Guess the Therapy Answer March 18, 2026

Sangamo is currently submitting a rolling BLA to the FDA for which investigational gene therapy in development for Fabry disease?

Answer: isaralgagene civaparvove (isa-vec, also known as ST-920).

See below for further reading on isa-vec:

Sangamo Starts Putting BLA for Fabry Disease Gene Therapy Isa-Vec in Front of FDA

December 19, 2025 — Sangamo Therapeutics has begun submission of a rolling biologics license application (BLA) application to the FDA for investigational Fabry disease gene therapy isaralgagene civaparvove (isa-vec, ST-920).

The BLA is supported by data from the phase 1/2 STAAR clinical trial (NCT04046224). Across all patients who were treated in STAAR, a positive mean annualized estimated glomerular filtration rate (eGFR) slop was observed at 52 weeks posttreatment. Notably, in an October 2025 meeting with Sangamo, the FDA confirmed its agreement that eGFR slope can be used as an end point for the support of an accelerated approval pathway for isa-vec. Shortly after this meeting, in November 2025, Sangamo received a green light from the FDA to begon submitting a rolling BLA for the therapy.

Sangamo Adds Preclinical and Clinical Modules to Rolling BLA for Fabry Disease Gene Therapy Isa-Vec

March 11, 2026 — Sangamo Therapeutics announced that it has submitted preclinical and clinical modules to the FDA for its rolling BLA for investigational Fabry disease gene therapy isa-vec. The company originally initiated submission of the rolling BLA in December 2025. Sangamo has previously noted that it expects the rolling BLA submission, which is taking place under the accelerated approval pathway, will be finished in the second quarter of 2026.