LentiGlobin BB305 gene therapy regulatory update

NIH’s Recombinant DNA Advisory Committee wants bluebird to delay a pediatric trial of its LentiGlobin BB305 gene therapy by 1-2 years. The recommendation followed RAC’s review of bluebird’s protocol for the HGB-208 trial to treat beta-thalassemia major in pediatric patients. bluebird said its protocol for the similarly designed HGB-207 trial in adult and adolescent patients “was supported by the RAC without comment.” bluebird is not commenting on timelines, but the company did say the timelines have not changed following the RAC recommendations. bluebird said it its moving forward as planned with its studies (see BioCentury, June 1). ...