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...
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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...
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 gene therapy product candidate for the treatment of Fabry disease. These data will be presented via four platform presentations...
CLEVELAND, Ohio –Sangrail Biologics, a clinical-stage gene therapy company focused on restoring life’s blueprint for children with rare genetic diseases, announced the launch of the company today and highlighted the company’s lead clinical asset, SNG-101 (f.k.a. ABO-101), a potential first-in-class AAV gene therapy designed to treat the underlying cause of...
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, France and Tarrytown, NY – The Ministry of Health, Labour and Welfare in Japan has granted marketing and manufacturing authorization for Dupixent (dupilumab) for the treatment of adults with moderate-to-severe bullous pemphigoid (BP). The approval in Japan is based on data from the pivotal LIBERTY-BP-ADEPT phase 2/3 study (clinical...
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, April 24, 2026. The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the approval of Cenrifki (tolebrutinib) in the EU for the treatment of secondary progressive multiple sclerosis (SPMS) without relapses in the last two years. A final decision is...
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...