… has found that a gene therapy treatment for haemophilia B … six years after treatment.  The gene therapy, called fidanacogene elaparvovec, … hemophilia B received the gene therapy and were followed for … results suggest that the gene therapy is generally safe and …

… Have a cell and gene therapy news update you’d … virus (AAV) vector-based gene therapy, has demonstrated the ability to … made in cell and gene therapy for Parkinson disease over … CSL Behring’s hemophilia B gene therapy etranacogene dezaparvovec (marketed as …

… designation to ATSN-201, a gene therapy candidate for the treatment of … designation to ATSN-201, a gene therapy candidate for the treatment of …

Newsletter | April 16, 2025 Novel analytical technologies like Droplet Digital PCR (ddPCR) provide a more rapid and robust approach to nucleic acid quantification for therapeutic development that better aligns with the industry need for better sensitivity, …

… (RMAT) designation to its investigational gene therapy, ATSN-201, for the treatment … Technology ATSN-201 is a gene therapy candidate that utilizes AAV.SPR …

… published findings on the gene therapy potential for hearing loss … of a cutting-edge gene therapy method using AAV (adeno … demonstrated partial recovery. The gene therapy was administered 30 days … the therapeutic window for gene therapy may be broader than …

… interim data show. “Several microdystrophin gene therapy programs are approved for Duchenne … IV microdystrophin-expressed, AAV gene therapy in a cohort of ambulatory … evidence of antibodies prior to gene therapy administration. At baseline, all …

Patients come to Michael Schilsky, MD, a Yale Medicine gastroenterologist and hepatologist, after struggling for years—sometimes decades—with a range of symptoms such as extreme fatigue, jaundice, personality changes, stutters, and movement issues (such as …

… into the world of retinal gene therapy with Kenji Fujita, M.D …

… (RMAT) designation to ATSN-201 gene therapy, a first-of-its-kind …