Sangamo Therapeutics Advances FDA Submission for Fabry Disease Gene Therapy

Key Facts

Sangamo Therapeutics has officially commenced the rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its gene therapy candidate, ST-920. The therapy, also known as isaralgagene civaparvovec, is designed as a potential one-time treatment for patients suffering from Fabry disease. A critical milestone was reached as the FDA agreed to use the estimated glomerular filtration rate (eGFR) slope at 52 weeks as a primary endpoint to support an accelerated approval pathway. Clinical data from the ongoing STAAR study has demonstrated positive results, showing sustained kidney function improvements across all treated patient groups. This regulatory progress signals a potential shift in the treatment paradigm for Fabry disease, moving away from chronic management toward genetic intervention. Investors are closely monitoring the submission process, as successful approval could significantly enhance Sangamo's market position in the genomic medicine sector.