The Alliance for Regenerative Medicine and US Pharmacopeia held a 1.5 day workshop on AAV Analytical Characterization on March 8-9, 2023, in the Washington DC area. This workshop covered various topics on AAV quality, including full / empty capsid characterization, potency assay, and titer measurements.
Adeno-associated virus (AAV)-based gene therapies have shown promise for the treatment of many diseases, including rare diseases with unmet medical needs. Despite their extensive clinical application and licensure as marketed products, further efforts are required to increase product understanding with regard to their characteristics that may have an impact on clinical efficacy, safety, or immunogenicity. This workshop will cover various topics on AAV analytical characterization, including full / empty capsid characterization, potency assay, and titer measurements. This workshop aims to review state-of-the-art methodologies, share learnings on approaches for different products, with the ultimate goal of moving towards harmonized methods and agreements on potency expectations.
Josephine Lembong, Ph.D., Manager, Scientific and Industry Affairs, Alliance for Regenerative Medicine (ARM)
Diane McCarthy, Senior Director, Biologics, US Pharmacopeia (USP)
Khandan Baradaran, Ph.D., Vice President, Regulatory CMC, Ultragenyx Pharmaceutical
Jerome Jacques, Principal Scientist, USP
Rahul Sheth, Ph.D., Senior Scientist 2, Purification Process Development, BioMarin Pharmaceutical
Lauriel Earley, Ph.D., Senior Scientist, Shape Therapeutics
Adriana Kita, Ph.D., Associate Director, Analytical Development, Ultragenyx Pharmaceutical
Khandan Baradaran, Ph.D., Vice President, Regulatory CMC, Ultragenyx Pharmaceutical (Moderator)
Andrew Byrnes, Ph.D., Chief, Gene Transfer and Immunogenicity Branch, U.S. Food and Drug Administration (FDA)
Lauriel Earley, Ph.D., Senior Scientist, Shape Therapeutics
Jerome Jacques, Principal Scientist, USP
Adriana Kita, Ph.D., Associate Director, Analytical Development, Ultragenyx Pharmaceutical
Rahul Sheth, Ph.D., Senior Scientist 2, Purification Process Development, BioMarin Pharmaceutical
Christopher Miyake, Senior Director, Regulatory Affairs CMC, Sarepta Therapeutics
Erandi De Silva, Ph.D., Co-founder and Senior Vice President, Product Development, Forge Biologics
Connie Tsai, Ph.D., Associate Director, Analytical Development Bioassay, Novartis Gene Therapies
Ravindra Kumar, Ph.D., Director, Bioassay Lead, Analytical Sciences and Quality Control, Spark Therapeutics
Josephine Lembong, Ph.D., Manager, Scientific and Industry Affairs, Alliance for Regenerative Medicine (ARM) (Moderator)
Erandi De Silva, Ph.D., Co-founder and Senior Vice President, Product Development, Forge Biologics
Denise Gavin, Ph.D., Chief, Gene Therapy Branch, U.S. Food and Drug Administration (FDA)
Christopher Miyake, Senior Director, Regulatory Affairs CMC, Sarepta Therapeutics
Ravindra Kumar, Ph.D., Director, Bioassay Lead, Analytical Sciences and Quality Control, Spark Therapeutics
Connie Tsai, Ph.D., Associate Director, Analytical Development Bioassay, Novartis Gene Therapies
Josephine Lembong, Ph.D., Manager, Scientific and Industry Affairs, Alliance for Regenerative Medicine (ARM)
Josephine Lembong, Ph.D., Manager, Scientific and Industry Affairs, Alliance for Regenerative Medicine (ARM)
Pierre Caloz, Chief Operating Officer, uniQure
Roberto Calcedo, Ph.D., Vice President, Preclinical and Immunology, Affinia Therapeutics
Matthew Hewitt, Ph.D., Vice President, Technical Officer CGT and Biologics, Charles River Laboratories
Adam Davis, Ph.D., Vice President, Analytical Development, Forge Biologics
David Dobnik, Ph.D., Co-founder and Chief Scientific Officer, Niba Labs
Andrea Hamilton, Ph.D., Associate Director, Quality Control, Gyroscope Therapeutics
Andrew Harmon, Ph.D., Lead Biological Reviewer, Gene Therapy CMC, U.S. Food and Drug Administration (FDA)
Matthew Hewitt, Ph.D., Vice President, Technical Officer CGT and Biologics, Charles River Laboratories (Moderator)
Roberto Calcedo, Ph.D., Vice President, Preclinical and Immunology, Affinia Therapeutics
Pierre Caloz, Chief Operating Officer, uniQure
Adam Davis, Ph.D., Vice President, Analytical Development, Forge Biologics
David Dobnik, Ph.D., Co-founder and Chief Scientific Officer, Niba Labs
Andrea Hamilton, Ph.D., Associate Director, Quality Control, Gyroscope Therapeutics
Andrew Harmon, Ph.D., Lead Biological Reviewer, Gene Therapy CMC, U.S. Food and Drug Administration (FDA)
Josephine Lembong, Ph.D., Manager, Scientific and Industry Affairs, Alliance for Regenerative Medicine (ARM)
12601 Twinbrook Pkwy
Rockville, MD 20852
Please contact Josephine Lembong at jlembong@alliancerm.org