Well, another gene therapy bites the dust, leading Evaluate to...

Well, another gene therapy bites the dust, leading Evaluate to observe “It will not go unnoticed that eye diseases are proving tough for gene therapy."

Biogen has announced the failure of its Phase 3 study of timrepigene (BIIB111) in the rare inherited retinal disease of choroideremia. Both primary endpoint and key secondary endpoints were missed.

Timrepigene is an AAV-2 mediated gene therapy,administered as a single, sub-retinal injection, and was one of two inherited retinal disease R & D gene therapy assets that came to Biogen through its  A$1.14bn acquisition of Nightstar Therapeutics in 2019.

Biogen’s latest miss adds to a growing list of setbacks in the space.

Nightstar’s other asset, BIIB112 for X-linked Retinitis Pigmentosa, failed in a Phase 1/2 trial last month.

Toxicity has also been a problem for gene therapies in eye disease, as seen with Adverum’s ADVM-022 and Regenxbio’s RGX-314.