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Biogen gene therapy misses goal in eye disease study

May 14, 2021

Biogen’s acquisition of Nightstar signaled the biotech company’s commitment to gene therapy research, an investment it’s expanded upon with subsequent deals like a January collaboration with Germany’s ViGeneron and another earlier this week with Capsigen.

The Nightstar deal bought Biogen two gene therapies in advanced trials, including the XLRP treatment that’s now fallen short in Phase 2/3 testing.

XLRP, short for X-linked retinitis pigmentosa, primarily affects males and is associated with progressive vision loss caused by the degradation of light-sensing cells in the retina. Biogen’s treatment is designed to deliver into the eye, via a subretinal injection, a functional gene encoding for a key protein that’s missing in XLRP patients.

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