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Establishes clinical pipeline of gene therapy candidates in ophthalmology
March 4, 2019
By: Tim Wright
Editor-in-Chief, Contract Pharma
Biogen has entered into an agreement to acquire Nightstar Therapeutics for $800 million. Nightstar is a clinical-stage gene therapy company based in London, UK, which is focused on adeno-associated virus (AAV) treatments for inherited retinal disorders. “Ophthalmology is an emerging growth area for Biogen, and we are excited about the opportunity to work with the talented employees at Nightstar to advance potentially transformative gene therapy programs for rare retinal diseases,” said Michel Vounatsos, chief executive officer, Biogen. “With this proposed acquisition, we are continuing to bolster our pipeline and further execute on our strategy to develop and expand a multi-franchise neuroscience pipeline across complementary modalities. Nightstar would accelerate our entry into ophthalmology by contributing two mid- to late-stage gene therapy assets, with the potential to create long-term shareholder value.” NST’s lead asset is NSR-REP1 for the treatment of choroideremia (CHM), a rare, degenerative, X-linked inherited retinal disorder, which leads to blindness and has no approved treatments. NST’s second clinical program is NSR-RPGR for the treatment of X-linked retinitis pigmentosa (XLRP), which is also a rare inherited retinal disease primarily affecting males with no approved treatments. NST’s preclinical pipeline includes NSR-ABCA4 for Stargardt disease and potential programs targeting Best vitelliform macular dystrophy (Best disease) and other genetic forms of retinitis pigmentosa. Biogen expects to complete the acquisition by mid-year 2019.
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