Isaralgagene civaparvovec (ST-920)
Fabry Disease
Phase 1/2Active, BLA Rolling Submission
Key Facts
Indication
Fabry Disease
Phase
Phase 1/2
Status
Active, BLA Rolling Submission
Company
About Sangamo Therapeutics
Sangamo Therapeutics is a genomic medicine company with a mission to develop lasting, potentially curative therapies for severe diseases using its foundational zinc finger protein (ZFP) technology. The company has achieved clinical proof-of-concept across multiple modalities, most notably with its lead gene therapy candidate, isaralgagene civaparvovec (ST-920), for Fabry disease, for which it is preparing a Biologics License Application (BLA) submission. Its strategy combines advancing a wholly-owned neurology pipeline with leveraging its platform through strategic collaborations with major pharmaceutical firms like Pfizer, Novartis, Takeda, and Lilly to expand into other therapeutic areas and share development costs.
View full company profileTherapeutic Areas
Other Fabry Disease Drugs
| Drug | Company | Phase |
|---|---|---|
| Corrector Program | Octant Biotech | Hit to Lead |
| GMAC for Fabry Disease | CellGenTech | Preclinical |
| JR-171 (lepunafusp alfa) | JCR Pharmaceuticals | Phase 3 |
| Undisclosed Gene Therapy | uniQure | Preclinical |
| Lucerastat | Idorsia | Phase 3 |
| PRX–115 (pegunigalsidase alfa) | Protalix BioTherapeutics | Submitted |