Biogen Makes Major Investment in Gene-Therapy Development at the University of Pennsylvania

May 20, 2016

Biogen, a leading international biotech company, has announced investments of up to $2 billion in the development of gene therapies and gene-editing technologies at the University of Pennsylvania for a variety of conditions, including inherited retinal diseases.

The company will collaborate with world-renowned gene-therapy experts James Wilson, M.D., Ph.D., director of the university’s gene-therapy program, and Jean Bennett, M.D., Ph.D., who led the advancement of RPE65 gene-replacement therapy for retinitis pigmentosa and Leber congenital amaurosis through a clinical trial. Spark Therapeutics is now seeking approval for the vision-restoring treatment from the U.S. Food and Drug Administration (FDA). It has the potential to become the first FDA-approved gene therapy for an inherited or ophthalmological condition.

Biogen will make an upfront payment to the university of $20 million, with an additional $62.5 million committed to fund costs extending over the next three to five years in seven preclinical research-and-development programs. Each program may trigger payments for reaching therapy-development milestones, from $77.5 million to $137.5 million per product as well as royalties.

“Biogen’s commitment tremendously boosts our strong momentum in getting sight-saving gene therapies out to the millions who need them,” says William T. Schmidt, chief executive officer at the Foundation Fighting Blindness (FFB). “We’ve been partnering with Dr. Bennett and the University of Pennsylvania for several decades to advance these promising treatments into human studies. It’s wonderful to see our sustained funding attract support from a leading biotech.”

The collaboration includes the development and enhancement of adeno-associated viruses (AAVs), human-engineered viruses which are performing well in delivering therapeutic genes in clinical and lab studies for retinal conditions, including Spark’s RPE65 clinical trial.

The university’s scientists will also advance gene-editing technologies such as clustered regularly interspaced short palindromic repeats (CRISPR/Cas9), which are used to modify — i.e., cut and/or paste — only the defective region of a gene instead of replacing the whole gene. Gene editing may, for some diseases, provide advantages over gene replacement using AAVs. For example, gene editing might be a way to treat retinal diseases in which AAVs don’t have the capacity to deliver large genes, such as USH2A and CEP290.

The University of Pennsylvania collaboration is Biogen’s second foray into the advancement of retinal-disease gene therapies. In July 2015, Biogen announced a licensing and collaboration agreement with Applied Genetic Technologies Corporation (AGTC), an FFB partner developing gene therapies for a number of retinal conditions, including: X-linked retinoschisis, achromatopsia, X-linked retinitis pigmentosa, and age-related macular degeneration. Under the terms of the agreements, Biogen has thus far made a $124 million payment to AGTC. AGTC is eligible to receive payments exceeding $1 billion, some of which are for achieving therapy-development milestones.