinical trial designed to test new monitoring and data capture technology while using COVID19 as a live-fire example.
• Opening IND for famotidine use for treatment and prevention of COVID19 disease with associated am drug master file.
• Principal Regulatory Consultant, Clinical Network Services (CNS)/Novotech, 2018-2019.

• Served as an expert witness with specialized training, 2017 - present.
• Ebola vaccine project for NewLink/[BioProtection Systems Inc.] (rVSVdG ZEBOV Ebola vaccine project), resulting in well over 100M USD non-dilutive capital to NL/BPS. This also included working with the World Health Organization as well as initial set up of the licensing deal to Merck Vaccines of the Ebola vaccine.
• Served as Medical Director, [Beardsworth Consulting Group, Inc.], half time position on retainer, 2010 – 2013.
• Service on federal biotechnology/vaccines proposal study sections (multiple).
• Served as Editor-In-Chief of Journal of Immune Based Therapies and Vaccines 2007-2012
• Service on Safety Monitoring Committee, Phase 1 safety/immunogenicity of novel Influenza vaccine
• Consulting support for multiple vaccine-focused clinical sites in US and Latin America.
• Served as Medical Director, Vaccines with Accelovance, Inc. (2008 – 2009).
• Served as medical monitor for multiple seasonal and pandemic (H1N1) studies.
• Review and edit clinical protocols.
• Examples of multi-year contract clients include Accelovance, Alchem Laboratories, Avancer, [Beardsworth Consulting Group, Inc.], Chesapeake Perl, Corium, DOAR, ITS, ITT-Exelis, [EpiVax], Jean Brown Research, Opgen, Quest Diagnostics (Focus), PaxVax, SAI, Soligenix, TASC, Univ of MA.
• Commercial intelligence work for two of the largest pharmaceutical companies in the world (subcontractor).
• Partnering with Galloway and Associates ([Dr. Darrell Ray Galloway (born 1946)]) 2012-2014.
• Acting as Managing Director, Clinical Development and Government Affairs for the Avancer Group. April 2012 – 2016.
• Proposal development (patch-based vaccine delivery, Tularemia vaccine, CDC contract for clinical trials site development, international government and NGO contract and grant solicitations) – Aeras Global TB Vaccine Foundation 2003-2005.
• Proposal development (plague vaccine- HHS), Technical diligence – VaxGen Corporation.
• Consulting services for [EpiVax], 2005-2018 (member, Scientific Advisory Board), 2020.
• Consulting services for - [Aldevron] 

• 2001-2005 (operating as Gene Delivery Alliance).
  • • Business and proposal development in the areas of Bioinformatics and Life Sciences (including telemedicine) and research at the University of Bern, Switzerland.
    • Consulting services for Molecular Histology, Inc. with the title of Medical Director.
    • Collaboration with [Inovio Pharmaceuticals, Incorporated], including incorporation of company in the USA.
    • Consulting services for MSD, Inc. for business/ technology development planning.

• Alchem Laboratories
  • Chief Medical Officer
  • This position was as a consultant, but then full time FTE. Consulting for Alchem and/or its CEO: 2012 – 2019. CMO 11/2019 to 4/2020.
  • • Led a high through-put screening and research team for drug development 2019-2020.
  • • Dr. Malone began modeling and focusing on the Plpro (papain-like protease) and Mpro (main protease) of then novel coronavirus (now SARS-CoV-2) using computational tools including Modeller to generate homology-modeled crystal structures for the SARS-CoV-2 Plpro and Mpro. Which generated a candidate list for COVID-19, which was reduced to a few candidates, based on binding sites, safety, licensure, efficacy, bioavailability of drug candidates.
  • • Lead the discovery and development of famotidine for the Treatment of COVID-19.
  • • Technical Lead/writer for funded full proposal under BAA-18-100-SOL-00003 Amendment 15 entitled: “A Multi-site, Randomized, Double-Blind, Multi-Arm Historical Control, Comparative Trial of the Safety and Efficacy of Hydroxychloroquine, and the Combination of Hydroxychloroquine and Famotidine for the Treatment of COVID-19 in Hospitalized Adults.”
  • • Developed and wrote initial clinical trial design for a comparative trial of the safety and efficacy of hydroxychloroquine, and the combination of hydroxychloroquine and famotidine for the treatment of COVID-19 in hospitalized adults.
• Atheric Pharmaceutical, LLC
• CEO, and Co-founder.

• Feb 2016-Dec 2017. Atheric™ Pharmaceutical LLC was a biopharmaceutical company focused on the rapid development and commercialization of re-purposed drugs to prevent and treat Zika and other Flavivirus disease. Optimization of high through-put screening techniques for anti-viral drug development.
• Kennesaw State University
  • Adjunct Associate Professor 2009-2013
• [Beardsworth Consulting Group, Inc.]
  • Medical Director, Vaccines (RW Malone MD, LLC under contract to Beardsworth),   2010-2013
  • Dr. Malone functioned as the in-house medical vaccine expert for medical monitoring and Scientific Liaison
    •  Medical liaison to investigator sites including oversight of clinical monitoring
    • Provided medical monitoring input including CRF review, 24x7 accessibility to site personnel, assess enrollment waiver requests, SAE review, etc.
    • Safety Officer and Medical Representative on project teams
    • Medical consultant to clients
    • Business development/proposal writing/government contracting
• Solvay Pharmaceuticals, Inc [aka Solvay pharmaceuticals unit]  (currently Abbvie)
  • • Director, Clinical Development & Medical Affairs, Influenza 2006-2008
      • • Led an extended clinical team (both internal and CRO components), providing project and clinical trials management oversight, serving as primary author on clinical protocols, strategic documents including clinical development plans, DSMB/SMC charters, and all clinical documents required to support IND filing. Support and review of outcomes including safety data assessment
        • Generated and managed cost projections and budgetary oversight, providing strategic management and serving as a communication hub for clinical aspects of a $300 million USD federal contract to develop and license a cell-based influenza vaccine
        • Solvay’s US Government contract for cell-based influenza vaccine was terminated around the end of 2007.
        • At which point the cell-based influenza vaccine project was dissolved.
• Summit Drug Development Services
  • • Senior Medical Director 2005-2006
      • • Directed due diligence assessments and strategic drug development planning and prepared regulatory submissions and implemented, monitored, and analyzed clinical trials for clients (oncology, vaccines, biologicals, cell/stem cell therapies). Primary author of three pIND, two IND, an Appendix M submission.
        • Served as proposal manager and primary author for a 129M USD federal contract submission focused on pandemic influenza.
• [AERAS Global TB Vaccine Foundation]
  • • Director, Business Development and Program Management 2004-2005
      • • Initially serving as consultant, provided leadership primarily focused on tuberculosis vaccine development and proposal development to NGO (B&M Gates), USG (CDC, NIH, DoD).
• [DynPort Vaccine Company, LLC]
  • • Associate Director, Clinical Research 2002-2003
      • • Served as liaison between product development teams and clinical research support groups.
        • Prepared planning documents and product development plans.
        • Participated in and supported safety review and assessment of smallpox vaccine product.
        • Identified new technologies relevant to product development teams, facilitating integration of same in product development plans.
        • Created documents for clinical trials including investigator brochures. Prepared proposal solicitations, technical review of subcontractor proposals. Performed technical review of potential subcontractors, new technologies.
        • Assisted business development group in strategic evaluation and planning concerning new business opportunities and managed in-house Publication.
• [Intradigm Corporation]
  • • Co-Founder (one of three co-founders), CSO, Board of Director Member 2000-2001
      • • Intradigm was a biotechnology company that develops gene therapeutic technology based on RNA interference. Intradigm merged with Silence Technologies in 2009 and the merged company is now publicly traded. Silence Technologies is involved in developmental research of targeted RNAi therapeutics for the treatment of serious diseases.
        • Dr. Malone co-founded and helped to secure $2.3 million in V.C. funding, including monies from the Novartis Venture Fund, ETP Venture Capital Fund and the State of Maryland. Performed facilities set-up, infrastructure set-up and Intellectual Property Development. Business and technology development planning, including in-depth business and scientific plan.
• Uniformed Services University of the Health Sciences
  • Dept of Surgery, Clinical Breast Care Program (CBCP) through the Henry M. Jackson Foundation
  • Adjunct Associate Professor
  • Chief of Laboratory Science and Director of Tissue Banking 2000-2001
  • Worked closely with architect firm to design space, set-up laboratory facilities for the Clinical Breast Care Project, including new facilities design (tissue banking facilities, laboratory, animal rooms, animal surgical suite, office suites) at USUHS and Windber Medical Center, PA
  • Hired faculty, technicians, staff for CBCP at both sites, including writing and initiating job descriptions, job interviews, hiring decisions, set-up for re-locations
  • Laboratory Supervisor: Tissue banking immunology, cell culture, gene transfer, genetic vaccination research, animal research.
• University of Maryland, Baltimore School of Medicine, Dept. of Pathology
  • Assistant Professor 1997-2000
  • Set-up and ran successful research laboratory in immunology (genetic vaccination) and gene transfer.
• University of California, Davis Department of Medical Pathology
  • 1991-1997
  • Assistant Professor 1993-1997
  • Director and Founder, Gene Therapy Program (pulmonary, dermal, heart, liver, mucosal and parenteral vaccines).
  • Research Fellow, Pathology Resident 1991-1993

