Astellas dives into gene therapy with a Harvard expert in tow

Harvard's Constance Cepko

Japan's Astellas Pharma is the latest big drugmaker allured by the promise of gene therapy, mounting an R&D effort that could lead to a new treatment for an inherited eye disease.

The company has signed a deal with Harvard Medical School investigator and gene therapy expert Constance Cepko to collaborate on a gene therapy approach to retinitis pigmentosa (RP), a degenerative eye disease that can damage vision and lead to blindness. The plan is to investigate whether harmless adeno-associated virus (AAV) vectors can be used to deliver corrective genes to RP patients, Astellas said, in the process mapping out the mutations at the heart of the disease.

Under the three-year agreement, Astellas has agreed to pay for discovery, development and, if things go according to plan, marketing, striking a commercialization agreement with Harvard with the potential to expand into other avenues of gene therapy.

"We are hopeful that some of our candidates will preserve the color and daylight vision of RP patients, regardless of the disease gene in their family," Cepko said in a statement. "These treatments may also be effective in people that have other types of ocular disease, such as age-related macular degeneration or glaucoma."

Astellas' effort is part of its Innovation Management program, a 2013 initiative designed to reshape its R&D machine by gutting some internal operations and putting a new emphasis on partnering. As part of the move, the company shut down its OSI Pharmaceuticals and Perseid Therapeutics subsidiaries, all the while scaling back its U.S.-headquartered research institute to focus solely on CNS treatments.

The Harvard deal makes for Astellas' first announced foray into gene therapy, a space in midst of a resurgence of popularity. Serious safety issues and deliverability woes long hampered R&D in the field, but, after years of work, a new generation of academics and investigators believes it has hammered out the right viral vectors to safely and predictably get corrective genes to their target tissues, spurring renewed hope for widespread clinical success.

And now deep-pocketed drugmakers are getting involved.

Last month, Biogen Idec ($BIIB) made a splash in the field by unveiling plans to build an internal gene therapy division, one that will stand alongside its existing efforts in antisense therapies, biologics and traditional small molecules. Before that, Celgene ($CELG) teamed up with bluebird bio ($BLUE) on a gene therapy approach to oncology, and Bayer signed a $252 million deal with Fierce 15 honoree Dimension Therapeutics to get its hands on a hemophilia A treatment. GlaxoSmithKline ($GSK), Baxter ($BAX) and others are working on in-house treatments in the field, as well.

- read the statement

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