* FAID 2022 has ended successfully! Please find below the flashback video.
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 an 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 an exhibitor, simply follow the Registration link.
Or contact our Organizers.
For details, see Exhibitor Opportunities (pdf)
* For more information about 2023 event, please contact Thi Le at tal018@ucsd.edu
Agenda Items
Thursday, November 17
8:00 AM — 8:30 AM
Breakfast & Registration
* Breakfast is included with registration
8:30 AM — 9:00 AM
Welcome & Introduction
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
Guest of Honor: Theodore Friedmann, Emeritus Professor, Department of Pediatrics, UC San Diego School of Medicine
Mireille Guyader, Counselor for Science and Technology, Embassy of France in the United States
9:00 AM — 11:10 PM
Inborn Error of Metabolism
Chair : Farah Sheikh, Professor in Medicine, UC San Diego School of Medicine
09:00 - 09:20
Hematopoietic stem cell gene therapy for cystinosis
Stephanie Cherqui, Professor, Department of Pediatrics, UC San Diego School of Medicine
09:20 - 09:40
Liver-directed gene therapy for inherited metabolic disorders
Antoine Gardin, Pediatric Hepatologist, Bicêtre University Hospital and Inserm Unit UMR951, Genethon
09:40 - 10:00
Gene therapy for Danon disease & cardiomyopathies
Eric Adler, Director of Cardiac Transplant Program, UC San Diego School of Medicine
10:00 - 10:20
Academics & Industry: LEXEO’s perspective on integrating capabilities to advance meaningful treatments
Nolan Townsend, Chief Executive Officer, LEXEO Therapeutics
10:20 - 10:40 PANEL
10:40 - 11:10 BREAK
11:10 AM — 12:00 PM
Eye Disorders
Chair : Pedro Gonzalez-Alegre, Head of Gene Therapy Research, Spark Therapeutics
11:10 - 11:30
Update on the current state of retinal gene therapy and gene editing
Shyamanga Borooah, Ophthalmology Assistant Professor, UC San Diego School of Medicine
11:30 - 11:50
From gene therapy in ophthalmology to cell therapy and visual restoration
Serge Picaud, Executive Director, Paris Vision Institute
11:50 - 12:00 PANEL
1:00 PM — 4:30 PM
Neuromuscular Disorders
Chair : Kumaran Deiva, Pediatric Neurologist, Assistance Publique–Hôpitaux de Paris
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
"Designer DNA drug" therapy for neurodegenerative disease
Don W. Cleveland, Department Chair of Cellular and Molecular Medicine, UC San Diego School of Medicine
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
Chair : Pascale Vincendon, Medical Director for Medical Affairs, Orchard Therapeutics
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
& Christina Theodore-Oklota, VP of Clinical Outcomes Research, 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
8:00 AM — 8:20 AM
Breakfast & Registration
* Breakfast is included with registration
8:20 AM — 8:30 AM
Welcome & Introduction
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
Chair : Sandip Patel, Medical Oncology Associate Professor, UC San Diego School of Medicine
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
* Lunch is included with registration
1:00 PM — 2:20 PM
Neurometabolic Disorders
Chair : Subhojit Roy, Pathology and Neuroscience Professor, UC San Diego School of Medicine
01:00 - 01:20
How to use gene therapy for neurometabolic diseases? In vivo or ex vivo?
Caroline Sevin, Pediatric Neurologist, Assistance Publique–Hôpitaux de Paris
01:20 - 01:40
Long term follow-up of intra cerebral gene therapy : experience of MPSIIIB
Kumaran Deiva, Pediatric Neurologist, Assistance Publique–Hôpitaux de Paris
01:40 - 02:00
For rare diseases uncovered medical need : clinical development in ex-vivo autologous HSC gene therapy
Pascale Vincendon, Medical Director for Medical Affairs, Orchard Therapeutics
02:00 - 02:20 PANEL
2:20 PM — 2:30 PM
Closing Remarks
2:30 PM — 4:00 PM
Networking Event
* Complimentary drinks & snacks will be served
Meet the Organizers
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
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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- Parking is available for $3 per hour on-site
- For detailed directions please CLICK HERE
- Nearby hotel recommendation -- Estancia
- Flight: San Diego International Airport (SAN)
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