• [Vical Incorporated]
  • Research Scientist 1989
    • Set up Vical’s molecular biology laboratory.
    • Initiated and carried out research in non-viral gene therapy and DNA vaccination.
    • Inventor of “naked DNA” gene therapy. (see issued patents for details).
    • Inventor of DNA vaccination (see issued patents for details).
    • Inventor of “mRNA” gene therapy. Salk institute.
    • Inventor of mRNA vaccination. Salk institute.

LICENSURE / CERTIFICATIONS

• Physician and Surgeon, State of Maryland License 1997-present. #DOO55466

BOARD OF DIRECTOR POSITIONS:

• Discovery Cure, Inc. Founding Board of Director. 2018-2020
• [EpiVax], Scientific Advisory Board, 2012-2019.

EDUCATION

• HARVARD MEDICAL SCHOOL Global Clinical Scholars Research Training Program (fellowship)
  • • A year-long comprehensive program that combines on-site (London, Boston) and distance learning, with an average of 15h per week lecture and practicum exercises. 2015-2016. Graduation with distinction (top 5% of graduating class).
• UNIVERSITY OF CALIFORNIA, DAVIS: RESEARCH FELLOWSHIP, 1992 – 1993
  • • Postgraduate Fellowship Award
• UNIVERSITY OF CALIFORNIA, DAVIS MEDICAL CENTER: 1992
  • • Clinical Pathology Internship
• NORTHWESTERN UNIVERSITY MEDICAL SCHOOL: 1991
  • • Doctor of Medicine
• UNIVERSITY OF CALIFORNIA, SAN DIEGO: 1988
  • • Master of Science, Biology
• UNIVERSITY OF CALIFORNIA, DAVIS: 1984
  • • Bachelor of Science, Biochemistry

TEACHING EXPERIENCE

• Kennesaw State University
• Associate Professor:

PROFESSIONAL OFFICES AND MEMBERSHIPS

• Royal Society of Medicine, Fellow 2020-Present.
• Harvard Medical School Alumni, 2016- present.
• American Society of Tropical Medicine and Hygiene Member (ASTMH): 2016-2018.
• Virginia Bio: 2016-2018
• IEEE Genomics and Bioinformatics Working Group Member: 2002
• Northern Virginia Technology Council BioMedTech Committee: Co-chair: 2002 – 2003
• Intradigm, Corp. – a new start-up from Novartis, Inc.: Scientific Advisory Board: 2000 – 2001
• Novartis, Inc. (GTI/Systemix & Pharmacokinetics): Scientific Advisory Board and External Portfolio Reviewer: 1999 – 2001
• University of Maryland, Medical School: Pathology Education Policy Committee: 1999 – 2000
• UC Davis:
  • • Education Policy Committee Graduate Group in Comparative Pathology: 1996 – 1/1997
    • Member, Biochemistry and Molecular Biology Graduate Group: 1993 – 1/1997
    • Member, Comparative Pathology Graduate Group: 1995 – 1/1997
    • Boehringer Mannheim: Scientific Advisory Board: 1992 – 1993

EDITORIAL BOARDS

• Topic Editor, Frontiers in Pharmacology (Respiratory Pharmacology): “Treating COVID-19 with Currently Available Drugs,” 2020-2021.
• Editor-In-Chief, Journal of Immune Based Therapies and Vaccines. 2009 – 2012, Editor: 2012.
• Gene Therapy/Molecular Biology International Society. 1997 – 2014.
• Reviewer for: Numerous peer-reviewed journals on infectious disease, public health 2016 to present.
• Nucleic Acids Research: 2001 – 2002.
• Molecular Therapy: 1999 – 2001.

ACADEMIC HONORS

• Harvard Medical School, Global Clinical Scholar Post Graduate: graduation with distinction (top 5% of graduating class).
• “DNA Vaccine” Recognizes Robert W. Malone, MD, MS, 2013.
• Trainee Investigator Award, American Federation for Clinical Research: 1993.
• Bank of America – Giannini Foundation Medical Research Fellow: 1992 – 1993.
• Henry Christian Award for Excellence in Research, American Federation for Clinical Research: 1992.
• UCDMC Medical Scholars Grant: 1992 – 1993.
• DNA and RNA Transfection and Vaccination (Abstract). First Place, Northwestern AOA Research Symposium Competition for Medical Students: 1989.
• USPHS Pre-Doctoral Fellowship: 1986 – 1988.
• San Diego Supercomputer Grant for RNA Structure Modeling: 1988.
• Northwestern University MD/ PhD Scholarship: 1984 – 1986.
• Dean's List, UC Davis: 1982 – 1984.
• President's Undergraduate Fellowship Grant for Investigation of Oncogene Expression in Breast Tumor Tissue: 1983 – 1984.
• Edmonson Summer Fellowship, Department of Pathology, UC Davis Medical School: 1984.

