Symposium 2024 Program

7:30 AM

Breakfast & Registration

8:30 AM

Welcome & Introduction - GTI Video

Stephanie Cherqui, PhD, Professor, Department of Pediatrics, UC San Diego

Nancy and Geoffrey Stack Family Foundation

Session 1: Ex Vivo Hematopoietic Stem Cell Gene Therapy:
Broad Applications and Therapeutic Potential

Chair: Chris Mason, MD, PhD
Clinical Professor, University College London

08:55 AM

Masayuki Kai, PhD, VP, Head of Research, Kyowa Kirin, Inc.

09:40 AM

From cystinosis to Friedreich’s ataxia and Alzheimer’s disease: lessons learned treating a rare metabolic disease using ex vivo gene therapy

Stephanie Cherqui, PhD, Professor, Department of Pediatrics, UC San Diego

10:10 AM

Break

Session 2: AAV-Mediated Gene Therapy:
Challenges, Innovations and Clinical Applications

10:25 AM

Redefining the landscape of arrhythmogenic cardiomyopathy:
from mechanistic understanding to lifesaving interventions

Farah Sheikh, PhD, Professor, Department of Medicine, UC San Diego

10:50 AM

Neuron-targeted caveolin gene therapy to mitigate neurodegeneration

Brian Head, PhD, Professor, Department of Anesthesiology, UC San Diego

11:05 AM

Design and evolution of new AAV serotypes with expanded functionality for gene therapy

Loren Looger, PhD, Professor, Department of Neurosciences, UC San Diego

11:20 AM

Onasemnogene Abeparvovec-xioi for spinal muscular atrophy: bench to bedside

Nayla Mumneh, MD, Executive Medical Director, NeuroMuscular, Novartis
Pharmaceuticals Corporation

11:45 AM

Q & A Panel

12:00 PM

Lunch / Scientific Poster session

1:00 PM

Afternoon Session Introduction

Gaby Haddad, MD, Chair of Pediatrics, UC San Diego

Alysson Muotri, PhD, Professor, Department of Pediatrics, UC San Diego

Session 3: Testing Gene Therapies in Induced Pluripotent Stem Cell Models

Chair: Martin Marsala, MD
Professor, UC San Diego

1:15 PM

Brain organoids as a pre-clinical model for neurological conditions

Alysson Muotri, PhD, Professor, Department of Pediatrics, UC San Diego

1:40 PM

CRISPR introduction of an amyloid plaque-protecting microglial gene

Angels Almenar-Queralt, PhD, Assistant Professor, Pediatrics, UC San Diego

1:55 PM

Q & A Panel

Session 4: Resources & Support for the Development of
Gene Therapy Strategies for Rare Diseases

Chair: Betty Cabrera, MPH
Director of Engagement and Operations, UC San Diego Gene Therapy Initiative

2:05 PM

The power of patient advocacy: passion, determination and collaboration

Nancy Stack, President, Cystinosis Research Foundation, Patient Advocate

2:15 PM

The Platform Vector Gene Therapy (PaVe-GT) program at NIH, a large collaboration among NCATS, National Human Genome Research Institute (NHGRI), and the National Institute for Neurological Disorders and Stroke (NINDS)

Rodica Stan, PhD, Senior Scientific Project Manager, Therapeutic Development Branch, Division of Preclinical Innovation, National Center for Advancing Translational Sciences (NCATS), NIH

2:25 PM

Advancing cell and gene therapies in California and beyond

2:35 PM

Moderated Panel

3:05 PM

Break

Session 5: Therapeutic Advances in RNA Technologies for the
Treatment of Rare and Ultra-Rare Diseases

Chair: Shyamanga Borooah, MD, PhD
Assistant Professor, UC San Diego

3:20 PM

Advancing novel precision medicine therapies: AI enabled antisense oligonucleotide design for treatment of an ultra-rare disorder

Nicole Coufal, MD, PhD, Associate Professor, Pediatrics, UC San Diego

3:45 PM

Vectorized RNA-targeting therapeutics 

Gene Yeo, PhD, MBA, Professor, Cell and Molecular Medicine, UC San Diego

4:10 PM

Developing RNA-therapeutics for neurological diseases

Holly Kordasiewicz, PhD, Vice President Neurology Research, Ionis Pharmaceuticals, Inc.

4:35 PM

Q & A Panel

4:50 PM

Summary and Concluding Remarks

Alysson Muotri & Stephanie Cherqui, Co-Directors of GTI

5:00 PM

Networking Hour/ Scientific Poster session