RICHMOND, Calif. — Sangamo Therapeutics, Inc., (Nasdaq: SGMO), a genomic medicine company, today announced updated preliminary data from the Phase 1/2 STAAR clinical study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for the treatment of Fabry disease. In the largest known clinical gene therapy program...
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RICHMOND, Calif. — Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, has initiated a rolling submission of a BLA to the FDA seeking accelerated approval of isaralgagene civaparvovec, or ST-920, a wholly owned investigational gene therapy for the treatment of adults with Fabry disease. Rolling submission allows for completed...
RICHMOND, Calif. — Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced detailed data from the registrational Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned investigational gene therapy for the treatment of adults with Fabry disease. These data were presented at the International Congress...
Saniona (OMX: SANION), a clinical-stage biopharmaceutical company focused on rare diseases, today announced it received approximately USD $2.9 million (SEK 24.2 million) in an upfront payment resulting from the now completed acquisition of Cadent Therapeutics by a third party. Saniona holds an ownership stake of approximately 3% in Cadent Therapeutics...
Paris, April 23, 2024. Positive results from the LUNA 3 phase 3 study demonstrated that rilzabrutinib 400 mg twice daily orally achieved the primary endpoint of durable platelet response in adult patients with persistent or chronic immune thrombocytopenia (ITP). The safety profile of rilzabrutinib was consistent with that reported in...
Paris and Tarrytown, N.Y. February 23, 2024. The U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Dupixent® (dupilumab) in a sixth potential indication as an add-on maintenance treatment in certain adult patients with uncontrolled chronic obstructive pulmonary disease (COPD). The...
Paris, France – The European Medicines Agency (EMA) has granted orphan designation to efdoralprin alfa (SAR447537, formerly known as INBRX-101), an investigational recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein, for the potential treatment of alpha-1 antitrypsin deficiency (AATD) related emphysema, a rare respiratory condition with great unmet medical need. The EMA...
Paris, France – The New England Journal of Medicine published results from a positive Phase 2 clinical trial demonstrating frexalimab significantly slowed disease activity in people with relapsing multiple sclerosis (MS), corresponding to 89% and 79% reduction in new gadolinium-enhancing (GdE) T1 brain lesions at Week 12 in the high-...
Paris, September 23, 2025. The U.S. Food and Drug Administration (FDA) has granted fast track designation to SAR446268, Sanofi’s one-time AAV gene therapy for the treatment of non-congenital (juvenile and adult onset) DM1 myotonic dystrophy type 1 (DM1). This designation process aims to facilitate the development and expedite the review...
PARIS, France – The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SAR446523, an IgG1-based Antibody-Dependent Cellular Cytotoxicity-enhanced (ADCC) monoclonal antibody (mAb) targeting G-protein coupled receptor family C group 5 member D (GPRC5D) for the potential treatment of patients with relapsed or refractory multiple myeloma (R/R...