Rocket Pharma rises on FDA alignment on trial design for rare disorder therapy

Rocket Pharmaceuticals

13/09/2023 11:08

** Shares of gene therapy developer Rocket Pharmaceuticals
Inc  RCKT.O  rise 19.29% to $18.24 premarket
    ** Co says has reached FDA alignment on mid-stage trial
design for its investigational gene therapy RP-A501 in Danon
disease
   ** The mid-stage study will study the efficacy and safety of
RP-A501 in 12 patients to support accelerated approval
    ** Danon disease is a rare inherited disorder that affects
many organ systems including the weakness of the heart and
skeletal muscles and intellectual disability
    ** Up to last close, stock down ~22% YTD

 (Reporting by Sriparna Roy in Bengaluru)
 ((Sriparna.Roy@thomsonreuters.com;))