Summit Schedule

All Times Listed in Pacific Standard Time

 

DAY 1: FRIDAY, JANUARY 19, 2024

 

7:00 AM - 8:00 AM


8:00 AM - 8:10 AM Introduction

 

SESSION I: ASOs FOR ALS

Chair: Don Cleveland, PhD, UCSD

 

8:10 AM - 8:40 AM

Neil Shneider, MD, PhD, Columbia - Silencing FUS and Other Rare Genetic Forms of ALS 

 

8:40 AM - 9:00 AM

Toby Ferguson, MD, PhD, Biogen - QALSODY

 

9:00 AM - 9:20 AM

Shila Mekhoubad, PhD, Maze Therapeutics - UNC13A as a Therapeutic Target for ALS

 

9:20 AM - 9:50 AM

Discussion

 

9:50 AM - 10:05 AM      BREAK

 

SESSION II: INDUSTRY AND CLINICAL TRIAL UPDATES I

Chair: Jill Goslinga, MD, MPH, UCSF & Michael McGrath, MD, PhD, UCSF, CSO, Neuvivo Inc


10:05 AM - 10:20 AM

Angela Genge, MD, FRCP(C), eMBA, ED ALS Center of Excellence, McGill University - A Randomized Phase 2 Study to Assess the Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Study of Darifenacin in Patients with ALS

 

10:20 AM - 10:35 AM

Michael McGrath, MD, PhD, UCSF, CSO, Founder: Neuvivo Inc. - NP001 and the Innate Immune System in ALS

 

10:35 AM - 10:50 AM

Wen-Hsuan Chang, PhD, AcuraStem - Development of a UNC13A Cryptic Exon Skipping Antisense Oligonucleotide as a Treatment for ALS

 

10:50 AM - 11:05 AM

Joe Lewcock, PhD, CSO, Denali Therapeutics - Updates on ALS Drug Development at Denali: Focus on DNL343 and DNL788

 

11:05 AM - 11:20 AM

Discussion

 

SESSION III: PLENARY SPEAKER

 

11:20 AM - 11:50 AM

Viviana Gradinaru, PhD, Director, Caltech - Delivering Therapies Across the Blood-Brain Barrier: Vehicles and Targets

 

11:50 AM - 12:00 PM

Discussion

 

12:00 PM - 1:00 PM   LUNCH BREAK - LA JOLLA BALLROOM, 2ND FLOOR

 

SESSION IV: THE WALS CLINICAL UPDATE

Chair: Nicholas Olney, MD, Providence

 

1:00 PM - 1:30 PM

Stephen Johnson, MD, Mayo Clinic - Quantifying ALS Disease Progression with Digital Biomarkers

 

1:30 PM - 2:00 PM

Maxwell Greene, MD, Stanford - New Medications and Controversies 


2:00 PM - 2:30 PM

Roundtable Discussion - Treatment Affordability and Access; Intrathecal Administration

 

2:30 PM - 2:45 PM     BREAK

 

SESSION V: CIRM FUNDED ALS RESEARCH AND CLINICAL TRIALS IN THE STATE OF CALIFORNIA

Chair: Leslie Thompson, PhD, UC Irvine

 

2:45 PM - 3:00 PM

Lisa McGinley, PhD, SSO, CIRM - Advancing Cell and Gene Therapies in California and Beyond

 

3:00 PM - 3:20 PM

Ziwei Huang, PhD, UCSD - Development of a New Therapeutic for Directing Target Specific Stem Cell Migration and Treatment

 

3:20 PM - 3:40 PM

Justin Ichida, PhD, USC - Development of a VAV2 Antisense Oligonucleotide (ASO) Treatment for ALS

 

3:40 PM - 4:00 PM

Clive Svendsen, PhD, Cedars-Sinai - Modeling and Treating ALS Using Stem Cell Technology

 

4:00 PM - 4:30 PM 

Discussion

 

4:30 PM - 6:00 PM

Barber ALS Research Award Poster Session - LA JOLLA BALLROOM, 2ND FLOOR

 

6:00 PM - 7:00 PM

Champions Cocktail Hour - LA JOLLA BALLROOM, 2ND FLOOR

 

7:00 PM

CHAMPIONS FOR CURES AND CARE - PACIFIC BALLROOM FOYER, 1ST FLOOR

Dinner, Awards, and Entertainment

For more information, to make a donation or view auction items visit http://alschampions.org


Please wear your Summit lanyard.



