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The acquisition will boost Biogen’s gene-therapy pipeline with the addition of two mid- to late-stage clinical assets and preclinical programs.
On June 7, 2019, Biogen announced that it completed its acquisition of Nightstar Therapeutics (NST), a clinical-stage gene therapy company focused on adeno-associated virus (AAV) treatments for inherited retinal disorders, for approximately $800 million. The acquisition adds two mid- to late-stage clinical assets, as well as preclinical programs, in ophthalmology to Biogen’s pipeline.
NST’s lead asset is NSR-REP1, a gene-therapy candidate for treating choroideremia (CHM), a rare, degenerative, X-linked inherited retinal disorder, which leads to blindness and has no approved treatments. CHM primarily affects males and is caused by loss of function in the CHM gene which encodes the Rab escort protein-1 (REP-1).
NST has a second clinical program, NSR-RPGR, for treating X-linked retinitis pigmentosa (XLRP), which is also a rare inherited retinal disease primarily affecting males with no approved treatments. XLRP is characterized by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene leading to a lack of active protein transport in photoreceptors, the company reports. The abnormality leads to loss of photoreceptor cells that results in retinal dysfunction by adolescence and early adulthood and progresses to legal blindness when patients reach their 40s.
“Today marks a significant achievement for Biogen,” said Michel Vounatsos, Biogen’s CEO, in a company press release. “The acquisition of Nightstar further bolsters our pipeline and is an important step forward toward our goal of a multi-franchise portfolio across complementary modalities. We look forward to working now as one Biogen team with the goal of bringing breakthrough therapies to patients to slow or halt blindness across a range of inherited retinal diseases.”