→ Bluebird Bio says that its European application for LentiGlobin, its gene therapy for transfusion-dependent β-thalassemia, has made it through the doors at the EMA. The acceptance sets up a marketing decision for this therapy as the biotech plots a shift to commercialization work.
→ Sarepta is all in on gene therapies. Banking on early but notable successes for its Duchenne MD program, Sarepta is following up with its partners at Nationwide Children’s Hospital to gain rights to the gene therapy candidate, neurotrophin 3, to treat Charcot-Marie-Tooth neuropathies, including CMT type 1A.
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