information: Clinical trial authorisation
Announcement: clinical trial authorisation
Company: Sangamo Therapeutics (USA - CA)
- gene therapy. ST-920 comprises an AAV2/6 vector carrying an Alpha Galactosidase (AGLA) gene construct driven by a proprietary liver-specific promoter. ST-920 gene therapy is designed to enable a patient's liver to produce a long-lasting and continuous supply of the ?-Gal A enzyme.
Disease: Fabry disease
area: Rare diseases - Genetic diseases
- This is the first in human treatment with ST-920, a recombinant AAV2/6 vector encoding the cDNA for human a-Gal A. The purpose of this study is to evaluate the safety and tolerability of ascending doses of ST-920. ST-920 aims to provide stable, long-term production of ?-Gal A at therapeutic levels in subjects with Fabry disease. The constant production of ?-Gal A in humans should, importantly, enable reduction and potentially clearance of Fabry disease substrates Gb3 and lyso-Gb3. On Day 1, patients will be infused intravenously with a single dose of ST-920 and followed for a period of 52 weeks. (NCT04046224)
- • On February 20, 2019, Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company, announced that the FDA has accepted the Investigational New Drug (IND) application for ST-920, a gene therapy candidate being evaluated for the treatment of adults with Fabry disease. Current standard of care for this rare, progressive condition involves regular lifelong infusions of enzyme replacement therapy (ERT). The active IND enables Sangamo to initiate a Phase 1/2 clinical trial designed to assess the safety, tolerability and efficacy of ST-920 in adults with Fabry disease. Sangamo expects to open several clinical sites later this year.