PATENTS ISSUED:

1. Lipid-mediated polynucleotide administration to deliver a biologically active peptide and to induce a cellular immune response. Assigned to Vical, Inc and licensed to Merck. No. 7,250,404, date of issue: 7/31/07 Cited in 105 articles.
2. Lipid-mediated polynucleotide administration to reduce likelihood of subject's becoming infected. Assigned to Vical, Inc and licensed to Merck. US Pat. Ser. No. 6,867,195 B1, date of issue: 3/15/05.
3. Generation of an immune response to a pathogen. Assigned to Vical, Inc and licensed to Merck. US Pat. Ser. No. 6,710,035, date of issue: 3/23/04. Citations: 37 articles.
4. Expression of exogenous polynucleotide sequences in a vertebrate, mammal, fish, bird or human Assigned to Vical, Inc, licensed to Merck. US Pat. Ser. No. 6,673,776, date of issue: 1/6/04.
5. Methods of delivering a physiologically active polypeptide to a mammal. Assigned to Vical, Inc, licensed to Merck. US Pat. Ser. No. 6.413.942, date of issue: 7/2/02. (cited in 150 articles).
6. Induction of a protective immune response in a mammal by injecting a DNA sequence (includes mRNA). Assigned to Vical, Inc, licensed to Merck. US Pat. Ser. No. 6,214,804, date of issue: 4/10/01. Cited in 359 articles.
7. DNA vaccines for eliciting a mucosal immune response (includes mRNA). US Pat. Ser. No. 6,110,898, date of issue: 8/29/00. Cited in 40 articles.
8. Formulations and methods for generating active cytofectin: polynucleotide transfection complexes. US Pat. Ser. No. 5,925,623 7/20/99.
9. Cationic Transport Reagents. US Pat. Ser. No. 5,892,071 issued 4/06/99.
10. Polyfunctional cationic cytofectins, formulations and methods for generating active cytofectin: polynucleotide transfection complexes. US Pat. Ser. No. 5,824,812 issued 10/20/98.
11. Cationic Transport Reagents. US Pat. Ser. No. 5,744,625 issued 4/28/98.
12. Generation of antibodies through lipid mediated DNA delivery. Assigned to Vical, Inc, licensed to Merck. US Pat. Ser. No. 5,703,055, date of issue: 12/30/97. Cited in 463 articles.
13. Induction of a protective immune response in a mammal by injecting a DNA sequence (includes mRNA). Assigned to Vical, Inc, licensed to Merck. US Pat. Ser. No. 5,589,466, date of issue: 12/31/96. Cited in 889 articles.
14. Delivery of exogenous DNA sequences in a mammal (includes mRNA). Assigned to Vical, Inc, licensed to Merck. US Pat. Ser. No. 5,580,859, date of issue: 12/3/96. Cited in 1234 articles.
15. Cationic Transport Reagents. US Pat. Ser. No. 5,527,928, date of issue: 6/18/96. Of note: Cationic Lipid-Mediated RNA and DNA Transfection. 1988 patent application, Salk institute assignee, patent abandoned without inventor permission or knowledge. Inventor: Robert Malone. Available upon request.

PUBLICATIONS (selected)

