About the Event

The event will take place at the University of California San Diego on November 17 and 18, 2022, divided in different sessions on clinical trials related to neurodegenerative, metabolic, eye, muscular, hematologic and oncologic disorders, giving the floor to attendees from France and  the US.

The Clinical trials in Gene Therapy FAID is intended for industry, academia, regulatory, clinician communities involved in gene therapy clinical trials. It is particularly dedicated to those involved or interested in international perspectives for the development of multicenter trials.

Areas of Intervention

  • Presentation of clinical trials in gene therapy (planning, design and execution of clinical trials)
  • Presentation of national clinical infrastructure
  • Collaboration between academic and industry for the conduct of clinical trials in gene therapy
  • Regulation and international expansion of gene therapy clinical trials
Registration Information

Price: $155

Price: $55

Price: $295

As a sponsor/exhibitor, your company or organization will be acknowledged in the handout materials, conference’s website, and will receive access to the digital course syllabus. To become a sponsor/ exhibitor, simply follow the Registration link.

Or contact our Organizers.

For details, see Exhibitor Opportunities (pdf)

* All registration options include breakfast, lunch, and networking event.

Agenda Items

Thursday, November 17

8:20 AM — 8:30 AM

Registration & Welcome

Stephanie Cherqui, Professor, Department of Pediatrics, UC San Diego School of Medicine

Caroline Sevin, Pediatric Neurologist, Assistance Publique–Hôpitaux de Paris

Karim Belarbi, Attaché for Science and Technology, Embassy of France in the United States

Clara Devouassoux, Deputy Attachée for Science & Technology, Embassy of France in the United States

8:30 AM — 12:00 PM

Inborn Error of Metabolism

 08:30 - 08:50    Hematopoietic stem cell gene therapy for cystinosis
Stephanie Cherqui, Professor, Department of Pediatrics, UC San Diego School of Medicine

 08:50 - 09:10    Gene therapy for rare cardiomyopathies
Eric Adler, Director of Cardiac Transplant Program, UC San Diego School of Medicine

 09:10 - 09:30    Liver-directed gene therapy for inherited metabolic disorders
Antoine Gardin, Pediatric Hepatologist, Bicêtre University Hospital and Inserm Unit UMR951, Genethon

 09:30 - 09:50    Academics & Industry: LEXEO’s perspective on integrating capabilities to advance meaningful treatments
Nolan Townsend, Chief Executive Officer, LEXEO Therapeutics

 09:50 - 10:10    PANEL

 10:10 - 10:40    BREAK

 10:40 - 11:00    How to use gene therapy for neurometabolic diseases? In vivo or ex vivo?
Caroline Sevin, Pediatric Neurologist, Assistance Publique–Hôpitaux de Paris

 11:00 - 11:20    Long term follow-up of intra cerebral gene therapy : experience of MPSIIIB
Kumaran Deiva, Pediatric Neurologist, Assistance Publique–Hôpitaux de Paris

 11:20 - 11:40    For rare diseases uncovered medical need : clinical development in ex-vivo autologous HSC gene therapy
Pascale Vincendon, Medical Director for Medical Affairs, Orchard Therapeutics

 11:40 - 12:00    PANEL

12:00 PM — 1:00 PM

Lunch

1:00 PM — 4:30 PM

Neuromuscular Disorders

 01:00 - 01:20    Latest gene therapies in neuromuscular diseases
Chamindra Laverty (Konersman), Neurology Associate Professor, UC San Diego School of Medicine

 01:20 - 01:40    Gene therapy for neuromuscular and neurodegenerative diseases. What’s new and where are we going?
Giorgia Querin, Medical Director of I-Motion Adult & Pediatric Clinical Trials Platforms, Myology Institut

 01:40 - 02:00    AAV-based Gene Transfer Therapies for Patients Living with Duchenne Muscular Dystrophy
Katherine Brandl, Senior Medical Science Liaison, Sarepta Therapeutics

 02:00 - 02:20    Intravenous gene therapy in neuro muscular disease and new improvements
Françoise Piguet, Scientific Researcher, Institut du Cerveau – Paris Brain Institute (ICM)

 02:20 - 02:40    PANEL

 02:40 - 03:10    BREAK

 03:10 - 03:30    A gene therapy approach for Pitt-Hopkins using brain organoids / Development of a gene therapy for Pitt Hopkins syndrome
Alysson R. Muotri, Director of the Stem Cell Program, UC San Diego School of Medicine
& Jennifer Panagoulias, Chief Regulatory and Compliance, Mahzi Therapeutics

 03:30 - 03:50    Engineered RNA-targeting gene therapy approaches
Gene Yeo, Founding Member of Genomic Medicine Institute, UC San Diego School of Medicine

 03:50 - 04:10    Growth factor gene therapy for Alzheimer’s disease
Mark Tuszynski, Director of Translational Neuroscience Institute, UC San Diego School of Medicine

 04:10 - 04:30    PANEL

Friday, November 18

8:20 AM — 8:30 AM

Registration & Welcome

Stephanie Cherqui, Professor, Department of Pediatrics, UC San Diego School of Medicine

