- In its latest investment into the field of gene therapy, Biogen has licensed technology that it believes could help create new treatments for central nervous system and neuromuscular disorders.
- The deal with Capsigen, a Washington-based biotech, has Biogen paying $15 million at the start and potentially up to $1.3 billion later on, provided certain research, development and sales milestones are hit. Capsigen is also eligible for royalties on future net sales on any resulting products that use its technology.
- Capsigen specializes in developing adeno-associated virus vectors, which are used like microscopic shuttles to deliver genetic medicine into human cells. The deal with Biogen tasks Capsigen with creating new capsids — the outer coating which protects the genetic payload — that are customized to target specific diseases.
Biogen's big move into gene therapy came just over two years ago with the $800 million acquisition of Nightstar Therapeutics, a developer of eye medicines.
Since then, the company has inked an agreement with Sangamo Therapeutics to explore gene editing as a way to treat neurological diseases, pushed further into gene therapies for the eye through a deal with Germany's ViGeneron, and just recently announced plans to spend $200 million on a gene therapy manufacturing facility in North Carolina.
These investments have come both as Biogen's peers pour money into gene therapy, and as the company faces investor pressure to diversify its business.
Gene therapy research has made a resurgence, in large part, because the technology and how it interacts with the body are now better understood. The Food and Drug Administration has approved a handful of cell and gene therapies over the last few years, and expects to be reviewing and approving between 10 and 20 annually by 2025.
Yet, there are still major challenges in this cutting-edge field, with one of the biggest being delivery. To be effective, gene therapies must achieve the complicated task of carrying their payloads to the appropriate cells without getting destroyed by the body's defense mechanisms. That task can be especially difficult for therapies going after highly protected areas, such as the brain.
Companies that specialize in gene therapy delivery have therefore been in high demand. Over the last year, for example, a startup called Dyno Therapeutics has caught the attention of heavyweight gene therapy developers like Novartis, Roche and Sarepta Therapeutics.
Dyno's capsid technology is meant to create more targeted gene therapies that are less likely to trigger an immune response. Just last week, the company said it had raised an additional $100 million in fresh funding from tech investor Andreessen Horowitz and several other venture firms.
Also recently, the gene editing startup Beam Therapeutics agreed to buy Guide Therapeutics, which makes tools to deliver genetic medicines into cells, in a deal valued at $120 million.
Capsigen now adds to this flurry of activity with its Biogen collaboration. Per deal terms, Biogen will hold an exclusive license to Capsigen's technology for an undisclosed number of CNS and neuromuscular disease targets.
"One of our priorities for technology innovation is the discovery of AAV capsids with improved delivery profiles," Al Sandrock, Biogen's head of research and development, said in a May 11 statement. "We are investing for the long-term by building platform capabilities and advanced manufacturing technologies with the goal of accelerating our efforts in gene therapy."