The alliance, which will use adeno-associated virus (AAV) vectors to target diseases of the central nervous system as well as conditions affecting skeletal muscle and the eye, has a personal dimension. Back in the 1980s, Olivier Danos, now the Biogen executive responsible for steering the company's gene therapy strategy, and James Wilson, director of the Gene Therapy Program at UPenn's Perelman School of Medicine, in Philadelphia, shared a bench as postdocs in the laboratory of Richard Mulligan at the Whitehead Institute at the Massachusetts Institute of Technology; Mulligan is now a member of Biogen's board and chairs its science and technology committee.
The alliance encompasses Wilson's lab, which has had a longstanding focus on gene therapy for a range of inherited diseases and on AAV vector technology, and the laboratory of Jean Bennett, a pioneer of gene therapy for inherited retinal disorders. Bennett is also a scientific co-founder of and advisor to Philadelphia-based Spark Therapeutics, which was spun out from the Children's Hospital of Philadelphia in 2013. “We're getting access to two programs in rare retinal disease,” says Danos, who is senior vice president of cell and gene therapy at Cambridge, Massachusetts–based Biogen. “We expect to enter the clinic within the next two years.” These programs will complement Biogen's existing alliance with Alachua, Florida–based AGTC, which includes AAV-based gene therapy programs in X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa and several other retinal diseases. Many of the programs on Wilson's side of the alliance are early stage and are “payload agnostic” at this point, Wilson says. Danos highlights one, in spinal muscular atrophy, given Biogen's existing alliance in the indication with Ionis Pharmaceuticals, of Carlsbad, California. Their anti-sense drug nusinersen, which targets SMN2, is currently in phase 3 trials. “For us it's strategically very important that we follow on with gene therapy.”
This is a preview of subscription content, access via your institution