The FDA has granted Orphan Drug designation to Affinia Therapeutics' AFTX-201, a one-time IV gene therapy for BAG3-associated dilated cardiomyopathy. It follows a recent Fast Track grant; AFTX-201 is in the Phase 1/2 UPBEAT trial recruiting across the U.S. and Canada.
Affinia Therapeutics will present new AFTX-201 preclinical data, the UPBEAT Phase 1/2 trial design, and proprietary capsid engineering and manufacturing advancements across two oral and four poster sessions at ASGCT 2026 in Boston, May 11-15.
DemeRx announced that the FDA has accepted its IND application for DMX-1001 (oral noribogaine), a novel neuroplastogen therapeutic for the treatment of alcohol use disorder (AUD).
Affinia Therapeutics announced Health Canada’s approval of its clinical trial application for AFTX-201, a potential best-in-class investigational genetic medicine for the treatment of BAG3-associated dilated cardiomyopathy (DCM).
Aspect Biosystems announced receiving a $79 million investment from the Government of Canada, supporting a $280 million, multi-year project to accelerate its pipeline of bioengineered cellular medicines for serious metabolic and endocrine diseases.
BELMONT, Mass. – March 23, 2026 – Oryon Cell Therapies (“Oryon”), a clinical-stage biotechnology company focused on developing autologous neuron replacement medicines for Parkinson’s disease and other neurodegenerative disorders, today announced new clinical and neuroimaging data from an ongoing Phase 1b/2a study evaluating its autologous dopaminergic neuron replacement therapy
BELMONT, Mass. – March 23, 2026 – Oryon Cell Therapies (“Oryon”), a clinical-stage biotechnology company focused on developing autologous neuron replacement medicines for Parkinson's disease and other neurodegenerative disorders, today announced it has emerged from stealth and has closed a new $21 million tranche of its Series A financing,
ZURICH, Switzerland — March 19, 2026 — AL-S Pharma AG, a clinical-stage biotechnology company discovering and developing human antibodies that target misfolded proteins implicated in amyotrophic lateral sclerosis (ALS), today announced the presentation of new clinical data from the global Phase 2 clinical trial (AP-101-02) evaluating the company’
About Media Contact Legal → Download PDF Corporate Overview Ocugen, Inc. About Ocugen, Inc. ▼ Ocugen, Inc. is a pioneering biotechnology leader in gene therapies for blindness diseases. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach.
✨ Onyx Summary Affinia Therapeutics announced that the U.S. Food and Drug Administration has granted Fast Track designation to AFTX-201, its investigational gene therapy for BAG3-associated dilated cardiomyopathy, which is currently being evaluated in the Phase 1/2 UPBEAT clinical trial. The designation signals regulatory support for accelerating
✨ Onyx Summary Belite Bio announced it will host a webcast on March 2, 2026 at 4:30 p.m. ET to discuss fourth-quarter and full-year 2025 financial results and provide a business update. A live webcast and a replay (available for about 90 days) will be accessible via
✨ Onyx Summary Affinia Therapeutics said the European Medicines Agency has granted Orphan Drug designation to AFTX-201, its investigational AAV gene therapy for BAG3-associated dilated cardiomyopathy, following the FDA’s acceptance of its IND. The designation adds regulatory incentives and market-exclusivity potential in the EU as Affinia advances
✨ Onyx Summary Verily launched self-serve access to its Pre precision health platform, adding a free Standard tier that lets individual researchers use Exchange and the Workbench secure analysis environment to discover and analyze select biomedical datasets under a data use agreement. The company also outlined additional data sources coming
✨ Onyx Summary Cellares and the University of Wisconsin School of Medicine and Public Health expanded their partnership to move UW’s CRISPR-edited GD2 CAR-T program for solid tumors into clinical manufacturing and support an FDA IND submission, with Cellares providing automated manufacturing (Cell Shuttle) and automated QC (Cell
✨ Onyx Summary Perspective Therapeutics said it will report full-year 2025 financial results and provide a business update after market close on Monday, March 16, 2025, with the release posted to the company’s newsroom. The update will frame progress across its 212Pb-based theranostic platform and Phase 1/2a
✨ Onyx Summary Verily says its Verily Pre precision health AI platform has achieved “Awardable” status on the Department of War CDAO’s Tradewinds Solutions Marketplace, positioning it as a readily awardable option for DoW agencies. The designation is intended to streamline federal procurement and expands Verily’s path from existing
