- Japan’s Ministry of Health, Labour and Welfare has granted SAKIGAKE Designation for AveXis' (NASDAQ:AVXS) AVXS-101, for the treatment of spinal muscular atrophy (SMA) Type 1. The designation was based on data from the Phase 1 clinical trial of the company’s proprietary gene therapy.
- SAKIGAKE, akin to Breakthrough Therapy status in the U.S. and PRIME status in the EU, is aimed at promoting R&D in Japan for new medical products that satisfy certain criteria and provides for accelerated review of the marketing application.
- AVXS-101 is a gene therapy that delivers a fully functional gene into target motor neuron cells via a non-replicating adeno-associated virus capsid (protein shell of a virus), called AAV9, without modifying the existing DNA of the patient. AAV9, unlike other capsids, crosses the blood-brain barrier, enabling intravenous administration.
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