AVRO - With bluebird bio's success what are other gene therapies companies to pay attention to?

With two overwhelmingly positive U.S. FDA advisory committee votes this week, bluebird bio (NASDAQ:BLUE) is poised to see beti-cel (betibeglogene autotemcel) approved for beta-thalassemia and eli-cel (elivaldogene autotemcel) for cerebral adrenoleukodystrophy. Should the FDA follow the recommendations of the committee, beti-cel and eli-cel would join only a handful of approved gene therapies. This includes Novartis' (NVS) Zolgensma (onasemnogene abeparvovec) and Spark Therapeutics' Luxturna (voretigene neparvovec). Gene therapies are lucrative for companies as they are extremely expensive. Zolgensma, which treats spinal muscular atrophy, has a list cost of $2.1M for a single dose. The high price of the treatments has to do with their complexity. Gene therapies involve modifying or replacing defective genes by inserting new genes into cells directly. This can be done through the use of viral vectors, viruses that have had their ability to cause infectious diseases removed but have been modified deliver therapeutic genes into human cells. Given the

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With bluebird bio's success, what are other gene therapies companies to pay attention to?