1. Famotidine and Celecoxib COVID-19 Treatment Without and With Dexamethasone; Retrospective Comparison of Sequential Continuous Cohorts, Submitted to Nature, Scientific Reports, May 2021. Robert W Malone, Kevin M Tomera, Leo Egbujiobi, Joseph K Kittah . Preprint at Research Square https://www.researchsquare.com/article/rs-526394/v1 .
2. More Than Just Heartburn: Does Famotidine Effectively Treat Patients with COVID-19? Malone RW. Dig Dis Sci. 2021 Feb 24:1–2. doi: 10.1007/s10620-021-06875-w. PMID: 33625612; PMCID: PMC7903029.
3. COVID-19: Famotidine, Histamine, Mast Cells, and Mechanisms.  Malone RW, et. al. Frontiers in Pharmacololgy, 23 March 2021. https://doi.org/10.3389/fphar.2021.633680
4. COVID-19: Famotidine, Histamine, Mast Cells, and Mechanisms.  Malone RW, et al DO.Res Sq. 2020 Jun 22:rs.3.rs-30934. doi: 10.21203/rs.3.rs-30934/v2. Preprint.PMID:  32702719 https://www.researchsquare.com/article/rs-30934/v2 Cited in 26 articles.
5. Hospitalized COVID-19 Patients Treated With Celecoxib and High Dose Famotidine Adjuvant Therapy Show Significant Clinical Responses (July 8, 2020). Tomera, K, Malone, R and kittah, J.  Available at SSRN: https://ssrn.com/abstract=3646583 or http://dx.doi.org/10.2139/ssrn.3646583 . Cited in 8 articles.
6. Medical Countermeasures Analysis of 2019-nCoV and Vaccine Risks for Antibody-Dependent
7. Enhancement (ADE). Ricke, D.O.; Malone, R.W. Preprints 2020, 2020030138 (doi: 10.20944/preprints202003.0138.v1). May, 2020 https://papers.ssrn.com/sol3/papers.cfm?abstract_id=3646583
8. Molecular evolution of Zika virus as it crossed the Pacific to the Americas. Schneider AB, Malone RW, et al. Cladistics. 2017; 12: 10.1111/cla.12178
9. Zika Virus: Medical Countermeasure Development Challenges. Malone RW, et al. PLoS Negl Trop Dis.  2016;10(3):e0004530. Cited in 70 articles, viewed over 54,000 times, full PDF downloaded over 11,000 times.
10. Zika Fetal Neuropathogenesis: Etiology of a Viral Syndrome. Klase ZA, Khakhina S, Schneider Ade B, Callahan MV, Glasspool-Malone J, Malone R. PLoS Negl Trop Dis. 2016;10(8):e0004877. Cited in 51 articles, viewed over 13,000 times.
11. Antibody mediated epitope mimicry in the pathogenesis of Zika virus related disease. Homan J, Malone RW, et al. BioRxiv. 2016.
12. Making vaccines "on demand": a potential solution for emerging pathogens and biodefense? De Groot AS, Einck L, Moise L, Chambers M, Ballantyne J, Malone RW Hum Vaccin Immunother. 2013;9(9):1877-84.
13. Electroporation enhances transfection efficiency in murine cutaneous wounds. Byrnes CK, Malone RW, et al. Wound Repair Regen. 2004;12(4):397-403.
14. DNA transfection of macaque and murine respiratory tissue is greatly enhanced by use of a nuclease inhibitor. Glasspool-Malone J, …, Malone RW. J Gene Med. 2002;4(3):323-2.
15. Marked enhancement of macaque respiratory tissue transfection by aurintricarboxylic acid. Glasspool-Malone J, …, Malone RW. Gene Med. 2002;4(3):323-2.
16. Enhancing direct in vivo transfection with nuclease inhibitors and pulsed electrical fields. Glasspool-Malone J, Malone RW. In Gene Therapy Methods: Methods Enzymol. 2002;346:72-91
17. Cutaneous transfection and immune responses to intradermal nucleic acid vaccination are significantly enhanced by in vivo electropermeabilization. Drabick JJ, Glasspool-Malone J, …, Malone RW. Mol Ther. 2001;3(2):249-55. Cited in 192 articles.
18. Theory and in vivo application of electroporative gene delivery. Somiari S, Glasspool-Malone J, … Malone RW. Mol Ther. 2000;2(3):178-87. Cited in 345 articles.
19. Nucleic acid vaccination with a single SIV can protect rhesus macaques from oral challenge with pathogenic SIVMAC239. Gary Rhodes, … Robert Malone, et al. Journal of Medical Primatology 29.3-4 (2000).
20. Efficient nonviral cutaneous transfection. Glasspool-Malone J, …, Malone RW. Mol Ther. 2000;2(2):140-6. Cited in 138 articles.
21. Transfer and expression of foreign genes in mammalian cells. Colosimo A, …, Malone RW, et al. Biotechniques. 2000;29(2):314-8, 20-2, 24 passim. Cited in 188 articles
22. Specific inhibition of macrophage TNF-alpha expression by in vivo ribozyme treatment. Kisich KO, Malone RW, …, Erickson KL. J Immunol. 1999;163(4):2008-16. Cited in 131 Articles.
23. Marked enhancement of direct respiratory tissue transfection by aurintricarboxylic acid. Glasspool-Malone J, Malone RW. Hum Gene Ther. 1999;10(10):1703-13
24. Developing dendritic cell polynucleotide vaccination for prostate cancer immunotherapy. Berlyn KA, …, Malone RW J Biotechnol. 1999;73(2-3):155-79
25. Models of Cationic Liposome Mediated Transfection. Gene Therapy and Molecular Biology. Ahearn A, Malone RW. Vol 4. Gene Therapy and Molecular Biology 1999;4
26. Feline dendritic-like cells: Isolation, culture, and genetic modification using monocytic precursors. Malone, J. G., Watts, T. L., Hale, A., & Malone, R. W. (1998, January). In JOURNAL OF LEUKOCYTE BIOLOGY (pp. 63-63): FEDERATION AMER SOC EXP BIOL.
27. Mucosal immune responses associated with polynucleotide vaccination. Malone JG, …, Malone RW. Behring Inst Mitt. 1997(98):63-72
28. Delivery of exogenous DNA sequences in a mammal. [Dr. Philip Louis Felgner (born 1950)], …, R Malone, [Dr. Dennis A. Carson (born 1936)]. Biotechnology Advances. 1997 15 (3-4), 763-763
29. Cationic lipid-mediated gene delivery to murine lung: correlation of lipid hydration with in vivo transfection activity. Bennett MJ, …, Malone RW, Nantz MH. J Med Chem. 1997;40(25):4069-78
30. Improved method for the removal of endotoxin from DNA. Montbriand PM, Malone RW. J Biotechnol.  1996;44(1-3):43-6. Cited in: 43 articles
31. Toxicity of cationic lipid-ribozyme complexes in human prostate tumor cells can mimic ribozyme activity.Freedland SJ, Malone RW, et al. Biochem Mol Med. 1996;59(2):144-53
32. Considerations for the design of improved cationic amphiphile-based transfection reagents. Bennett MJ, …, Malone RW. Journal of Liposome Research 1996;6(3):545-65
33. Escherichia coli beta-glucuronidase and Photinus pyralis luciferase reporter. Ayar, S. F., & Malone, R. W.  (1996, November). In CLINICAL CHEMISTRY (Vol. 42, No. 11, pp. 35-35).
34. Structural and functional analysis of cationic transfection lipids: the hydrophobic domain.  Balasubramaniam RP, …, Malone RW. Gene Ther. 1996;3(2):163-72. Cited in 172 articles.
35. The counterion influence on cationic lipid-mediated transfection of plasmid DNA.Aberle AM, Bennett MJ, Malone RW, Nantz MH. Biochim Biophys Acta. 1996;1299(3):281-3
36. Direct gene tranfer into mouse muscle in vivo. N Shafee, ..., RW Malone, et al. International Journal of Virology 2 (1), 33-38
37. A flexible approach to synthetic lipid ammonium salts for polynucleotide transfection. MJ Bennett, RW Malone, MH Nantz. Tetrahedron letters 36 (13), 2207-2210
38. Tfx-50 Reagent, a new transfection reagent for eukaryotic cells. Schenborn E, …, Malone RW, et al. 1995
39. Hepatic gene expression after direct DNA injection. Hickman MA, Malone RW, et al. Advanced Drug Delivery Reviews. 1995;17(3):265-71
40. Ribozyme and messenger-RNA delivery using cationic liposomes RW MALONE 1995/1/5 Conference  JOURNAL OF CELLULAR BIOCHEMISTRY Pages 206 Publisher WILEY-LISS
41. Cholesterol enhances cationic liposome-mediated DNA transfection of human respiratory epithelial cells.  Bennett MJ, …, Malone RW. Biosci Rep. 1995;15(1):47-53
42. Dexamethasone enhancement of gene expression after direct hepatic DNA injection. Malone RW, et al. J Biol Chem. 1994;269(47):29903-7
43. Gene expression following direct injection of DNA into liver. Hickman MA, Malone RW, et al. Hum Gene Ther. 1994;5(12):1477-83. Cited in 306 articles.
44. Cationic liposome-mediated RNA transfection. Dwarki VJ, Malone RW, Verma IM. Methods Enzymol.  1993;217:644-54. Cited in 88 articles.
45. Successful gene transfection of respiratory epithelium invitro using polyamine containing cationic lipids. CB Robinson, RW Malone, J Jessee, G Gebeyehu, R Wu AMERICAN REVIEW OF RESPIRATORY DISEASE 147 (4), A546-A546
46. Direct gene transfer into mouse muscle in vivo. [Dr. Jon Asher Wolff (born 1956)], Malone RW, et al. Science. 1990;247(4949 Pt 1):1465-8. Cited in 4,695 articles.
47. Cationic liposome-mediated RNA transfection. Malone RW,  [Dr. Philip Louis Felgner (born 1950)],  Verma IM. Proc Natl Acad Sci U S A. 1989;86(16):6077-81. Cited in 717 articles.
48. mRNA Transfection of cultured eukaryotic cells and embryos using cationic liposomes. Malone RW.  Focus. 1989;11:61-8
49. High levels of messenger RNA expression following cationic liposome mediated transfection tissue culture cells. Malone R, Kumar R, [Dr. Philip Louis Felgner (born 1950)],  NIH Conference: “Self-Cleaving RNA as an Anti-HIV Agent” (Abstract). Washington, DC June 1989.
50. A novel approach to study packaging of retroviral RNA by RNA transfection (Abstract). RW Malone, [Dr. Philip Louis Felgner (born 1950)], I. Verma. RNA Tumor Viruses, May 17-18, 1988. Cold Spring Harbor
51. Mammary tumors in feral mice lacking MuMTV DNA. [Dr. Murray Briggs Gardner (born 1945)], Malone RW, …, Cardiff RD, et al. J Exp Pathol. 1985;2(2):93-8
52. Hyperplastic and neoplastic changes in the mammary glands of feral mice free of endogenous mouse mammary tumor virus provirus. Faulkin LJ, …, Malone RW, et al. J Natl Cancer Inst. 1984;73(4):971-82.

