Seeking out new treatments that have the potential to transform care for patients with rare diseases is part of the pioneering spirit of Biogen. Last year, Biogen expanded its focus to help patients with rare diseases of the retina. The acquisition of Nightstar Therapeutics plc brought with it two mid- to late-stage gene therapies for the potential treatment of rare retinal diseases, BIIB111 (timrepigene emparvovec) and BIIB112 (RPGR gene therapy). These assets expand Biogen’s focus in ophthalmology, a core growth area, as well as strengthen Biogen’s foothold in gene therapy.
“Our focus is simple – to improve or preserve vision in patients with rare retinal diseases, empower patients to live independently and maintain hope for their future,” said Samantha Vieira, Executive Director, Product Development and Commercial Lead at Biogen, and former Nightstar Therapeutics researcher. “Working now as part of the Biogen team, we have the combined expertise, resources and passion to deliver what we hope will be potentially transformative treatments to patients globally.”
BIIB111 is in Phase 3 development for the potential treatment of choroideremia (CHM), which is a rare, degenerative, X-linked inherited retinal disorder that leads to blindness and which currently has no approved treatments. In November 2019 we announced that all patients had been enrolled in the pivotal Phase 3 STAR study which is evaluating BIIB111 as a potential first-in-class treatment for CHM.
“Imagine being told that you will go totally blind by a certain age – that your eyesight will deteriorate until it’s gone and there are no treatments available,” said Marina Feschenko, Director of Analytical Development for Gene Therapy at Biogen. “As a researcher, it’s incredible to think we may be able to help these patients, help someone from going blind. I can’t imagine anything more rewarding.”
In early 2020 Biogen announced expanding into genomic medicines further, with a new collaboration with Sangamo Therapeutics to collaborate on gene regulation therapies for several neurological diseases. This latest collaboration bolsters Biogen’s position as an emerging leader in gene therapy in neuroscience. Biogen’s multi-franchise neuroscience pipeline stretches across complementary modalities, which now includes biologics, small molecules and antisense oligonucleotides along with potential gene therapies.
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