PENNSVILLE TWP. — Sandy McWilliams, a former Pennsville resident, will walk 700 miles this summer from her home in Kentucky to her mother’s house in Pennsville to raise awareness on progressive supranuclear palsy, a rare disease that her mother is fighting. PSP is a rare, fatal brain disease that McWilliams’...
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BRISBANE, Calif. – Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) released details supporting the Orphan Designation of BIVV003, an investigational ex vivo gene-edited cell therapy product candidate currently being evaluated for the treatment of sickle...
BRISBANE, Calif. – Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) released details supporting the Orphan Designation of BIVV003, an investigational ex vivo gene-edited cell therapy product candidate currently being evaluated for the treatment of sickle...
BRISBANE, Calif. – Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced preliminary results 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. As of the September 17, 2021 cutoff date, results...
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...
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-...