Summit Schedule

1:40 PM – 1:50 PM  DISCUSSION

SESSION I: WHAT CAN ALS LEARN FROM SMA?
Chair: John Day, MD, PhD, Professor of Neurology and Neurological Sciences, Stanford University

1:50 PM – 2:10 PM Monitoring Unmet Needs in SMA After Genetic Corrections

John Day, MD, PhD

2:10 PM - 2:30 PM Generating hindbrain motor neurons from pluripotent stem cells

Kyle Loh, PhD, Assistant Professor of Developmental Biology, Stanford University

2:30 PM - 2:50 PM Glial Factors in Persistent SMA Deficits

Michelle Monje, MD, PhD, Milan Gambir Professor of Pediatric Neuro-Oncology, Stanford University

2:50 PM – 3:00 PM  DISCUSSION

3:15 PM – 3:30 PM Modality.AI -- Multimodal Speech Biomarkers for Remote Monitoring of ALS Disease Progression in Clinical Trials
Dr. David Suendermann-Oeft, Chief Executive Officer & Dr. Vikram Ramanarayanan, Chief Scientific Officer
Dr. Vikram Ramanarayanan, Chief Scientific Officer

3:30 PM – 3:45 PM Macrophage-Targeted Innate Immune System Regulation with NP001 (NaClO2)
Michael McGrath, MD, PhD
Professor Emeritus, Department of Medicine, UCSF 

3:45 PM – 4:00 PM Development of LTX-002, an ASO for the treatment of ALS

Asa Abeliovich, CEO, Leal Therapeutics

4:20 PM – 4:40 PM Discovery research at CIRM
Chan Lek Tan, PhD, Senior Science Officer, CIRM

4:40 PM – 5:00 PM Developing a universal CRISPR genome editing therapy for ALS caused by C9orf72 mutations
Claire Clelland, MD, PhD, University of California, San Francisco 

5:00 PM – 5:20 PM C9orf72 Repeat Expansion-Tuned Allelic Suppression by CRISPRi as an ALS Therapy
Ritchie Ho, PhD, Cedars-Sinai

5:20 PM – 5:30 PM DISCUSSION

5:30 PM – 5:45 PM SPECIAL TOPIC
Other Collaborations and ALS How They Intersect with ALS Research
Marg Sutherland, PhD, Neuroscience Program Manager Chan Zuckerberg Initiative

8:30 AM – 8:50 AM Time to SPARCL(1) – The role of astrocytes in synaptic dysfunction
Rita Sattler, MSc, PhD, Barrow Neurological Institute

8:50 AM – 9:10 AM Annexin A11 Proteinopathy in ALS-FTD Spectrum Diseases

Edward B. Lee, MD, PhD, University of Pennsylvania

9:10 AM – 9:30 AM Integrating isogenic iPSCs and proteomics to identify TBK1 networks in ALS/FTD
Daniel Mordes, MD, PhD, Weill Neurohub Assistant Professor, Institute for Neurodegenerative Diseases, UCSF

9:30 AM – 9:40 AM  DISCUSSION

9:55 AM – 10:15 AM Alternative Polyadenylation in ALS or Single Cell Transcriptomics of Motor Neuron Degeneration in ALS
Yi Zeng PhD, Postdoctoral Fellow, and Olivia Gautier, Graduate Student, Gitler Lab, Stanford University 

10:15 AM – 10:35 AM New Mechanisms of TDP-43 Nuclear Export
Sitao Zhang, PhD, Postdoctoral Fellow, Cleveland Lab, University of California, San Diego 

10:35 AM – 10:55 AM TDP- 43 Phase Separation
Jim Shorter, PhD, University of Pennsylvania

11:05 AM – 11:25 AM Helping ALS Patients Speak Again with AI and Brain-Machine Interface Systems
Nicholas Card, PhD, Postdoctoral Fellow, Department of Neurological Surgery, University of California, Davis

11:25 AM – 11:45 AM Circulating cell-free DNA as a new biomarker for ALS
Albert La Spada, MD, PhD, FACMGG, University of California Irvine School of Medicine

