AVXS-101 Is Getting Fast-Tracked Through the Review Process in Japan

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      AVXS-101 has been awarded the SAKIGAKE designation in Japan for the treatment of spinal muscular atrophy (SMA) type 1. This designation will speed the therapy’s development and review process and allow faster license applications in Japan.

      AVXS-101, the lead product candidate of AveXis, is a gene therapy under development for the treatment of SMA type 1 and SMA type 2. It delivers a normal copy of the SMN1 gene to the motor neurons of patients with SMA who carry a faulty SMN1. The therapy is intended to re-establish the production of functional SMN1 in motor neurons, avoiding their degeneration and preventing further muscle weakening and wasting.

      Read more about this exciting news here: “AveXis’ SMA Type 1 Gene Therapy Granted Speedier Review in Japan

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