Why Shares of Rocket Pharmaceuticals Are Taking Off on Wednesday
Shares of Rocket Pharmaceuticals (NASDAQ: RCKT) were up by more than 36% as of 11 a.m. on Wednesday after the clinical-stage biotech company said late Tuesday that the Food and Drug Administration (FDA) had signed off on its phase 2 pivotal trial for a treatment for Danon disease.
Rocket's gene therapy, RP-1501, is designed to treat Danon disease, a fatal inherited disease that often leads to death in men by age 20 and in women by age 40. According to Rocket, Danon affects between 15,000 to 30,000 patients in the U.S. and Europe. Patients with the rare disease are missing the DNA instructions needed for the body to make the LAMP2 protein, which is crucial to lysosome function and cellular recycling. Danon can cause heart muscle disease, muscle weakness, intellectual disability, and eye disease, and can also affect the lungs and liver. There is no FDA-approved cure for the disease.
The small trial will look at the effectiveness and safety of RP-A501 in a dozen Danon disease patients.
Source Fool.com
