Gene Therapy
New preclinical findings demonstrate elunetirom’s direct effects on key gene programs that drive mitochondrial biogenesis, ATP production, and neuroplasticity in neuronal cultures Topline data anticipated from Phase 2 AMPLIFY-BD study in adjunctive bipolar depression in 2Q26 and Phase 2 AMPLIFY study in adjunctive major depressive disorder in 3Q26 Excerpt from the Press Release: SAN DIEGO–(BUSINESS…
Excerpt from the Press Release: WALTHAM, Mass.–(BUSINESS WIRE)–Affinia Therapeutics (“Affinia”), an innovative clinical-stage gene therapy company with a pipeline of first-in-class and/or best-in-class adeno-associated virus (AAV) gene therapies initially for devastating cardiovascular diseases, today announced the presentation of new preclinical and translational data on the company’s lead program AFTX-201, the design of the Phase 1/2…
U.S. pivotal trial to assess efficacy and safety of AURN001 compared to placebo Potential first-in-class cell therapy to regenerate corneal clarity and restore vision with a single procedure Excerpt from the Press Release: SEATTLE & CAMBRIDGE, Mass. & TOKYO–(BUSINESS WIRE)–Aurion Biotech, Inc. (“Aurion Biotech”), a commercial-stage regenerative cell therapy company dedicated to restoring vision and…
AFTX-201 uses Affinia’s proprietary capsid engineered for efficient cardiac transduction at doses that are 5-10-fold lower than doses of gene therapies using conventional capsids Company on track to initiate the UPBEAT© clinical trial to investigate safety, pharmacodynamics, and preliminary efficacy of AFTX-201 in BAG3 DCM in the first half of 2026 Excerpt from the Press Release:…
Largest DLBCL cohort studied to date with a genome-wide structural assay highlights the limitations of target-limited FISH and the added diagnostic context of Hi-C from FFPE tissue Excerpt from the Press Release:. CARLSBAD, Calif.–(BUSINESS WIRE)–Arima Genomics, Inc., a company leveraging whole-genome sequence and structure information to provide comprehensive cancer therapy selection insights, today announced new…
Overcoming limitations of traditional assay enables definitive diagnosis, refinement of prognosis and opportunities for optimized treatment Excerpt from the Press Release: CARLSBAD, Calif.–(BUSINESS WIRE)–Arima Genomics, Inc., a company leveraging whole-genome sequence and structure information to provide comprehensive cancer therapy selection insights, today announced the publication of a study from investigators at Johns Hopkins University showing…
Excerpt from the Press Release: SOLANA BEACH, CALIFORNIA / ACCESS Newswire / October 7, 2025 / ClearPoint Neuro, Inc. (NASDAQ:CLPT) (the “Company”), a global device, cell, and gene therapy-enabling company offering precise navigation to the brain and spine, today announced results from a Phase I-II clinical study at Skåne University Hospital in Lund, Sweden evaluating…
Key CMC achievements advance EB-003 on the path to first-in-human trials Excerpt from the Press Release: CAMBRIDGE, Mass.–(BUSINESS WIRE)–Enveric Biosciences (NASDAQ: ENVB) (“Enveric” or the “Company”), a biotechnology company advancing next-generation neuroplastogenic small molecules to address psychiatric and neurological disorders, today announced significant progress in the chemistry, manufacturing, and controls (CMC) development of its lead…
Excerpt from the Press Release: NEWTOWN SQUARE, Pa., Sept. 09, 2025 (GLOBE NEWSWIRE) — ArriVent BioPharma, Inc. (Company or ArriVent) (Nasdaq: AVBP), a clinical-stage company dedicated to accelerating the global development of innovative biopharmaceutical therapeutics, today presented positive final proof-of-concept data from the randomized global Phase 1b FURTHER trial for first-line firmonertinib monotherapy in patients…
~ All patients in the first cohort achieved between 27- to 208-fold increases in α-Gal A activity relative to mean normal level ~ ~ All patients in first cohort discontinued enzyme replacement therapy ~ ~ Preliminary data show AMT-191 has a manageable safety profile at the highest dose ~ ~ Updated clinical results expected in…