Dr. Jim Wilson Departs UPenn to Launch New Gene Therapy Companies

Dr. Jim Wilson has officially departed the University of Pennsylvania after 31 years, during which he served as a professor at the Perelman School of Medicine and led the Gene Therapy Program (GTP) and Orphan Disease programs for decades. The GTP is now being transformed into two new affiliated companies: GEMMA Biotherapeutics (GEMMABio) and Franklin Biolabs. Both companies will take over the GTP facility located at the Discovery Labs campus in King of Prussia, PA. This expansive one-million-square-foot facility, previously the GDK R&D site, is also home to SK Pharmateco's Advanced Therapy CMDO, formerly known as the Center for Breakthrough Medicines.

Under Dr. Wilson's leadership, the GTP became a leader in discovery and translational research, concentrating on next-generation gene transfer vectors for various diseases. His efforts have significantly advanced gene transfer technology, and the GTP's team of over 350 employees has transitioned to new roles in the affiliated companies, with Wilson becoming CEO of GEMMA Biotherapeutics and chair of Franklin Biolabs.

Gemma Bio will focus on biotechnology research aimed at rare diseases, while Franklin Biolabs will operate as a contract research organization, providing essential support for gene therapy services and production. Both companies will be based at the Discovery Labs campus, continuing Dr. Wilson's legacy of innovation and commitment to advancing the future of gene therapy.

About Dr. James Wilson

Dr. Wilson dedicated over thirty years as a professor at the Perelman School of Medicine at UPenn, where he spearheaded the Gene Therapy Program (GTP). His research focused on developing next-generation gene transfer vectors for both acquired and inherited diseases, particularly virus-based vectors for in vivo gene therapy. He was instrumental in creating best-in-class adeno-associated viruses (AAVs), which are the basis for therapies like Glybera® (AAV1), Luxturna® (AAV2), and Zolgensma® (AAV9).

A significant milestone in his career was advancing therapeutic in vivo genome editing in large animal models, which enhanced the potential of AAVs. His pioneering work led to the discovery of novel capsids such as AAV8, which targets the liver and muscle, and AAV9, known for its ability to cross the blood-brain barrier.

Despite challenges, including the tragic 1999 death of Jesse Gelsinger, which temporarily slowed progress in the field, Dr. Wilson redirected his focus toward developing safer AAV vectors, which are now essential to modern gene therapy. His contributions have resulted in numerous innovations, more than 550 published papers, 110 patents, and the establishment of successful gene therapy companies, cementing his legacy in the field.

Franklin Biolabs

Franklin Biolabs, one of two companies recently spun out from Dr. James Wilson’s Gene Therapy Program at UPenn, is a newly launched Contract Research Organization (CRO) set to reshape the genetic medicines landscape. Headquartered in Philadelphia, named after Ben Franklin, Franklin Biolabs offers a full range of services, from discovery to clinical vector manufacturing. The company is backed by Savanne Life Sciences, the investment arm of Bioculture Group based in Mauritius. As the sole investor, Savanne Life Sciences provides the financial support and strategic resources necessary to drive Franklin Biolabs' mission forward.

Franklin Biolabs focuses on advancing gene therapy by providing specialized expertise in immunology, vector production, next-generation sequencing, bioinformatics, and nonclinical pharmacology/toxicology studies. With over 130 experts, the company aims to bridge the gap between cutting-edge research and the delivery of transformative therapies for severe diseases. Dr. Wilson emphasized their mission to accelerate the development and market entry of gene therapies through a full suite of services designed to reduce both time and costs.

The partnership with Savanne Life Sciences enhances Franklin Biolabs' capacity to bring disruptive gene therapy solutions to the forefront, leveraging extensive resources and expertise to ensure rapid translation of research into clinical applications. This collaboration positions Franklin Biolabs as a key player in the gene therapy sector, offering new hope to patients worldwide.

GEMMABio

GEMMA Biotherapeutics (GEMMABio), a new therapeutics company founded by gene therapy pioneer Dr. Jim Wilson, has announced a substantial $100 million partnership with Brazil's Oswaldo Cruz Foundation (Fiocruz). This collaboration aims to improve access to gene therapy treatments through Brazil's publicly funded healthcare system, Sistema Único de Saúde (SUS). Fiocruz, which supplies immunotherapies and biopharmaceuticals to the Brazilian Ministry of Health, will allocate the funding toward clinical research and manufacturing, facilitating the approval and distribution of rare disease treatments at more affordable prices.

"By combining our resources and expertise, this partnership represents a significant step forward in making gene therapies accessible to more patients quickly and at a fraction of the typical cost," said Dr. Wilson, who will serve as President and CEO of GEMMABio. "We are hopeful that this agreement will serve as a global model for expanding access to advanced medical therapies and for exploring innovative approaches to funding biotech companies."

Dr. Wilson emphasized that the collaboration marks a major advancement in making gene therapies accessible to more patients quickly and economically. A critical component of the partnership includes technology transfer to replicate GEMMABio's manufacturing processes in Brazil. This agreement will also support clinical development for six core programs, targeting diseases such as GM1 gangliosidosis and Krabbe Disease. In the press release Marco Krieger, Vice President of Production and Innovation in Health at Fiocruz, expressed pride in bringing innovative therapies to Brazil’s vast public health system, making previously restricted technologies accessible to the entire population. GEMMABio's mission is to expedite research and global access to life-changing therapies for those living with rare diseases, translating scientific discoveries into clinical applications effectively.

In August GEMMABio candidates from Passage Bio, a company previously founded by Wilson. These therapies target pediatric conditions such as GM1 gangliosidosis and Krabbe disease, with Gemma Bio committing $10 million to Passage Bio and taking over its remaining financial obligations to UPenn. Since joining UPenn in 1993, Wilson has been a pivotal figure in the gene therapy landscape, playing a key role in advancing safer gene delivery methods.