Stamford, Conn. — SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced that data from the Phase 3 DeFi trial of nirogacestat, an investigational oral gamma secretase inhibitor, in adult patients with progressing desmoid tumors, were...
treatment News
SOUTH SAN FRANCISCO, Calif. — Spruce Biosciences, Inc., a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet medical need, today announced completion of enrollment in its CAHmelia-204 clinical trial of tildacerfont for the treatment of adult classic congenital adrenal hyperplasia (CAH)....
ALISO VIEJO, Calif. — SpyGlass Pharma™, a privately-held ophthalmic biotechnology company, announced today 1-year follow up data from a first-in-human study of 23 patients with glaucoma or ocular hypertension implanted with SpyGlass’ Intraocular Lens (IOL) based Drug Delivery Platform with bimatoprost at the time of cataract surgery. These data will...
MEMPHIS, Tenn. – Scientists at St. Jude Children’s Research Hospital comprehensively identified genes directly regulated by a protein associated with high-risk pediatric leukemias. High-risk leukemias, particularly MLL-rearranged (MLL-r) leukemia, often overexpress the homeodomain transcription factor HOXA9 protein, which cannot currently be targeted with drugs. This study provides a foundation for...
Guangzhou, China – Researchers from the South China University of Technology identified a novel approach to effectively silence the expression of transthyretin (TTR), with potential implications for innovative therapeutic approaches in TTR amyloidosis, including transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). The proposed strategy involves stabilizing the G-quadruplex structure—a fundamental genomic structural feature—and...
Stanford, CA – Cell therapies for cancer can be potentially enhanced using a CRISPR RNA-editing platform, according to a new study published Feb. 21 in Cell. The new platform, Multiplexed Effector Guide Arrays, or MEGA, can modify the RNA of cells, which allowed Stanford University researchers to regulate immune cell metabolism in...
PARAMUS, N.J. — Starton Therapeutics Inc., a clinical-stage biotechnology company focused on transforming standard-of-care therapies with proprietary continuous delivery technology, announced Regional Medical Oncology Center (RMOC) in Wilson, NC as the second site activated in the STAR-LLD Phase 1b clinical trial. The ongoing study evaluates the safety, pharmacokinetics, and efficacy...
NEEDHAM, Mass.— Stealth Biotherapeutics, a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing its New Drug Application (NDA) for elamipretide for the treatment of Barth syndrome....
Hoboken, NJ – Cell therapy represents a potential regenerative treatment for osteoarthritis. A recent analysis of all relevant published studies indicates that stem cell transplantation from different sources is effective for treating knee osteoarthritis, the most prevalent chronic joint disease. The review and meta-analysis, which is published in the Journal...
Vancouver, Canada – An innovative stem cell-based treatment for Type 1 diabetes can meaningfully regulate blood glucose levels and reduce dependence on daily insulin injections, according to new clinical trial results from the University of British Columbia (UBC) and Vancouver Coastal Health (VCH). “This is a significant step toward a...