Caroline Sevin, Pediatric Neurologist, Assistance Publique–Hôpitaux de Paris

Karim Belarbi, Attaché for Science and Technology, Embassy of France in the United States

Clara Devouassoux, Deputy Attachée for Science & Technology, Embassy of France in the United States

8:30 AM — 12:00 PM

Cancer & Blood Diseases

 08:30 - 08:50    Clinical advances in solid tumor cell therapy
Sandip Patel, Medical Oncology Associate Professor, UC San Diego School of Medicine

 08:50 - 09:10    Chimeric antigen receptor T-cells therapy in pediatric acute lymphoblastic Leukemia : recent advances
Marie-Emilie Dourthe, Pediatric Hematologist, Assistance Publique–Hôpitaux de Paris

 09:10 - 09:30    Transport of an oncolytic virus to tumor cells via an innate immune cell
Pamela Contag, Chief Executive Officer, Bioeclipse Therapeutics

 09:30 - 09:50    Leukemia stem cell generation by base editing enzymes
Catriona Jamieson, Director Sanford Stem Cell Clinical Center & CIRM Alpha Stem Cell Clinic

 09:50 - 10:10    PANEL

 10:10 - 10:40    BREAK

 10:40 - 11:00    Cell-based gene therapy progress and challenges for pediatric cancers
Véronique Minard-Colin, Professor, Children and Adolescent Oncology Department, Gustave Roussy

 11:00 - 11:20    Engineering human pluripotent stem cells to produce immune cells with improved anti-tumor activity
Dan S. Kaufman, Director of Cell Therapy Program, UC San Diego School of Medicine

 11:20 - 11:40    Clinical AAV gene transfer for Hemophilia
Pedro Gonzalez-Alegre, Head of Gene Therapy Research, Spark Therapeutics

 11:40 - 12:00    PANEL

12:00 PM — 1:00 PM

Lunch

1:00 PM — 2:00 PM

Eye Disorders

 01:00 - 01:20    Update on the current state of retinal gene therapy and gene editing
Shyamanga Borooah, Ophthalmology Assistant Professor, UC San Diego School of Medicine

 01:20 - 01:40    From gene therapy in ophthalmology to cell therapy and visual restoration
Serge Picaud, Executive Director, Paris Vision Institute

 01:40 - 02:00    PANEL

2:00 PM — 2:10 PM

Closing Remarks

2:10 PM — 4:00 PM

Networking Event

Meet the Organizers

Professor
Department of Pediatrics
University of California, San Diego

Pediatric Neurologist
Assistance Publique–Hôpitaux de Paris
Institut du Cerveau ICM Paris

Attaché for Science & Technology
Office for Science and Technology
Embassy of France in the United States

Deputy Attachée, Science & Technology
Office for Science and Technology
Embassy of France in the United States

Speakers

Clinical Professor
UCSD School of Medicine

Assistant Professor
UCSD School of Medicine

Senior Medical Science Liaison
Sarepta Therapeutics

Chief Executive Officer
BioEclipse Therapeutics

Senior Neurologist
Bicêtre Hospital

Pediatrician
Assistance Publique–Hôpitaux de Paris

Pediatrician
Bicêtre University Hospital

Head of Gene Therapy Research
Sparks Therapeutics

Professor
UCSD School of Medicine

Professor
UCSD School of Medicine

Associate Professor
UCSD School of Medicine

Professor
Gustave Roussy

Professor
UCSD School of Medicine

Chief Regulatory & Compliance Officer
Mahzi Therapeutics

Associate Professor
UCSD School of Medicine

Executive Director
Paris Vision Institute

Head of Research Team
Paris Brain Institute

Lead Neurologist
Institute of Myology

Chief Executive Officer
Lexeo Therapeutics

Distinguished Professor
UCSD School of Medicine

Medical Director
Orchard Therapeutics

Professor
UCSD School of Medicine

Organizers

Karim Belarbi, PhD – Office for Science and Technology
Embassy of France in the United States

Stephanie Cherqui, PhD – Department of Pediatrics
University of California, San Diego

Caroline Sevin, MD, PhD – Assistance Publique
Hôpitaux de Paris – Institut du Cerveau ICM Paris

Our Sponsors

The Clinical Trials in Gene Therapy FAID will bring together principals investigators, sponsors, industry professionals, patient advocacy groups, and regulatory experts to share their experience on all aspects of Clinical Trials in Gene Therapy, around a program of case-study presentations, interactive panel discussions and networking opportunities.

Platinum

Gold

Silver

Bronze

Location

Sanford Consortium for Regenerative Medicine - Roth Auditorium
2880 Torrey Pines Scenic Drive, La Jolla, CA92307
Phone: (858) 246-1076
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Contacts

Stephanie Cherqui
scherqui@health.ucsd.edu

 

Caroline Sevin
caroline.sevin@inserm.fr

 

Karim Belarbi
attache-sdv.la@ambascience-usa.org

 

Clara Devouassoux
deputy-sdv.la@ambascience-usa.org