BERKELEY, Calif. — Ray Therapeutics (RayTx), a clinical-stage biopharmaceutical company pioneering optogenetic therapies for those living with retinal degenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its lead candidate, RTx-015, for the treatment of retinitis pigmentosa (RP)....
treatment News
Dublin, Ireland. Boosting levels of a deficient protein has clear survival benefits for people with the genetic condition alpha-1 antitrypsin deficiency (AATD), according to new research led by RCSI University of Medicine and Health Sciences. The findings underscore a call to make augmentation therapy accessible to patients with AATD in...
Ann Arbor, Mich. – High-grade glioma, an aggressive form of pediatric and adult brain cancer, is challenging to treat given the tumor location, incidence of recurrence and difficulty for drugs to cross the blood-brain barrier. Researchers from the University of Michigan, Dana Farber Cancer Institute and the Medical University of...
New York, NY – In people with pulmonary arterial hypertension (PAH), adding Winrevair (sotatercept-csrk) to standard therapy was linked to longer survival, fewer safety events, and a lower need for lung transplant compared with standard therapy alone, according to new real-world data. “These findings extend clinical trial evidence, suggesting sotatercept [Winrevair] offers meaningful...
OSAKA, Japan & CAMBRIDGE, Mass. – VPRIV (velaglucerase alfa), an approved enzyme replacement therapy (ERT) for Gaucher disease, appears to be safe and effective in children under 4—a population not included in the clinical trials that supported its approval. A recent U.S. real-world study evaluated VPRIV in a small group...
Rzeszów, Poland – One year of treatment with Kaftrio significantly reduced levels of key inflammatory immune cells, lowered the frequency of pulmonary exacerbations, and decreased detection of Pseudomonas aeruginosa in people with cystic fibrosis (CF), according to a real-world study. The anti-inflammatory effects were most pronounced in patients who had...
NEW HAVEN, Conn. – Long-term treatment with Cerezyme (imiglucerase), or its discontinued predecessor Ceredase (alglucerase), led to rapid and progressive improvements in blood counts, growth, and bone health, as well as reductions in liver and spleen size, in children with Gaucher disease (GD) — including among youngsters with a more severe disease type....
NORFOLK, Va. — ReAlta Life Sciences, Inc., a mid-stage clinical biotech company dedicated to saving lives by rebalancing the inflammatory response to address life-threatening diseases, today announced enrollment of the first patient in a Phase 2 clinical trial evaluating the safety and efficacy of RLS-0071, an investigational new drug based...
NORFOLK, Va. — ReAlta Life Sciences, Inc. (“ReAlta” or the “Company”), a clinical-stage biopharmaceutical company dedicated to saving lives by rebalancing the inflammatory response to address rare and acute inflammatory diseases, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to RLS-0071 (pegtarazimod) for the treatment of...
PLANO, Texas – Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the “Company,” or “we”), a clinical-stage biopharmaceutical company, today announced that it received a communication from the Division of Neurology Products 1 (“Division”) of the U.S. Food and Drug Administration (“FDA”) stating that, after a preliminary review of briefing materials for...