PUBLISHED ABSTRACTS: Over 50 published

CHAIRPERSON/ORAL PRESENTATIONS BY INVITATION: Over 40 Invitations

• Vaccines R&D, 2019. Keynote Speaker, Panel Moderator: Boston, MA. 18-20 November, 2019.
• Repurposing drugs for Infectious Disease Outbreaks. International Conference on Zika Virus. Washington, DC Feb 22-25, 2017 (Chairperson)
• Accelerated Discovery and Development of re-purposed licensed drugs for Zika virus outbreak antiviral prophylaxis and therapy. International Conference on Zika Virus. Washington, DC Feb 22-25, 2017. (Oral Presentation)
• Zika Virus: Accelerating Development of Medical Countermeasures by Re-purposing Licensed Drugs. Bridging the Sciences: Zika Virus. Emery, Atlanta, GA 1-3 May, 2016. (Oral Presentation)
• Speaker/Round table- Zika virus: Challenges for Medical Countermeasure Development. World Vaccine Conference. Washington, DC. 29-31 March, 2016.
• The World Health Organization (WHO) Consultation for Zika Virus: Research and Development. Presentation of Drug Development TPP. Geneva, Switzerland. 12-14 March, 2016. (Oral Presentation)
• Keynote Speaker: Ebola Vaccine in 12 months, Global Village, and the Need for Speed. Vaccines R&D, Baltimore, MD. 2-4 November, 2015. (Keynote Speaker)
• Current USG contracting Opportunities and Initiatives from the point of View of Vaccine Developers. World Vaccine Conference, Washington, DC. 24-26 March, 2014. (Oral Presentation)
• World Vaccine Conference, Washington, DC. 24-26 March, 2014 Preclinical and Clinical Vaccine Research. (Session Chair)
• PHEMCE Modeling Workshop “Operational Decision Making using Innovative Modeling, Analysis, and Visualization Tools”, Sponsored by Deloitte. 2013 (Conference Co-Organizer and Coordinator/Oral Presentation)
• "Vaccine Production Strategies: Ensuring Alignment and Sustainability" The World Health Organization (WHO) Global Action Plan for Influenza Vaccines. Geneva, Switzerland. 12-14 July 2011 (Oral Presentation)

RECENT STUDY SECTIONS (selected):

• Accelerated COVID-19 Therapeutic Interventions and Vaccines: ACTIV Therapeutics Clinical Working Group, NIH. Invited Participant. June, 2020-present.
• Chairperson, NIH/NIAID/DMID Special Emphasis Panel, Development of Vaccines to Combat Antibiotic Resistant Bacteria September 2019.
• Chairperson, NIH/NIAID Special Emphasis Panel, December 2018.
• Reviewer, NIH/NIAID Special Emphasis Panel, December 2017.
• Chairperson and scientific reviewer for Department of Defense, U.S. Army Medical Research and Materiel Command, for “Congressionally Directed Medical Research Programs (DMRDP), 2012.
• Committee member and reviewer for NIH/NIAID Committee for Development of Technologies that Accelerate the Immune Response to BioDefense Vaccines. 2011
• Chair and reviewer for NIH/NIAID: Partnerships in Biodefense Immunotherapeutics. 2011
• NIH/NIAID Committee member and reviewer for Development of Technologies to Facilitate the Use of, and Response to Biodefense Vaccines,” Special Emphasis panel. 2010
• Chairperson and scientific reviewer for NIH/NIAID Omnibus BAA 2017-1: Research Area 5 (N01) ZAI1-KP- M-C6 (Topic 5: Advanced Development of Vaccine Candidates for Biodefense and Emerging Infectious Diseases), September 2017.
• Scientific reviewer for NIH/NIAID Special Emphasis Panel/Scientific Review Group 2017/08 ZRG1 IMM-R (12) B (Non-HIV Microbial vaccines), June 2017.
• Chairperson and scientific reviewer for Department of Defense, U.S. Army Medical Research and Materiel Command, “CDMRP: Defense Medical Research & Development Program (DMRDP), 2012.
• Chairperson and scientific reviewer for NIH/NIAID Committee on Partnerships in Biodefense Immunotherapeutics, Fall 2011.
• Committee member and reviewer for NIH/ NIAID Committee for Development of Technologies that Accelerate the Immune Response to BioDefense Vaccines, Fall 2011.
• NIH/ NIAID Committee member and reviewer for Development of Technologies to Facilitate the Use of, and Response to Biodefense Vaccines,” Special Emphasis panel, 2010.
• NIH Study Section K01 Breast Cancer Study Section: July 1997
• NIDDK Special Emphasis Panel Review Committee for Competing Continuation Program Project:  April 1999 and April 1998
• NIAID Study Section “Innovative Grant Program for Approaches in HIV Vaccine Research”: 1998

BOOKS AND BOOK CHAPTERS

• Malone RW. "Present and Future Status of Gene Therapy.' Intro Chapter in Advanced Gene Delivery: From Concepts to Pharmaceutical Products.” Editor: Allain Rolland. Harwood Academic Pub. 1998, republished 2014.
• Enhancing direct in vivo transfection with nuclease inhibitors and pulsed electrical fields. Glasspool-Malone J, Malone RW. In Gene Therapy Methods: Methods Enzymol. 2002;346:72-91
• Malone RW. “Toxicology of non-viral gene transfer”. Editor, Walsh B. In: “Non-Viral Therapeutics: Advances, Challenges and Applications for Self-Assembling Systems.” IBC’s Biomedical Library Series. (1996) 4.1

The evolutionary conservation of genetically transmitted retrovirogenes has suggested that they may provide a critical function for normal development ( 1 ) . In the MuMTV system , specifically, it has been speculated that MuMTV expression may be required for normal functioning of the mammary gland. Bent velzen and Hilgers ( 2 ) hypothesized that MuMTV germinal provirus was associated with a mam gene, analogous with src of the avian sarcoma virus. They proposed that controlled expression of this gene was essential for normal mammary gland development, whereas uncontrolled expression ( as after exogenous infection with MuMTV ) resulted in mammary neoplasia ( 2).

COnversely, the great variation in endrogenous MuMTV restruction patterns in inbred mouse strains and individual wils mice implied a lack of selective advantage of these genes (3). This belief was supported by the finding of several apparently normal feral mice lacking detectabl eMuMTX provirus in their liver DNA (2). COhen and Varmus *3) thus favored the hypothesis that endogeneous proviruses were acquired by multiple independent infections of germ cells, and they saw no funcitonal ruole for proviral DNA in normal growth and deveopment. 

