Biogen Aligns with Two Super Stars and Bags a Deal with REGENXBIO in Gene Therapy Push
May 16, 2016
By Alex Keown, BioSpace.com Breaking News Staff
CAMBRIDGE, Mass. – Biogen has made several bold steps to further its work in gene therapy. This morning the company announced a collaboration with two gene therapy experts from the University of Pennsylvania, as well as a gene therapy technology licensing deal with Maryland-based RegenxBio.
Biogen and RegenxBio struck a deal for an undisclosed amount of money in both upfront and milestone payments, which will allow the Massachusetts company to license that company’s proprietary NAV Technology Platform for the treatment of two rare genetic vision disorders. The NAV Technology Platform is an adeno-associated virus (AAV) gene delivery platform consisting of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. The deal between both companies has investors pleased, with shares of both companies up. Biogen is trading at $264.32 per share this morning, while RegenxBio is up more than 3 percent to $11.70 per share this morning.
Biogen’s Olivier Danos, vice president of Cell and Gene Therapy at Biogen, said the company continues to look for opportunities to advance gene therapies to address certain illnesses. Danos said the work with RegenxBio will allow Biogen to expand its pipeline of treatments for eye disease.
Kenneth T. Mills, president and chief executive officer of RegenxBio, called the deal an important step in advancing NAV-based gene therapies to people suffering from rare genetic vision disorders.
Not only has Biogen struck the deal with RegenxBio, the Cambridge company announced a collaboration with two leading gene therapy experts from the University of Pennsylvania, James Wilson and Jean Bennett. The collaboration between the university and Biogen has multiple objectives, but will primarily focus on the development of therapeutic approaches that target the eye, skeletal muscle and the central nervous system (CNS). Another aspect of the alliance will focus on validating next-generation gene transfer technology using AAV gene delivery vectors. The collaboration will also explore the expanded use of genome editing technology&mdashthe insertion, deletion or replacement of DNA in the genome of an organism&mdashas a potential therapeutic platform, Biogen said in a statement this morning. Danos touted the collaborative agreement with the two gene therapy experts, saying it will improve the way Biogen approaches potential gene therapy treatments.
“Joining forces with gene therapy pioneers, James Wilson and Jean Bennett, further enables Biogen to approach the technology from a powerful perspective. 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,” Danos said in a statement.
The agreement between the biotech leader and the university, valued at up to $2 billion, will allow Penn to harness its gene therapy resources and expertise to develop therapeutic candidates and aid in the development of new manufacturing approaches needed to support commercialization of gene therapy products. In turn, Biogen will combine its therapeutic area identification prowess and drug development capabilities to advance the collaboration programs into the clinic and toward approval, the company said.
Under terms of the deal, 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. 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.