Falling star offers no hope for rare eye disease patients

15 June 2021

Biogen’s (Nasdaq: BIIB) near-billion dollar bet on Nightstar Therapeutics looks set to lose spectacularly, following the failure of the Phase III STAR study of lead candidate timrepigene emparvovec.

The news comes just a month after the US biotech major announced negative top-line results from the Phase II/III XIRIUS study of Nightstar’s number two runner, cotoretigene toliparvovec.

Both novel gene therapies are under investigation for the treatment of rare retinal diseases causing blindness, the former as an option for choroideremia and the latter for X-linked retinitis pigmentosa (XLRP).

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