Sangamo Therapeutics Inc., a genomic medicine company, has announced that its investigational gene therapy, isaralgagene civaparvovec, has received significant regulatory designations from various health authorities. The U.S. Food and Drug Administration (FDA) has granted the therapy Orphan Drug, Fast Track, and RMAT designations, while the European Medicines Agency has provided Orphan Medicinal Product designation and PRIME eligibility. Additionally, the U.K. Medicines and Healthcare products Regulatory Agency has included it in the Innovative Licensing and Access Pathway. These designations are expected to expedite the development and potential approval of the therapy, which aims to treat Fabry disease. Sangamo is preparing a Biologics License Application $(BLA.AU)$ submission for 2026, following positive results from the Phase 1/2 STAAR study.
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