Newsroom | 20237 results

FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum to Host Call to Share Topline Results from the Phase 3 EXPAND Study of LIVMARLI® (maralixibat) in Ultra-Rare Cholestatic Liver Diseases...

OLDSMAR, Fla.--(BUSINESS WIRE)--Cryo-Cell International, Inc. (NYSE American, Symbol: CCEL) (the “Company”, “Cryo-Cell”), the world’s first private cord blood bank to separate and store stem cells in 1992, announced results for the fiscal third quarter ended August 31, 2026. Financial Results Revenue Consolidated revenues for the third quarter of fiscal 2026 were $7.82 million compared to $7.83 million for the third quarter of fiscal 2025. Net Income The Company reported net income of $1.27 mil...

BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced new interim data from the ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor) 2 to 5 years old open-label extension study, highlighting recovery of exocrine pancreatic function in some children in that age group, allowing those children to discontinue pancreatic enzyme replacement therapy (PERT). Pancreatic exocrine insufficiency in children with cystic fibrosis (CF) was previously believed to be irreversib...

WALTHAM, Mass.--(BUSINESS WIRE)--Affinia Therapeutics (“Affinia”), an innovative clinical-stage gene therapy company with a pipeline of first-in-class and/or best-in-class adeno-associated virus (AAV) gene therapies initially for devastating cardiovascular diseases, today announced that AFTX-201 preclinical and translational data and the Phase 1/2 UPBEAT™ clinical trial investigating AFTX-201 in patients with BAG3-associated dilated cardiomyopathy (DCM), will be featured at the Heart Failure So...

AUSTIN, Texas--(BUSINESS WIRE)--Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA and precision medicine, today announced that its multi-cancer early detection (MCED) assay has been granted Breakthrough Device designation (BDD) by the U.S. Food and Drug Administration (FDA). The BDD is designed for novel technologies that enable more effective diagnosis or treatment of life-threatening or irreversibly debilitating diseases or conditions. Natera’s MCED assay analyzes cell-free DNA in...

LONDON--(BUSINESS WIRE)--AviadoBio Presents New Safety and Biomarker Data from the Phase 1/2 ASPIRE-FTD Trial at the International Conference on Frontotemporal Dementias...

SAN ANTONIO--(BUSINESS WIRE)--bioAffinity Technologies receives notice of allowance for new patent in Japan covering its noninvasive CyPath Lung diagnostic test for lung cancer....

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of patients with rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology, today announced it has resubmitted the Marketing Authorisation Application (MAA) for apitegromab for the treatment of children and adults with spinal muscular atrophy (SMA) to the European Medicines Agency (EMA). Informed by ongoing dialogue...

BASEL, Switzerland--(BUSINESS WIRE)--aiPTO TechBio today released a preprint [link] describing DELPHAI (Deep Explainable Perturbation Heterogeneity-Aware Inference), its virtual tumor model for perturbation prediction. In two independent benchmarking frameworks (Wei et al., Nature Methods 2026; Radig et al., Genome Biology 2026), DELPHAI achieved the #1 ranking on differentially expressed gene (DEG) recovery, outperforming state-of-the-art virtual cell models. It predicts drug responses for prev...

NEW YORK--(BUSINESS WIRE)--TG Therapeutics, Inc. (NASDAQ: TGTX), today announced the schedule of upcoming presentations at the 10th Joint ACTRIMS-ECTRIMS Meeting, also referred to as MSToronto2026, being held October 21-23, 2026, in Toronto, Canada. The presentations include a late-breaking oral presentation highlighting initial results from a Phase 1 study evaluating azer-cel in patients with primary progressive multiple sclerosis (PPMS), as well as several presentations highlighting BRIUMVI®...