Biogen commits to gene therapy

Soon to split off its hemophilia drug business, Biogen is making gene therapy a big part of its new focus on neurological and ophthalmological diseases.

The company will spend up to $2 billion to work on gene transfer and gene editing methods with University of Pennsylvania professors James Wilson and Jean Bennett. Biogen also has licensed adeno-associated virus (AAV) gene delivery technology from Regenxbio, a seven-year-old spin-off from Wilson’s lab.

Biogen will pay Penn $20 million up-front and commit $62.5 million over the next three to five years to fund seven preclinical R&D programs. Also in the potential payout are up to $137.5 million in milestone payments per resulting product.

In 2015, Biogen agreed to pay the Florida biotech firm AGTC $124 million up front and up to $1.1 billion more in milestone payments for at least two gene-based ophthalmic therapies. And Biogen was among investors who recently put $43 million into Solid Biosciences’ Duchenne muscular dystrophy gene therapy program.

After decades of work and several setbacks, only two gene therapies have been approved, both in Europe. Wilson, who directs Penn’s gene therapy program and orphan disease center, has been developing the use of AAV vectors to overcome past problems with delivery. Meanwhile Bennett, a founder of Spark Therapeutics, has advanced ophthalmic gene therapies.

Moving beyond traditional approaches, Biogen and firms such as Sangamo Biosciences and Bluebird Bio are looking to combine gene-therapy methods with new gene-editing techniques. Editas Medicine has one of the most advanced programs combining CRISPR gene-editing and AAV vectors. It expects to begin clinical testing of a gene repair therapy for a rare eye disease in 2017.