Axovant: Rises on FDA's rare pediatric disease status for gene therapy

Sio Gene Therapies

09/10/2020 12:48

** Shares of gene therapies developer  AXGT.O  up 6.5% at
$4.1 premarket
    ** Co says it has received rare pediatric disease
designation from U.S. FDA for its gene therapy, AXO-AAV-GM1, to
treat GM1 gangliosidosis  urn:newsml:reuters.com:*:nGNX1s2FW4
    ** GM1 gangliosidosis is a genetic and rare disease that
progressively destroys nerve cells in brain and spinal cord
    ** AXO-AAV-GM1 also has Orphan Drug designation
    ** Co says Axovant is on track to report 6-month early-stage
data from low dose juvenile cohort by Q4
    ** Up to Thursday's close, stock had fallen 25% YTD
    

 (Reporting by Dania Nadeem in Bengaluru)
 ((Dania.Nadeem@thomsonreuters.com;))