Verve Therapeutics’ VERVE-102 shows early success in lowering LDL cholesterol by 55% with a single dose, offering a potential one-time gene editing treatment for familial hypercholesterolemia...
Beam Therapeutics announces groundbreaking results from its Phase 1/2 trial of BEAM-302, demonstrating the first-ever clinical genetic correction for Alpha-1 Antitrypsin Deficiency (AATD), offering hope for a potential one-time cure for this genetic disorder...
Sarepta Therapeutics announces FDA approval of ELEVIDYS, the first gene therapy to treat Duchenne Muscular Dystrophy. ELEVIDYS addresses the root genetic cause of Duchenne – mutations in the dystrophin gene that result in the lack of dystrophin protein – by delivering a gene that codes for a shortened form of dystrophin to muscle cells known as ELEVIDYS micro-dystrophin...
2seventy bio announced new data featuring novel approaches combining the company’s platform CAR T cell and T cell receptor technology and unique cell therapy engineering capabilities to potentially enhance treatment potency in a range of cancers...
Preclinical data demonstrates that hypoimmune-modified allogeneic iPSCs evade immune response and rejection without immunosuppression in a non-human primate model and long-term survival and immune evasion was at least equivalent to the survival of autologous iPSCs...
Company files its first Investigational New Drug application for its lead program KYV-101, a novel fully human CD19 CAR T-cell therapy, for the treatment of lupus nephritis. Kyverna’s therapeutic platform combines advanced T-cell engineering and synthetic biology technologies to suppress and eliminate...
Kyverna Therapeutics, a cell therapy company engineering a new class of therapies for serious autoimmune diseases, today announced it has closed an oversubscribed $85 million Series B financing round led by Northpond Ventures. Additional investors included the company’s founding investors Westlake Village BioPartners (“Westlake”), Vida Ventures, and Gilead Sciences (“Gilead”), with new investors RTW [...]
