James Wilson Departs Penn to Launch to Spin Out Penn Programs into Two New Gene Therapy Companies

Rare Daily Staff

Gene therapy pioneer James Wilson is stepping down from his post as director of the Gene Therapy Program at the Perelman School of Medicine at the University of Pennsylvania to spin out the work of the program into two new gene therapy companies he founded to commercialize the work.

The effort reflects a desire to ensure that discoveries made at the Gene Therapy Program over 30 years are translated into effective treatments that will be accessible to patients at a time when a downturn in funding for biotech companies has left the advancement of several gene therapy programs faltering.

“Forming these two new entities is the next step to accelerate the future of gene therapy and deliver therapeutics to patients significantly faster,” said Wilson, who will serve as CEO of GEMMABio and chairman of Franklin Biolabs.

GEMMA Biotherapeutics will serve as the research and innovation arm to speed the research of and global access to advanced therapies for those living with rare diseases. The second company, Franklin Biolabs, is a contract research organization that will serve as the services and production arm. It will provide services from discovery to development and distribution for the global genetic medicines industry.

The two companies will operate independently and both be based in the greater Philadelphia area with an international network extending to the United Kingdom, Brazil, South Korea and more.

Funding for GEMMABio is coming from a syndicate of several investors and investment groups, while funding for Franklin Biolabs is coming from a single investor.

Wilson began his work in gene therapy nearly 40 years ago and was recruited to Penn in 1993. He created the first and largest academic-based program in gene therapy. His laboratory discovered a family of viruses from primates called adeno-associated viruses (AAV) that could be engineered to be effective gene transfer vehicles. These “vectors” have become the technology platform of choice and have set the stage for the recent resurgence of the field of gene therapy.

Wilson has also been active in facilitating the clinical and commercial development of these new gene therapy platforms through the establishment of eight biotechnology companies. His research has been focused on rare inherited diseases, ranging from cystic fibrosis to dyslipidemias to a variety of neurologic disorders and liver metabolic diseases, and on addressing these unmet needs for patients in marginalized populations. To date, his team’s accomplishments include three FDA-approved AAV-based gene therapies, more than 40 active programs in development, 95 patents on gene therapy-related technologies, and first-in-patient studies in 15 different diseases.

Photo: James Wilson, CEO of GEMMABio and chairman of Franklin Biolabs