SEONGNAM, South Korea — Rznomics Inc., a South Korea based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, has received an Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for RZ-001, in the treatment of patients with Hepatocellular carcinoma (HCC). The ODD granted to RZ-001...
treatment News
SEOUL, South Korea — S.BIOMEDICS Co., Ltd. (KOSDAQ: 304360) announced that it has successfully completed the brain transplant of TED-A9 (hESC-derived dopaminergic progenitors) for Phase 1/2a study for treating Parkinson’s disease. The clinical trial was conducted on 12 participants who have been diagnosed with Parkinson’s disease for more than 5...
HONG KONG, SHANGHAI and FLORHAM PARK, N.J. — HUTCHMED (China) Limited (“HUTCHMED”) (Nasdaq/AIM:HCM; HKEX:13) today highlights that results from the SACHI Phase III trial were published in The Lancet. SACHI is a Phase III study of the savolitinib (ORPATHYS®) and osimertinib (TAGRISSO®) combination for the treatment of patients with locally advanced or...
CAMBRIDGE, Mass.– Sage Therapeutics, Inc. (Nasdaq: SAGE), a biopharmaceutical company leading the way to create a world with better brain health, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to SAGE-718 for the treatment of Huntington’s disease (HD). SAGE-718 is in development as a...
SHANGHAI, China — Gannex Pharma Company Ltd, a wholly-owned company of Ascletis Pharma Inc., announced that its strategic partner, Sagimet Biosciences Inc., today reported positive topline results from Sagimet’s FASCINATE-2 Phase 2b clinical trial of denifanstat versus placebo in biopsy-confirmed non-alcoholic steatohepatitis (NASH) patients with stage 2 or stage 3...
SOMERVILLE, Mass. — Sail Biomedicines, Inc., a Flagship Pioneering company and leader in RNA-based programmable medicines, today provided an update on its research funded by the Cystic Fibrosis Foundation (CF Foundation), with preclinical data that suggest that Sail’s Endless RNA™ (eRNA™) may offer the potential to treat the 10%-15% of...
LEXINGTON, Mass. — SalioGen Therapeutics, a biotechnology company developing next-generation genetic medicines based on its novel Gene Coding™ technology, today announced the nomination of a development candidate, SGT-1001, for the treatment of Stargardt disease, a rare and progressive inherited retinal disease. SGT-1001 is being developed as a one-time, non-viral therapy to...
Basel, Switzerland – Sandoz, the global leader in generic and biosimilar medicines, today announces the launch of Tyruko (natalizumab) in Germany from February 1. Developed by Polpharma Biologics, Tyruko is the first and only biosimilar to treat RRMS. Tyruko is indicated as a single disease-modifying therapy (DMT) in adults with...
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...