Voyager updates on SOD1 ALS gene therapy program

12 February 2025

US biotech Voyager Therapeutics (Nasdaq: VYGR) saw its shares close down 21% at $4.20 on Tuesday, after it revealed its decision to assess alternate payloads related to its gene therapy program for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS).

Voyager noted that emerging three-month non-human primate (NHP) data suggest that an alternate payload would be necessary to achieve the desired product profile. No changes are planned to the novel capsid component. The same capsid in the VY1706 (tau silencing) gene therapy program achieved desired activity levels and was well-tolerated in three-month NHP studies.

As a result, Voyager no longer anticipates filing an investigational new drug (IND) application for VY9323 in mid-2025. Voyager’s cash runway is now expected to extend into mid-2027; this does not include any potential milestone payments from existing partnerships, it noted.

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