To determine whether enogeneous MuMTX provirueses are essential to normal development of the mammary gland and/or the development of mammary hyperplasia and neoplasia, we have studies these featres in a coloony of selectively bred feral mice that are totaly free of endogenous MuMTV copies in their cellular genome ( 5 ) . For ease of discussion we have labeled these endogenous virus -negative mice " ev ." In the ev mice , mammary gland development and function are compared to other feral mice homozygous for a single MuMTV provirus labeled " ev ” and to well established strains of inbred mice carrying multiple MUMTV proviruses ( 5 ) . In addition , we describe the formation in the mammary tissue of the ev and ev * mice of hyperplastic and neoplastic lesions that, although infrequently seen , are similar to those of laboratory mice ( 6 , 7 ) . We confirmed by Southern blot analysis that the ev' and ev * mouse colonies bred true for this trait and that the tumor DNA in these mice showed no alteration from the germ line MuMTV genotype. 

[...]

The wide variety of methods to introduce genetic material into cells includes relatively simple manipulations like mixing high molecular weight DNA with calcium phosphate, DEAEdextran, polylysine, or polyornithine. Other methods involve electroporation, protoplast fusion, liposomes, reconstituted viral envelopes, viral vectors, or microinjection. In nearly all cases DNA has been introduced into cells because of its inherent stability and eventual integration in the host genome. By comparison, progress in introducing RNA molecules into cells has been very slow and restricted to a few cases (1-4). Inability to obtain sufficient amounts of intact RNA and its rapid degradation have been a major hindrance in the past. The limitation of obtaining sufficient quantities of RNA can now be alleviated by synthesizing large amounts of RNA in vitro, using bacteriophage RNA polymerases (5).

Since we were interested in studying the cis- and transacting factors influencing both the translational efficiency and the stability of eukaryotic mRNAs, we undertook the development of a reliable method to efficiently introduce RNAs into cells. We report the use of RNA transfection mediated by lipofectin (a liposome containing a cationic lipid) for efficient and reproducible RNA introduction and expression in tissue culture cells. The RNA/lipofectin complex can be used to introduce RNA into a wide variety of cells, including fibroblasts, hematopoietic cell lines, F9 teratocarcinoma cells, JEG choriocarcinoma cells, PC12 pheochromocytoma cells, amphibian cells, insect cells, and a variety of cells grown in suspension.

MATERIALS AND METHODS

Tissue Culture and Plasmids. All the cell lines used were obtained from the American Type Culture Collection and grown in either Dulbecco's modified Eagle's medium (DMEM) + 10% fetal calf serum or RPMI 1640 medium + 10%o fetal calf serum. Drosophila KC cells were obtained from Michael McKeown (Salk Institute) and maintained in D22 medium (Whittaker M. A. Bioproducts).

Cloning procedures were carried out essentially as described (6). T7 RNA polymerase transcription templates, as well as various mRNAs produced from them, are outlined in

Fig. 1. Xenopus laevis f3-globin sequences were derived from the plasmid pSP64 T (7), with the 5' f-globin sequences obtained as the HindIII/Bgl II fragment and the 3' /3-globin sequences released as the Bgl II/EcoRI fragment. These 3' sequences include a terminal polynucleotide tract of A23C30. The Photinus pyralis luciferase sequences were obtained as the HindIII/BamHI fragment ofpJD206 (8), and they include 22 bases of luciferase cDNA sequence preceding the open reading frame, as well as 45 bases of cDNA sequence downstream of the termination codon, but they are devoid of the luciferase polyadenylylation signal. The 30-nucleotide poly(A) tail of the plasmid Luc An was obtained from pSP64 An. All transcripts were generated from the T7 RNA polymerase promoter (9).

RNA Synthesis and Purification. The capped RNAs were transcribed from a linearized plasmid DNA in a reaction mixture containing 40 mM Tris HCl at pH 8.0, 8 mM MgCl2, 5 mM dithiothreitol, 4 mM spermidine, 1 mM ATP, 1 mM UTP, 1 mM CTP, 0.5 mM GTP, 0.5 mM m7G(5')ppp(5')G (New England Biolabs), T7 RNA polymerase (New England Biolabs) at 4000 units/ml, RNasin (Pharmacia) at 2000 units/ ml, and linearized DNA template at 0.5 mg/ml for 60 min at 370C. Transcription reaction mixtures were treated with RQ1 DNase (2 units/,ug of template; Pharmacia) for 15 min at 370C, and, after extraction with phenol/chloroform, the samples were precipitated with ethanol/NaOAc. Uncapped RNAs were prepared in a similar fashion, except that m7G(5')ppp(5')G was omitted and the GTP concentration was raised to 1 mM. Radioactive RNA was prepared without capping as described above by adding 4 ,uCi (1 Ci = 37 GBq) of [32P]UTP per ag of template DNA. All RNA species used for the data presented herein were prepared in bulk, using reactions yielding 0.1-1 mg of purified RNA.  [...]

The experiment was so elementary, and the results so surprising, that researchers working with San Diego’s Vical Inc. couldn’t really believe what they were seeing. It all seemed too simple.

They had been injecting submicroscopic fatty globules containing DNA or RNA into mice to see what would happen. The idea was that the fat globules, called liposomes, would be taken up by cells. The cells would use the genetic material inside to make proteins they couldn’t otherwise make.

The researchers found moderate success with that, but the rigors of science demanded that the experiment have a “control” portion--injecting the raw DNA or RNA into the mice to show that the liposomes themselves were making it possible for the new genes to be incorporated into the cell’s processes.

It turned out the cells like the raw material even better and began making the new proteins for as long as six months.

“This was a big surprise, and that’s really what you’re looking for in this area,” said [Dr. Philip Louis Felgner (born 1950)], director of product development at Vical. Felgner worked on the experiment with [Dr. Jon Asher Wolff (born 1956)] and others at the University of Wisconsin at Madison.

Researchers spent several months longer trying to find flaws in their methods or their conclusions. The literature of science is littered with examples of experimental results that deserved the label of too good to be true, explained [Dr. Karl Yoder Hostetler (born 1939)], vice president for research and development at Vical.

“We didn’t want any fiascoes,” he said.

Vical hopes that the results of this checking and double-checking, reported in today’s issue of the journal Science, will convert the company from a bare-bones start-up to a major player in the ranks of San Diego’s biotechnology community.

The company, which was founded in 1987, hopes to find financing to more than double its scientific staff of 22 as a result of the study. It is talking with several large drug companies to see if any would like to buy into the follow-up studies on the new gene transfer method, said Vical President Wick Goodspeed.

Some familiar names in San Diego science and business have played a role in Vical. Among them:

[Dr. Karl Yoder Hostetler (born 1939)], who is on leave from his longtime post as professor of medicine in residence at UC San Diego. His specialties include investigating ways to use lipid chemistry to improve the effectiveness of drugs.

[Dr. Douglas Daniel Richman (born 1943)], a founder and scientific adviser to the firm. Richman is a professor in residence of medicine and pathology at UCSD, specializing in virology and clinical trials of AIDS treatments.

