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...
RICHMOND, Calif. — Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the U.S. Food and Drug Administration (FDA) has accepted Sangamo’s request for a rolling submission and review of the Biologics License Application (BLA) for isaralgagene civaparvovec, or ST-920, a wholly owned investigational gene therapy for...
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...
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...