DAY 2: SATURDAY, JANUARY 20, 2024

 

8:00 AM - 9:00 AM     Breakfast - LA JOLLA BALLROOM, 2ND FLOOR

 

9:00 AM - 9:10 AM Introduction

 

9:10 AM - 9:30 AM

Presentations from the 2024 Barber ALS Research Award Winners

 

SESSION VI: HOT TOPICS - TDP-43 AND CRYPTIC EXONS

Chair: John Ravits, MD, UCSD

 

9:30 AM - 9:50 AM   

Eugene Yeo, PhD, MBA, UCSD - RNA Processing Misregulation in Aged Neurons

 

9:50 AM - 10:10 AM

Claire Clelland, MD, PhD, UCSF - Developing CRISPR Gene Therapy for C9orf72 FTD/ALS

 

10:10 AM - 10:30 AM

Ji-Young Youn, PhD, Hospital for Sick Children - Dynamic Changes in TDP-43 Nuclear Functions Coupled to its Condensate Properties

 

10:30 AM - 11:00 AM

Discussion

 

11:00 AM - 11:15 AM Break

 

Session VII: INDUSTRY UPDATES II

Chair: Angela Genge, MD, FRCP(C), eMBA, ED ALS Center of Excellence, McGill University

 

11:15 AM - 11:30 AM

Michael Hayden, CEO, Prilenia - Update on Progress of Pridopodine for ALS

 

11:30 AM - 11:45 AM

Lahar Mehta, MD, Head of Global Clinical Development, Amylyx Pharmaceuticals - Amylyx Clinical Update

 

11:45 AM - 12:00 PM

Alex Huang MD, PhD, Chief Medical Advisor, Amydis - Ocular Tracer Targeting TDP-43 for ALS/FTD

 

12:00 PM - 1:00 PM   Lunch Break

 

SESSION VIII: PROTEIN MISFOLDING AND TDP-43 BIOLOGY IN ALS

Chair: Albert La Spada, MD, PhD, FACMGG, UCI 


1:00 PM - 1:15 PM

Albert La Spada, MD, PhD, FACMGG, UCI - TDP-43 Dysregulation of Polyadenylation Site Selection is a Defining Feature of ALS/FTD Disease Pathogenesis 

 

1:15 PM - 1:45 PM 

David S. Eisenberg, D.Phil, UCLA - The Amyloid State of Proteins and of TDP-43 in Particular

 

1:45 PM - 2:05 PM 

Aaron Gitler, PhD, Stanford - TDP-43 Nuclear Loss in FTD-ALS Causes Widespread Alternative Polyadenylation

 

2:05 PM - 2:25 PM 

Daniel Mordes, MD, PhD, UCSF - A C9ORF72 Dipeptide Repeat Protein Induces STMN2 Loss of Function

 

2:25 PM - 2:45 PM 

Discussion

 

2:45 PM - 3:00 PM      Break

 

SESSION IX: INNOVATIONS, MODELING AND AI IN ALS RESEARCH

Chair: Justin Ichida, PhD, USC

 

3:00 PM - 3:10 PM 

Samuel Sances, PhD, Insitro - ML-Enabled Pooled High-Content Screening Platform for ALS Drug Target Discovery 

 

3:10 PM - 3:30 PM 

Jessica Rexach, MD, PhD, UCLA - Dynamic Disease-Associated Gene Regulatory Transitions Unmask Genetic Risk Associated Cell States 

 

3:30 PM - 3:50 PM 

Ryan Lim, PhD, Modulo Bio - Deciphering Neuroimmune Contributions in ALS: A Tri-Culture Platform Coupled with Causal Representation Learning

 

3:50 PM - 4:15 PM 

Discussion

 

4:15 PM - 4:45 PM 

Looking Ahead: Expanding the Impact of the California ALS Research Network

 

4:45 PM - 5:00 PM

Closing Remarks and Thoughts for the 15th Annual California ALS Research Summit