20th Gordon L. Snider Critical Issues Workshop: The Promise of Gene-based Interventions in Alpha-1 Antitrypsin Deficiency

December 12, 2020

The Alpha-1 Foundation hosted the virtual 20th Gordon L. Snider Critical Issues Workshop entitled, The Promise of Gene-based Interventions in Alpha-1 Antitrypsin Deficiency on Saturday, December 12, 2020. The day was divided into three sections: The Advantages and Limitations of Gene Therapy, RNA Editing and Silencing and Gene Editing. The overall goal of the workshop was accomplished as exchange was facilitated between alpha-1 antitrypsin experts and the research community, including those working with viral vectors, RNA therapeutics and gene editing. The meeting allowed researchers to exchange their findings in a collaborative forum to help advance science in the pursuit of a cure for Alpha-1. This is promising news for Alphas that reinforces the advancement of research programs in Alpha-1 and the genome editing treatment approach.

“Researchers from both academia and industry presented their latest work on gene and cell-based therapies, gene silencing and gene editing.  Presentations including some that were particularly focused on alpha-1 antitrypsin deficiency-related lung disease or liver disease, while others provided examples of clinically successful programs that might provide insights into new platforms for alpha-1 therapies. Potential opportunities and limitations and future directions were actively discussed.” – Dr. Terry Flotte

The workshop was chaired by Terry Flotte, MD and Kenneth Irvine.  Dr. Terence Flotte currently is the Provost and Executive Deputy Chancellor, Celia and Isaac Haidak Professor of Medical Education, and Dean of the University of Massachusetts Medical School. An internationally known pioneer in human gene therapy, Dr. Flotte is currently investigating the use of gene therapy for genetic diseases, including alpha-1 antitrypsin deficiency and Tay-Sachs disease. In 1995, he led the team at Johns Hopkins that became the first to use the apparently harmless adeno-associated virus, or AAV, as a vehicle to deliver corrective genes to targeted sites in the body, including the damaged airways of adults with cystic fibrosis. He is the author of more than 250 scholarly papers and his research has been funded by the National Institutes of Health and the Alpha-1 Foundation. Since 2015, he has also been Editor-in-Chief of his field’s oldest journal family, Human Gene Therapy. Mr. Kenneth Irvine is a long time Board Member of the Alpha-1 Foundation, and Chair of the Development and the Investment Committees. His life pursuit is using his Wall Street expertise to help find a cure for Alpha-1, which his late wife, Bettina, was diagnosed with 25 years ago. She received the best of care, a double lung transplant and enjoyed many years of additional life. Ken will always have skin in the game with three sons who are carriers and three beautiful granddaughters. He earned an MBA from Harvard Business School and a BA from University of Michigan.

“Terry Flotte brought together the best minds of the day. It is clear from the presentations that, just as genetic engineering expedited the development of a vaccine for COVID, this innovative approach has the power to find a CURE for Alpha-1.”- Kenneth Irvine

The agenda included presentations from researchers from both academia and industry. Thank you for making our program a success:

Mark L. Brantly, MD– University of Florida

Sean Burns, MD– Intellia Therapeutics

David T. Curiel, MD, PhD- Washington University School of Medicine in St. Louis

John F. Engelhardt, PhD– University of Iowa

Terence R. Flotte, MD- University of Massachusetts Medical School

James C. Hamilton, MD, MBA– Arrowhead Pharmaceuticals, Inc.

Kenneth Irvine, CFA- Alpha-1 Foundation Board Member

Allison Keller-Klunk, PhD- University of Massachusetts Medical School

Jin Billy Li, PhD- Stanford University

Christian Mueller, PhD– Sanofi Genzyme

Miriam O’Day- Alpha-1 Foundation

Michael Packer, PhD– Beam Therapeutics

Natalie Pursell, PhD- Dicerna Pharmaceuticals, Inc.

R. Jude Samulski, PhD- University of North Carolina

Erik J. Sontheimer, PhD-University of Massachusetts Medical School

Jeffrey Teckman, MD– St. Louis University School of Medicine

Bruce Trapnell, MD– Cincinnati Children’s Hospital Medical Center

James M. Wilson, MD PhD- University of Pennsylvania

Wen Xue, PhD– University of Massachusetts Medical School

Kate Zhang, PhD– Editas Medicine

“We were proud to host this open workshop on a virtual environment that fostered the collaboration of researchers, physicians and patients. With the patient as the focus of the mission of the Alpha-1 Foundation, the virtual platform allows Alphas to be a part of the day and hear first-hand about the advancements towards a cure through the dedication and commitment of our partners,” said Miriam O’Day, President & CEO of the Alpha-1 Foundation. 

Thank you to the generous sponsors for their support of the 20th GLS Workshop: AlphaNet, CSL Behring, Grifols, Takeda, University of Massachusetts Medical School and Vertex.

Save the Date for the 21st Gordon L. Snider Critical Issues Workshop titled Exacerbations and Pulmonary Comorbidities on January 29th & January 30th, 2021.