Ultragenyx Pharmaceutical said Thursday that its experimental gene therapy DTX301 (avalotcagene ontaparvovec) met one of the main goals in a late-stage trial of ornithine transcarbamylase (OTC) deficiency, helping control ammonia levels in patients with the disorder. The therapy, delivered as a single intravenous infusion using an AAV8 vector, is designed to provide stable expression and activity of the OTC enzyme, addressing the genetic defect that prevents patients from properly detoxifying a

- Study to Enroll 80 Patients Unlikely to Benefit from Venetoclax/Azacitide (Aza/Ven) Therapy in the US and Europe - - SELLAS’ Predictive Biomarker and AI Assisted Precision Medicine Models to be Utilized - - Topline Data Expected in Q4 2026 – March 12, 202 -- SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS” or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today announced the first

Groundbreaking First-in-Human Study Demonstrates Potential to Treat Type 1 Diabetes by Transplanting Insulin-Secreting Cells Without Immunosuppression 14-Month Follow-up Data Show Hypoimmune (HIP)-Modified Islets are Safe, Evade Detection by the Immune System, Survive Long-Term, and Continue to Produce Insulin C-Peptide Levels at Month 14 Comparable to Initial Six Months of Study; Results Highlight the Importance of Improved Glycemic Control on Islet Function Full 14-Month Data to Be Presente

March 13, 2026 -- On March 12, Unixell Biotech achieved a significant milestone as UX-GIP001, its iPSC-derived allogeneic cell therapy targeting focal epilepsy, secured Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA). This breakthrough marks UX-GIP001 as the first iPSC-derived allogeneic epilepsy cell therapy approved in the U.S. and simultaneously establishes it as China's pioneering cell therapy for epilepsy to advance into the clinical stage. Epileps

- Designation Underscores the Significant Unmet Medical Need as AMXT 1501 Advances in Two Active National Clinical Trials — Including a Pediatric Study Now Enrolling Children with DIPG March 12, 2026 -- Aminex Therapeutics, Inc., a clinical-stage biotechnology company focused on developing a novel metabolic-targeted therapy to treat adult and pediatric cancers, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AMXT 1501 in combination

The approval is based on positive results from two phase 3 trials (POETYK PsA-1 and POETYK PsA-2), both showing that the drug improves disease activity. Al Reba, senior vice president, Cardiovascular & Immunology Commercialization, Bristol Myers Squibb, said: “This latest approval of Sotyktu confirms its important role in managing both skin and joint symptoms of psoriatic disease and is a key milestone as we continue to explore its development in diseases that have limited or no treatment optio

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