- Biogen said on Monday it will partner with two gene therapy experts from the University of Pennsylvania in a collaboration deal to develop new treatments targeting the eye, skeletal muscle, and central nervous system.
- Biogen will pay Penn $20 million upfront as well as commit $62.5 million to funding R&D over the next three to five years. Including milestone payments, the deal could be worth as much as $2 billion.
- The collaboration's main focus will be on advancing gene therapy candidates using existing adeno-associated virus (AAV) vectors, but will also investigate therapeutic application of genome editing.
Biogen will be partnering with the laboratories of James Wilson and Jean Bennett, two gene-therapy experts at the Perelman School of Medicine. Both have been involved in gene-therapy work for decades and Bennet was one of the first to deliver a gene therapy into the eye, as Xconomy notes.
"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," said Olivier Danos, senior vice president of Cell and Gene Therapy at Biogen.
Despite the promise of gene therapies to potentially cure a wide range of rare diseases, none are currently approved for use in the U.S. Two have been okayed in Europe: UniQure's Glybera and, just recently, GlaxoSmithKline's Strimvelis.
But cost concerns and manufacturing challenges loom large for the sector. Glybera, for example, is priced at around $1 million per patient, although it has only be used on one patient, according to MIT Technology Review.
The collaboration with Penn will focus on seven preclinical research programs, each eligible for between $77.5 million to $137.5 million in milestone payments if any come to fruition. Biogen also gains an option to license any new AAV vectors developed by either laboratory, although that work falls outside the collaboration.