Redefining conditioning for a new era of gene therapy

The conditioning process is an essential element of the ex vivo lentiviral gene therapy process, designed to enable durability and head-to-toe reach. In this expert-led session, you’ll learn about an advance in precision dosing of busulfan hailed as “a real game-changer” by Harry Malech, M.D., Chief of the Genetic Immunotherapy Section at the NIH’s NIAID, and how one company, AVROBIO, is leading the way in its goal of transforming the patient experience.

You’ll also hear about:
  • A seminal study that, after many years of uncertainty, pinpointed the optimal range of busulfan dosing for conditioning
  • New technology advancements for titrating busulfan exposure for each individual patient, designed to maximize engraftment and minimize side effects
  • Emerging clinical data about the tolerability and safety profile of busulfan when used as a single agent in a single cycle of conditioning
  • Early clinical data on busulfan’s effect on transiently depleting the bone marrow while sparing B and T cells
  • Busulfan’s impact on durability and head-to-toe reach – throughout both the body and the brain – in ex vivo lentiviral gene therapy
The discussion will be led by:
  • Harry L. Malech, M.D., Chief of the Genetic Immunotherapy Section at the National Institute of Allergy and Infectious Diseases, of the National Institutes of Health.
  • Chris Mason, M.D., Ph.D., FRCS, FMedSci, Chief Scientific Officer of leading lentiviral gene therapy developer AVROBIO, Professor of Cell and Gene Therapy at University College London and Senior Editor of the journals Cell and Gene Therapy Insights and Regenerative Medicine.
  • Arsalan Arif, Founder and Publisher, Endpoints News
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Harry L. Malech, M.D.

Harry L. Malech, M.D.

Chief, Genetic Immunotherapy Section and Deputy Chief, Laboratory of Clinical Immunology and Microbiology in the National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH)

Dr. Harry Malech obtained his M.D. at Yale University, followed by clinical residency at the University of Pennsylvania and postdoctoral training at the National Cancer Institute and Yale University. He was an assistant and associate professor of Medicine at Yale University before returning to the NIH, where he has been a senior investigator since 1986. Dr. Malech is currently chairman of the NIH Institutional Biosafety Committee, which reviews all gene therapy protocols conducted at the NIH Clinical Center. He serves on the medical advisory board of the Immune Deficiency Foundation and is a clinical reviewer for the California Institute for Regenerative Medicine. Dr. Malech is an elected member of the Association of American Physicians and the American Society for Clinical Investigation. He was president of the American Society of Gene & Cell Therapy between 2014 and 2015.

Chris Mason, M.D., Ph.D.

Chris Mason, M.D., Ph.D.

Chief Scientific Officer, AVROBIO

Chris is a clinician-scientist with more than 25 years of cell and gene therapy experience spanning R&D, clinical medicine, bioprocessing and commercialization. He works to train the next generation of world-class scientists as professor of cell and gene therapy at University College London and reviews cutting-edge research from around the world as senior editor of the journals Cell and Gene Therapy Insights and Regenerative Medicine. Chris was instrumental in the founding of the Alliance for Regenerative Medicine (ARM), the UK-Israel Science Council and the London Regenerative Medicine Network. He’s on the scientific advisory board for several public and private companies, the UK Cell & Gene Therapy Catapult and the Canadian Centre for the Commercialization of Regenerative Medicine.

Arsalan Arif

arsalan arif

Founder & Publisher, Endpoints News

Arsalan Arif is a news media entrepreneur who set out in 2015 to build his vision of an independent biotech news company at Endpoints News.