PCI facilitated a sizable multi-year collaborative alliance between Biogen and Penn Medicine faculty focused on gene therapy and gene editing. The collaboration has multiple main objectives:
- developing therapeutic approaches that target the eye, skeletal muscle and the central nervous system (CNS);
- validating next-generation gene transfer technology using adeno-associated virus (AAV) gene delivery vectors; and
- exploring the expanded use of genome editing technology as a potential therapeutic platform.
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 are the principle investigators.
Under the terms of the agreement, Penn may receive up to $2 billion in research funding, options and milestone payments. Biogen will make an upfront payment to Penn of $20 million with an additional $62.5 million committed to fund R&D costs extending over the next three to five years in seven distinct preclinical research and development programs conducted by the Wilson and Bennett laboratories. 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 also receives an option to license next generation AAV vectors for certain indications from Penn, for Biogen’s use outside of the collaboration.
FierceBiotech – Biogen enlists gene therapy stars, adds vectors in $2B deal