Prominent gene therapy researcher Jim Wilson is departing the University of Pennsylvania after three decades to establish two new companies that will spin out the majority of the university's Gene Therapy Program. Wilson, who has been a major figure in the field since joining UPenn in 1993, will serve as CEO of Gemma Biotherapeutics and chairman of Franklin Biolabs.
The decision reflects Wilson's desire to ensure that discoveries made at the Gene Therapy Program over 30 years are translated into effective treatments accessible to patients, particularly during a downturn in biotech funding that has left several gene therapy programs struggling. "Forming these two new entities is the next step to accelerate the future of gene therapy and deliver therapeutics to patients significantly faster," Wilson stated.
Company Structure and Focus
Gemma Biotherapeutics will function as a biotechnology company focused on rare disease research and serve as the research and innovation arm to speed global access to advanced therapies. Franklin Biolabs will operate as a contract research organization, providing services from discovery to development and distribution for the global genetic medicines industry.
Both companies will be based in the greater Philadelphia area with an international network extending to the United Kingdom, Brazil, South Korea and other locations. The majority of the Gene Therapy Program's staff will be offered positions at one of the companies.
Clinical Pipeline and Licensing
Gemma Bio has licensed three clinical-stage gene therapy candidates from Passage Bio, a company previously founded by Wilson. The three treatments all target pediatric conditions, including GM1 gangliosidosis and Krabbe disease. Gemma Bio is paying Passage Bio $10 million and taking over remaining financial obligations that Passage Bio had to UPenn.
An unnamed investor syndicate has backed Gemma Bio, while Franklin Biolabs received funding from a single investor.
Wilson's Legacy and Impact
Wilson began his work in gene therapy nearly 40 years ago and created the first and largest academic-based program in gene therapy at Penn. 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 set the stage for the recent resurgence of gene therapy.
Wilson's career has included both triumphs and setbacks. He led a trial of an early gene therapy that resulted in the 1999 death of study volunteer Jesse Gelsinger, a tragedy that curtailed research for years until safer methods for delivering genes into the body were developed, with significant contributions from Wilson's program at UPenn.
His research has focused on rare inherited diseases, ranging from cystic fibrosis to dyslipidemias to neurologic disorders and liver metabolic diseases, addressing 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.
Industry Impact
UPenn research helped lead to three of the gene therapies that have received FDA approval. In addition to Passage Bio, Wilson has helped found Regenxbio and G2 Bio, bringing his total number of biotechnology companies established to eight. The new companies represent Wilson's continued effort to facilitate the clinical and commercial development of gene therapy platforms through entrepreneurial ventures.
