By Sabela Ojea

 

Shares of Rocket Pharmaceuticals surged 34% to $20.46 in post-market trading on Tuesday after the company said it reached alignment with the Food and Drug Administration regarding its Phase 2 trial for the treatment of Danon disease.

The Cranbury, N.J.-based pharmaceutical company said its study would evaluate the use of the investigational gene therapy RP-A501 to treat 12 patients with Danon disease.

Danon is a rare genetic disorder that has no cure, and is known for weakening the heart and movement muscles, as well as intellectual disability.

The trial will support accelerated approval of Rocket's treatment by the FDA.

 

Write to Sabela Ojea at sabela.ojea@wsj.com; @sabelaojeaguix

 

(END) Dow Jones Newswires

September 12, 2023 17:13 ET (21:13 GMT)

Copyright (c) 2023 Dow Jones & Company, Inc.
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