[Dr. Dennis A. Carson (born 1936) ], also a scientific adviser to the firm. Carson recently resigned as head of the division of clinical immunology at Scripps Clinic to become head of UCSD’s new institute for research on aging.

Timothy Wollaeger, chairman of the board. Wollaeger formerly was senior vice president in charge of finance and administration for Hybritech Inc., the monoclonal antibody firm whose success was capped in 1986 with its $485-million acquisition by Eli Lilly & Co.

Howard E. (Ted) Greene, a director of Vical. He formerly was chief executive officer for Hybritech. Greene and Wollaeger were the driving forces behind Biovest Partners, a venture capital firm that financed several San Diego biotech firms.

W. Larry Respess, a Vical director. A leader in biotech patent law, he formerly was general counsel of Gen-Probe and Hybritech.

Until now, the best combination of science and business for Vical has been the multi-year research contract it received last summer from Burroughs Wellcome Co. to develop new forms of AZT for AIDS therapy. The study is investigating the idea that encasing AZT in fat globules would make it more powerful within the body.

The gene-insertion technique reported in Science this week is being suggested as a way to cause the body to generate proteins that would block persistent viral infections, ranging from AIDS to herpes. It also is seen as having potential use as a way to trigger cells to immunize the body against diseases, researchers say.

Vical is calling the new method “gene therapeutics,” to distinguish it from the traditional goal of gene therapy, which uses viruses to insert missing genes into the genetic codes of people with genetic diseases.

The so-called retroviral method has proved difficult and slow, despite several years of intense effort by research groups around the country, including a group led by Dr. Theodore Friedmann at UCSD.

Because retroviruses insert their own genetic code into the cells of their host, the method is also expected to be problematic as a gene therapy technique--since some scientists worry that this could harm the patient irreversibly in some unforeseen way.

Inserting the genes themselves into muscle cells--without any retroviral carrier--avoids this stumbling block entirely, [Dr. Philip Louis Felgner (born 1950)] said. The genes do their work of producing proteins, called expression, but they don’t seem to affect the cell’s own genetic structure, he said.

“People have worked in the gene therapy area for years assuming that a rather complex viral delivery system would be required in order to get expression. And we have found that you can do it very simply,” Felgner said.

It was the slowness of the gene therapy field that led Felgner’s collaborator,  of the University of Wisconsin, to decide less than two years ago to get out of it altogether, Wolff said in a telephone interview.

Wolff was an assistant professor and a researcher in Friedmann’s UCSD lab before going to Wisconsin as an assistant professor of pediatrics and medical genetics in 1988.

“I had pretty much planned to get out of the gene therapy field because I got discouraged with the retroviral approach. Scientifically, it wasn’t very challenging,” he said. “Everybody was doing the same thing, and nothing was working that well.”

The results of the research contract with Vical, begun in January, 1989, have rekindled his enthusiasm, [Dr. Jon Asher Wolff (born 1956)] said.

He believes that, in the end, genetic therapies will involve a variety of techniques, not just the Vical method. But he and [Dr. Philip Louis Felgner (born 1950)] acknowledge that they expect some resistance to their ideas from the traditional gene therapy community.

“You’re talking about somebody who has spent his life in this field, and who would like to make the real breakthroughs that are going to allow it to be used in patients with diseases,” Felgner said. “There’s quite a bit at stake.”

Other collaborators with Wolff and Felgner on the research were [Dr. Robert Wallace Malone (born 1959)] of Vical and Phillip Williams, Wang Chong, Gyula Acsadi and Agnes Jani in Wisconsin.

Vical is planning to try to patent the technique, even though it involves no novel or complex steps unfamiliar to molecular biologists. In essence, it involves preparing DNA or RNA with standard techniques and then injecting it in the conventional way into muscle.

“The reason why we have patent position is that it was such a total surprise. Some of those things are the best patents you can get,” Felgner said. “Nobody who was ‘skilled in the art’ would have ever thought that what we have accomplished here was even possible. Nobody would have even thought to do the experiment.”

Abstract : A variety of gene delivery technologies can be used to express antigens within somatic tissues, resulting in systemic humoral and cellular immune responses. This observation has led to the development of polynucleotide vaccine preparations for stimulation of systemic immunity. Mucosal immune responses are functionally distinct from systemic immune responses, and are stimulated by antigen presentation within specialised mucosal-associated inductor tissues. We hypothesize that mucosal genetic vaccine will require gene transfer methods which target mucosal-associated inductor tissues such as the oropharyngeal Waldeyer's ring or intestinal Peyer's patches. We have tested this hypothesis by expressing a test antigen using a replication-defective recombinant Semliki Forest Virus (SFV) preparation. Mice treated with recombinant SFV via an intravascular or intratracheal route generated systemic immune responses against the test antigen. In contrast, intranasal inoculation resulted in the production of IgA within pulmonary fluids, one hallmark of a mucosal immune response. These results indicate that transfection of mucosal effector tissues may not be sufficient for the generation of a universal mucosal immune response. Furthermore, the results predict that techniques which target transfection or transduction to mucosal inductor tissues will enable the development of a new class of polynucleotide vaccines which exploit current concepts in mucosal immunology.

• License Number: D55466 Dr. Robert Wallace Malone

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• Licensed Issued: 10/27/1999  License Expiration: 09/30/2023 

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•  Education :   NORTHWESTERN UNIV MED SCH  /   Graduation Year: 1991

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      • • ► University of California (Davis), Sacramento, CA

        • ► University of Maryland Medical School, Baltimore, MD

        • ► University of California (Davis), Davis, CA

Gene therapy burst into the news last fall, when a teenager from Tucson died in an experiment at the University of Pennsylvania. But while politicians and the press have spent the year focusing on accusations of lax government oversight and shoddily run clinical trials, the science of gene therapy has been making quiet, steady progress.

Little by little, researchers say, they are finding new and better ways to deliver genes to their target cells. Clinical trials of gene-based treatments for hemophilia are showing promise, and certain cancer patients appear to respond to gene therapy. And the field got a big ego boost this spring when French scientists reported they had used gene therapy successfully to treat babies born with defective immune systems.

And so it was with mixed emotions -- worry and regret, enthusiasm and a little bit of defiance -- that 2,500 gene therapy scientists from around the nation, and the world, gathered here this week for the third annual conference of the American Society of Gene Therapy. It has been, they agreed, a roller coaster of a year.

''It's finally coming together,'' said Dr. Savio L. C. Woo, a soft-spoken molecular biologist who, as the society's president, found himself testifying in Congress on more than one occasion in the past several months. ''We are finally seeing this glimpse of hope that this new technology is going to bear fruit in the clinic. And yet we have had this serious setback.''

The setback, of course, was the death of 18-year-old [Jesse Gelsinger (born 1981)], who suffered from a metabolic disorder and had volunteered for an experiment to test gene therapy for babies with a fatal form of the disease. His presence was acutely felt at the conference. In his presidential address, Dr. Woo asked those attending to stand in a moment of silence for ''the young man who has given his life in pursuit of an ideal treatment'' and ''to assure him in spirit that the scientific community is galvanized to do our very best to help fulfill his dream one day.''

