L’Hospitalet de Llobregat, Spain – Once they enter the body, drugs, apart from carrying out their therapeutic function, are biochemically transformed by the action of the metabolic machinery, a process that facilitates their expulsion. This biotransformation results in a gradual disappearance of the drug, which is converted into its metabolites....
treatment News
Paris, France – SparingVision, a clinical-stage genomic medicine company transforming the treatment of retinal disease, today announces a key advancement in its Phase I/II clinical trial (PRODYGY, NCT05748873) for SPVN06, the Company’s lead gene-agnostic investigational gene therapy for the treatment of retinitis pigmentosa (RP). The trial has now progressed to...
HENDERSON, Nev. – Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, announced the presentation of safety and efficacy results from Cohort 4 of the ZENITH20 clinical trial. This data is from 48 first-line patients with non-small cell lung cancer (NSCLC) with HER2 exon...
Urbana, IL – The Speech Accessibility Project has expanded its recruitment and is inviting U.S. and Puerto Rican adults living with amyotrophic lateral sclerosis to participate. Those interested in participating can sign up online. Funded by Big Tech companies Amazon, Apple, Google, Meta, and Microsoft, the University of Illinois Urbana-Champaign aims...
STAMFORD, Conn. — SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced today that the U.S. Food and Drug Administration (FDA) has approved OGSIVEO™ (nirogacestat), an oral gamma secretase inhibitor, for the treatment of adult patients with progressing desmoid tumors who require...
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...
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...