If approved, mirdametinib is expected to be the first and only therapy in the European Union with marketing authorization for both adults and children with NF1-PN Decision from European Commission expected in the third quarter of 2025 STAMFORD, Conn. — SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused...
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)....
SOUTH SAN FRANCISCO, Calif. – Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to tralesinidase alfa enzyme replacement...
LONDON, UK — Spur Therapeutics today announced updated clinical data for avigbagene parvec (FLT201), its Gaucher disease gene therapy candidate, demonstrating clinical benefit and favorable safety maintained for more than two years to date following administration of a single low dose. These data are being presented at the European Society of...
STEVENAGE, UK – FLT201, an experimental gene therapy developed by Spur Therapeutics, has shown sustained clinical benefits for up to 21 months in people with Gaucher disease type 1, according to new data from a clinical trial. The findings, presented at the 28th Annual Meeting of the American Society of...
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...