Mr. Gelsinger's death has had ripple effects throughout the field. It touched off a discussion of financial conflict of interest in gene therapy experiments, prompting the society to issue guidelines barring members from running clinical trials if they had a financial stake in the companies sponsoring their studies.

It has also caused a slowdown in human experiments; an official from the Food and Drug Administration, which recently issued more stringent regulations to govern the conduct of gene therapy clinical trials, said here that requests to test gene therapy in people had dropped off sharply in recent months.

''The field is in transition,'' said [Dr. Robert Wallace Malone (born 1959)], a researcher at the University of Maryland. ''I think it is transitioning to a more sober, realistic recognition of what is achievable. I believe there is a new humility in the field.''

Dr. Rajendra Kumar-Singh is typical. At 32, Dr. Kumar-Singh is an assistant professor of ophthalmology at the University of Utah and is just starting his career in gene therapy. He is studying treatments for retinitis pigmentosa, an inherited condition that causes blindness. By administering an infusion of gene-altered viruses to baby mice, he said, he has staved off blindness for 10 weeks in animals that would otherwise have lost their sight within 17 days of birth.

But Dr. Kumar-Singh said he would be cautious before testing the therapy in people. ''Perhaps we were moving too fast,'' he acknowledged, echoing the sentiments of some critics, including [Dr. Harold Eliot Varmus (born 1939)], the former director of the National Institutes of Health, who felt gene therapy researchers moved too quickly into clinical trials. Still, Dr. Kumar-Singh is optimistic about gene therapy's future. ''We are seeing a revolution in medicine right now,'' he said, ''and gene therapy is at the forefront of it.''

The idea behind gene therapy is disarmingly simple: to treat or cure disease by giving healthy genes to patients with defective ones. But in the 10 years since the first human experiment was conducted by researchers at the National Institutes of Health in Bethesda, Md., the results have been largely disappointing.

One reason is that scientists have had trouble devising delivery vehicles, called vectors, that can direct genes into the proper cells and get them to function once they are there. Vectors are typically made by inserting genes into deactivated viruses that target certain cells, literally infecting them with healthy DNA.

Mr. Gelsinger's death, however, raised safety questions about one of the most commonly used viruses, adenovirus, which causes the common cold. In most patients, adenovirus produces mild, flulike symptoms. But in Mr. Gelsinger, it provoked a fatal immune response.

Even before Mr. Gelsinger's death, molecular biologists had been turning their attention to a different virus, the adeno-associated virus, or AAV, which is thought to be safer than adenovirus. Now, a team of researchers at Children's Hospital of Philadelphia, Stanford University and Avigen Inc., a biotech company, is reporting promising results in hemophilia patients who received a genetically engineered form of AAV that contains the gene for production of Factor IX, a protein that is needed to make blood clot.

The team, led by Dr. Katherine High of the Philadelphia children's hospital, began a small safety study after they found the treatment could essentially cure dogs of hemophilia. So far, six patients have been enrolled. The first three received the gene therapy at a dose so low it was not effective in the dogs. But to the scientists' surprise, the patients began expressing minute amounts of Factor IX -- enough that it improved their conditions and reduced their need for the standard hemophilia treatment, injections of Factor IX.

''We were delighted, but skeptical,'' said Dr. Catherine S. Manno, who is running the clinical trial. ''Only after repeated measurements over a period of months did we become convinced that these levels were real.''

Although the hemophilia experiments are still in their early stages, leaders in gene therapy say that, aside from the French work, Dr. High's research is the most exciting in the field. Dr. Donald B. Kohn, an immunologist at Children's Hospital of Los Angeles, said, ''Hemophilia may be within a shot of being cured by this approach.''

The word cure -- the ''C-word,'' as [Dr. Robert Michael Blaese (born 1939)], who performed the first human gene therapy experiment in 1990, calls it -- is one that gene therapy researchers have learned to use with caution.

''We will not talk about cure,'' said Dr. Alain Fischer of the Necker children's hospital in Paris. ''Cure means forever.'' Yet Dr. Fischer's work on babies with a form of severe combined immune deficiency, or SCID (pronounced like skid), is the closest thing gene therapy has seen to a cure. His study provided gene therapy advocates with what they have long lacked: proof, in principle, that the concept can work.

Dr. Fischer's findings, published in April in the journal Science, were a popular topic among the scientists in in Denver. ''The field is now an established principle in medicine,'' said Dr. Theodore Friedmann, a professor of pediatrics at the University of California at San Diego who said he had been pursuing gene therapy since 1968. ''That's the story -- a brand new concept in biomedicine, irretrievable, and it's beginning to work.''

In the Science article, Dr. Fischer recounted the successful treatment of two babies, both of whom had normal immune systems 10 months after receiving gene therapy. In Denver, he told reporters that he had since treated three more children. Of the five, four have had ''a complete or near complete recovery'' of their immune systems, he said. The outlook for the fifth, who had severe complications from infection at the time of treatment, is less certain.

Experts say one reason Dr. Fischer was successful where others had failed is that SCID is particularly suited for gene therapy. The first human gene therapy experiment, conducted by Dr. Blaese and [Dr. William French Anderson (born 1936)], was directed at curing adenosine deaminase, or ADA, deficiency, another form of SCID. But it has been difficult to gauge that study's outcome because a drug, PEG-ADA, is available to children with the disease, and scientists consider it unethical to withdraw the medicine.

In Denver, however, an Italian researcher, Dr. Claudio Bordignon, announced that he had solved that problem by being ''lucky to find a patient who was unlucky'' -- a child who does not respond well to PEG-ADA. The patient, now 5, was given her first infusion of corrective genes in 1996 and was slowly weaned from the drug. She has not taken PEG-ADA for one year now, and her immune system is functioning better now than before, Dr. Bordignon said.

He theorized that the drug might have somehow suppressed the effects of the gene therapy. ''After discontinuation of PEG-ADA,'' he said, ''all the genetically engineered cells have come out.''

Whether, or when, gene therapy will ever become a part of mainstream medicine remains a matter of debate. Most gene therapy experiments are still being conducted in animals, and those being tested in people are producing mixed results.

At the Denver conference, for instance, scientists from [Vical Incorporated], a San Diego company, reported preliminary results from 52 patients who have been enrolled in a 70-patient study of gene therapy for advanced skin cancer. In the study, a gene that alerts the immune system to recognize and kill foreign tissue is administered directly into the patients' tumors.

According to Dr. Deirdre Y. Gillespie, Vical's chief operating officer, 10 percent of patients responded extremely well to the therapy, with their tumors shrinking in size by 50 percent or more. In another 15 percent of patients, the therapy stopped the progression of disease, and the therapy reduced the size of tumors in some of those patients as well.

Those may not sound like spectacular results, but as Dr. Gillespie noted, there is currently no effective treatment for advanced skin cancer, and the patients in the study had failed all other therapies. In a sense then, the cancer study encapsulates the state of gene therapy as a whole.

Dr. Blaese put it this way: ''For the average person, the progress may look to be minor. But you need to put the developments of this field in context. We just started 10 years ago.''