RARE Webinar: Understanding Gene Therapy
May 11, 2021
Recorded webinar/Download slides
Scientific discovery is advancing rapidly, and new frontiers in gene therapy may yield promise for new rare disease treatments. You may have heard the term gene therapy and even know a little bit about the science, but this webinar will cover all the basics and walk through the potential impact on the rare disease community, including case study examples of collaborations between companies and patient advocacy groups. If you need a refresher or are new to gene therapy and how patients are getting engaged in research and trials, this Webinar can’t be missed!
If you are unable to attend the live webinar register anyways and you will receive a link to the recorded content.
Michelle Berg, Vice President, Patient Advocacy for Abeona Therapeutics
Michelle joined the company in June of 2015. Previously, she was the first hire with Aldevron, LLC now a leading global contract manufacturing organization specializing in biological reagents for R&D through clinical trial use. During her time there, she held multiple positions with increasing responsibility, contributing to the growth and reach of the organization. Additionally, she performed research in the Plant Sciences Department at North Dakota State University. Her B.S. in Biotechnology was attained from North Dakota State University in 1997. She brings over 17 years of experience in gene and cell therapies, vaccines, gene editing and molecular diagnostics.
Alison Rockett Frase, President and Founder, Joshua Frase Foundation
Alison and Paul Frase founded the The Joshua Frase Foundation (JFF) 20 years ago in hopes of finding a cure for their late son Joshua who was born with a rare deadly neuromuscular disorder, myotubular myopathy (MTM), and continue forging a path towards clinical trials for his peers. JFF’s fundraising efforts raised over 6 million dollars which initiated a research program focused on MTM and other related congenital myopathies. Alison founded and supported the only MTM canine colony currently used for gene therapy research, implemented a global patient registry, the registry is now a member of the NIH/NCATS GRDR Program and serves on the access to data committee as a patient advocate representative, and provides a platform for patient advocacy and educational programs.
Karen Kozarsky, Ph.D., President, Vector BioPartners
Dr. Kozarsky is a consultant in gene therapy and President of Vector BioPartners, LLC. Most recently, she was Vice President of Research and Development at REGENXBIO Inc. where she was responsible for development of therapeutic programs utilizing adeno-associated virus (AAV) therapeutics for rare, inherited disease as well as acquired diseases. Previously, she headed the Gene Therapy group in the Biopharmaceuticals R&D Unit at GlaxoSmithKline. Prior to that, she worked at the University of Pennsylvania and the University of Michigan. Dr. Kozarsky received a Ph.D. in Biology from the Massachusetts Institute of Technology and a B.A. in Biology from Amherst College.
Daniel Levine, Founder & Principal, Levine Media Group
Daniel Levine is an award-winning business journalist who has reported on the life sciences, economic development, and business policy issues throughout his 25-year career. Since 2011, he has served as the lead editor and writer of Burrill Media’s acclaimed annual book on the biotech industry and hosts The Burrill Report’s weekly podcast. His work has appeared in The New York Times, The Industry Standard, TheStreet.com, and other national publications.
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