Danon disease data drives Rocket 83% higher

Rocket Pharmaceuticals (Nasdaq: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, closed 83% higher on Wednesday.

The New York-based company had announced preliminary data from its open-label, Phase I clinical trial of RP-A501, an adeno-associated viral vector (AAV)-based gene therapy candidate expressing LAMP2B for the treatment of Danon disease.

This condition is a rare X-linked inherited disorder caused by genetic mutations in the LAMP2 gene resulting in accumulation of autophagosomes and glycogen, particularly in cardiac muscle and other tissues, which ultimately leads to severe and frequently fatal cardiomyopathy.