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

— 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

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 -- 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

Phase I results demonstrate favorable safety, sustained target engagement, and early signs of anti-tumor activity Reinforce EP4 receptor antagonism as a validated, high-value strategy to overcome immunotherapy resistance March 10, 2026 -- Kainova Therapeutics (“the Company”), a key player for breakthrough treatments in immuno-oncology and inflammation, today announced positive topline results from its Phase I EPRAD study evaluating DT-9081, a proprietary, oral small molecule EP4 receptor (EP4R)

Niraparib and abiraterone acetate regimen demonstrates clinically meaningful delay in disease progression, nearly halving the risk of progression or death, with an early trend toward improved overall survival versus standard of care1 Approximately one in ten patients with mHSPC harbour BRCA1/2 alterations, reinforcing the need for new targeted treatment options2 Expanded indication for niraparib and abiraterone acetate delivers a precision-based treatment approach for this patient population

March 9, 2026 -- Harbour BioMed (HKEX: 02142), a global biopharmaceutical company committed to the discovery and development of novel antibody therapeutics in immunology and oncology, and Kelun-Biotech (6990.HK) today announced that the National Medical Products Administration (NMPA) of China has approved the Investigational New Drug (IND) application for HBM7575/SKB575, a long-acting bispecific antibody targeting thymic stromal lymphopoietin (TSLP) and an undisclosed target co-developed by the