11:45 AM – 11:55 AM DISCUSSION

1:15 PM – 1:30 PM Why have Phase III Trials Struggled to Replicate Phase II Trials?
Jeremy Shefner, MD, PhD, Barrow Neurologic Institute

1:30 PM – 1:45 PM What is Working and What Isn't with Current Clinical Trial Outcome Measures

Angela Genge, MD, McGill University 

1:45 PM – 2:00 PM ALS Clinical Trials - Challenges and Updates
John Ravits, MD, Professor of Neurosciences, University of California, San Diego; Member, ALS Network Scientific Advisory Committee

2:00 PM – 2:15 PM The Future of ALS Clinical Trials
Merit Cudkowicz, MD, Chief of Neurology Mass General Hospital, Julieanne Dorn Professor of Neurology Harvard Medical School, Director Sean M. Healey & AMG Center for ALS at Mass General Hospital

2:15 PM – 2:45 PM DISCUSSION

3:00 PM – 3:20 PM Clustering of ALS Cases Using ML-based Phenotyping of Patient Fibroblasts
Steven Altshuler, PhD, University of California, San Francisco
Lani Wu, PhD, University of California, San Francisco 

3:20 PM – 3:40 PM Development of an UNC13A ASO for ALS
Wen-Hsuan Chang, PhD, AcuraStem – CIRM TRAN1 Project  

3:40 PM – 4:00 PM Development of a VAV2 Antisense Oligonucleotide (ASO) Treatment for ALS
Justin Ichida, PhD, University of Southern California  – CIRM DISC2 Project 

4:00 PM – 4:20 PM Survival, Migration and Differentiation into Non-reactive GDNF-secreting GLAST-Positive Astrocytes Following Transplantation into the Motor Cortex of ALS Patients
Clive Svendsen, PhD, Cedars-Sinai – CIRM CLIN2 Project

4:20 PM – 4:30 PM DISCUSSION

4:30 PM – 5:00 PM  Special Plenary Lecture
Synaptic Plasticity in Alzheimer’s Disease – Lessons for ALS
Thomas Sudhof MD, PhD, Avram Goldstein Professor in the School of Medicine, Professor of Neurosurgery, Nobel Laureate 2013   

5:00 PM – 5:15 PM DISCUSSION

5:15 PM END OF DAILY SESSION

9:20 AM – 9:35 AM Prilenia Updates
Michael Hayden, CEO, Prilenia
Merit Cudkowicz,  MD, Chief of Neurology Mass General Hospital, Julieanne Dorn Professor of Neurology Harvard Medical School, Director Sean M. Healey & AMG Center for ALS at Mass General Hospital

9:35 AM - 9:55 AM Updates on ALS Drug Development at Denali
Joe Lewcock, Chief Scientific Officer, Denali Therapeutics

9:55 AM - 10:10 AM SEANOBI-ALS Expanded Access Program for ibudilast in ALS
Jess Rabourn, CEO, WideTrial, Inc.

10:10 AM - 10:25 AM Trace Neuroscience 

Restoring UNC13A as a treatment for people with ALS
Shila Mekhoubad, PhD,  Head of Biology

10:25 AM – 10:30 AM DISCUSSION

10:45 AM – 11:00 AM Longitudinal Characterization of Reproducible ALS Phenotypes in iPSC-derived Sensorimotor Organoids
Joáo Pereira, PhD, University of Alabama, Birmingham  

11:00 AM – 11:15 AM Benchmarking Region-specific Organoids and Cortico-motor Assembloids to Study ALS
Jimena Andersen, PhD, Emory University 

11:15 AM – 11:30 AM Probing Consistent and Reproducible Pathophysiological Phenotypes in the Novel Forebrain-spinal Circuitoid Models Derived from ALS Patients
Liang Oscar Qiang, MD, PhD, Drexel University and Daryl Bosco, PhD University of Massachusetts

11:30 AM – 11:45 AM A Novel Human Cortico-spinal Assembloid Model of ALS/FTD
Kornelia Szenbenyi, HUN-REN Research Centre for Natural Sciences, Hungary 

11:45 AM – 12:00 PM Characterization of the Human ALS Motor Neuraxis Organoid System for Disease-modeling
András Lakatos, PhD, University of Cambridge, UK

12:00 PM – 12:30 PM DISCUSSION