bluebird bio says achieved general agreement on regulatory path for TDT
bluebird bio announced it will outline today key activities underway intended to advance the company's LentiGlobin programs in transfusion-dependent beta-thalassemia, or TDT, and severe sickle cell disease, or SCD. The presentation will focus on three key aspects of these activities: 1) potential improvements in transduction efficiency and manufacturing; 2) updates to the protocol for the company's ongoing HGB-206 clinical trial in SCD; and 3) regulatory plans for the company's LentiGlobin drug product candidate in TDT. bluebird bio is working closely with regulatory agencies in the United States and Europe to bring LentiGlobin to patients with TDT who can benefit, as quickly as possible, and has reached general agreement with the U.S. FDA and the European Medicines Agency, respectively, on the regulatory paths forward, the company said. "At bluebird bio we have an incredibly ambitious goal: to deliver one-time, transformative therapies to patients with rare genetic diseases and cancer. We are relentless in our efforts to continue innovating in pursuit of that goal, and we have made substantial advances in the transduction and manufacturing processes, translational research, and clinical development. By incorporating manufacturing Process 2 into our LentiGlobin clinical trials, we believe we can achieve our ultimate goal of increasing hemoglobin production in patients treated with LentiGlobin drug product. In sickle cell disease we have used our early clinical data to identify and implement multiple distinct improvements with the potential to overcome the unique challenges presented by this complex disease. We are hopeful that the tremendous work done by our research and development teams will yield improved outcomes for patients in the clinic in 2017 and beyond, and that the regulatory progress we are making will enable us to bring these treatments to patients as quickly as possible. We anticipate seeing many catalysts across our programs in the next 15 months, including initial clinical data from our Phase 1 clinical study of our bb2121 product candidate in relapsed/refractory multiple myeloma and updates on our Lenti-D program in cerebral adrenoleukodystrophy," said bluebird CEO Nick Leschly.