RICHMOND, Calif. – Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced important U.S. and European regulatory updates for isaralgagene civaparvovec, or ST-920, its wholly owned gene therapy product candidate for the treatment of Fabry disease. The FDA has agreed in a Type D meeting that data from a...
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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...
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 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-...
Barcelona, Spain – Researchers from the Neuromuscular Diseases Group and the Dementia Neurobiology Group of the Sant Pau Research Institute (IR Sant Pau) and the Memory Unit of the Sant Pau Hospital, led by neurologist Dr. Ricard Rojas-García, have identified a new mutation in the ARPP21 gene that could be...
Santa Susana High School graduate Sean Shrager doesn’t believe in giving up. Though he was diagnosed with Duchenne muscular dystrophy (DMD) when he was 3 years old, Sean has never let his disease become an excuse for not attaining his goals. In fact, it has only motivated him to work...
Pratteln, Switzerland – Santhera Pharmaceuticals (SIX: SANN) and ReveraGen Biopharma announce new clinical data of 2.5-year treatment outcome with vamorolone in patients with Duchenne muscular dystrophy (DMD). These Phase 2a long-term treatment data demonstrate a maintenance of treatment effect, equivalent to a delay of about two years in decline for...
Pratteln, Switzerland, and Rockville, MD, USA – Santhera Pharmaceuticals (SIX: SANN) and ReveraGen BioPharma, Inc (US: private) announce positive key results from the VISION-DMD study, demonstrating robust efficacy across multiple efficacy endpoints and favorable safety and tolerability of vamorolone in the treatment of patients with DMD. VISION-DMD is a pivotal...