Regenxbio Inc., a Maryland-based gene therapy company, announced new interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202 in patients with Duchenne muscular dystrophy. The data, presented at the Muscular Dystrophy Association Clinical and Scientific Conference, showed that participants receiving the pivotal dose exceeded external controls across functional measures at one year, according to the company. The AFFINITY DUCHENNE trial is a Phase I/II study evaluating RGX-202 as a gen

Thursday March 12, 2026 – Omios Biologics, a biotechnology company revolutionizing cancer treatment through next-generation oncolytic virus immunotherapies and systemic gene delivery platforms, today announced the signing of a Material Transfer Agreement with the Beckman Research Institute of City of Hope. Under the agreement, City of Hope, one of the largest and most advanced cancer research and treatment organizations in the United States, will conduct advanced studies to further characteri

— Fast Track Designation facilitates development and expedites regulatory review of therapies addressing serious conditions with significant unmet medical need — — QRX003 lotion (4%) currently being evaluated in two late-stage whole-body clinical trials for treatment of Netherton Syndrome — — Fast Track Designation follows Pediatric Rare Disease and Orphan Drug Designation previously granted by the FDA and Orphan Drug Designation granted by the European Medicines Agency for QRX003 in Netherton S

A new synthesis finds that common epilepsies are driven by thousands of tiny-effect genetic variants, most still undetected. March 10, 2026 --- An insightful mini-review published in Genomic Psychiatry synthesizes the rapidly expanding landscape of molecular genetic research on common epilepsies, assembling evidence from genome-wide association studies (large-scale genetic scans across the human genome), whole-exome sequencing projects, and advanced statistical modeling to illuminate the polyge

Multi-target therapeutic aims to treat all key and interconnected problems of atopic dermatitis March 11, 2026 -- Alphyn, a clinical-stage dermatology company developing the first Multi-Target Therapeutics® drug platform, announced today that it has completed enrollment in its CLEAR-AD1 Phase 2b clinical trial of Zabalafin Hydrogel, a first-in-class topical therapeutic for the treatment of mild and moderate atopic dermatitis (AD) derived from the company's proprietary Multi-Target Therapeutic D

March 11, 2026 -- ARTHEx Biotech, a clinical-stage biotechnology company advancing RNA-based therapeutics for neuromuscular disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to ATX-01 for the treatment of Myotonic Dystrophy type 1 (DM1). ATX-01 is ARTHEx's investigational RNA therapeutic designed to address the underlying genetic cause of DM1, a highly disabling neuromuscular disorder with no known cure. "Receiving Fast Track Designa

Zemcelpro® (dorocubicel) achieved remarkable and clinically meaningful survival results in patients with high- and very high-risk acute leukemias and myelodysplastic syndromes Topline results from two Phase 2 studies to be presented on March 23, 2026 at the EBMT Annual Meeting in Madrid, Spain March 10, 2026 -- ExCellThera Inc. (ExCellThera), a world leader in blood stem cell expansion and metabolic fitness, and its wholly owned subsidiary Cordex Biologics (Cordex), announce positive results fr

March 10, 2026 / Today Technomark Life Sciences and MAX BioPharma announced their collaboration in the development of MAX BioPharma's oxysterol drug candidate, Oxy210, for targeting metabolic dysfunction-associated steatohepatitis (MASH). As a strategic lead investor, Technomark is investing in MAX BioPharma by participating in its $13 million Series A raise to initiate and complete a Phase 1a/1b first-in-human study and invites aligned investors from the industry to join this opportunity. "We

March 10, 2026 -- Eledon Pharmaceuticals, Inc. (“Eledon”) (NASDAQ: ELDN) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to tegoprubart for the prevention of allograft rejection in liver transplantation. Tegoprubart has previously received orphan drug designation from the FDA for the prevention of allograft rejection in pancreatic islet cell transplantation and for the treatment of amyotrophic lateral sclerosis (ALS). “Clinical studies in kid

Results demonstrated early signals of C-peptide preservation T1D key opinion leader, Michael J. Haller, MD, provided a recorded webinar reviewing the SAB-142 Phase 1 data March 10, 2026 -- SAB Biotherapeutics, Inc. (Nasdaq: SABS), a clinical-stage biopharmaceutical company developing a fully human anti-thymocyte immunoglobulin (hATG) for type 1 diabetes (T1D) and other autoimmune diseases, today announced additional data from the Phase 1 HUman anti-thymocyte biologic in first-in-MAN (HUMAN) cl