Biogen and the University of Pennsylvania said today they are launching up to $2 billion in collaborations aimed at developing gene therapies targeting the eye, skeletal muscle, and central nervous system (CNS).

Penn and Biogen will seek to validate next-generation gene transfer technology using adeno-associated virus (AAV) gene delivery vectors, as well as explore expanded use of genome editing technology as a potential therapeutic platform.

Biogen said it will work with two gene therapy researchers at Penn’s Perelman School of Medicine—James Wilson, M.D., Ph.D., professor of medicine and pediatrics, and director of Penn’s gene therapy program, and Jean Bennett, M.D., Ph.D., professor of ophthalmology and cell and developmental biology and director of the Center for Advanced Retinal and Ocular Therapeutics.

“By exploring next-generation delivery in various tissues such as the retina, skeletal muscle, and CNS, we will explore the potential for extending gene therapy beyond disorders linked to single-gene mutations and into a broader spectrum of complex diseases, including devastating neurological conditions that affect a multitude of patients throughout the world,” Olivier Danos, Ph.D., svp, cell & gene therapy at Biogen, said in a statement.

Penn has agreed to use its gene therapy resources and expertise to develop gene therapy candidates using both existing and newly developed AAV vectors, as well as to aid in the development of new manufacturing approaches toward commercializing of gene therapy products. Biogen agreed to use its therapeutic area and target identification expertise and drug development capabilities to help advance the collaboration programs into clinical phases and eventual approval.

Biogen agreed to pay Penn $20 million upfront, and an additional $62.5 million to fund R&D costs over the next 3–5 years in seven distinct preclinical R&D programs conducted by the laboratories of Dr. Wilson and Dr. Bennett. Each program may trigger milestones that range from $77.5 million to $137.5 million per product as well as royalties payable on net sales of products.

Biogen will also receive an option to license next-generation AAV vectors for certain indications from Penn for Biogen’s use outside of the collaboration.

The alliance is separate from a collaboration announced today by Biogen and Regenxbio, under which Regenxbio granted an exclusive worldwide research license to its NAV® AAV8 and AAV9 vectors to Biogen. The companies aim to develop gene therapy candidates for two rare genetic vision disorders in humans.

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