- The U.S. Food and Drug Administration (FDA) granted regenerative medicine advanced therapy (RMAT) designation to Rocket Pharmaceuticals' ( NASDAQ: RCKT ) gene therapy RP-A501 to treat Danon Disease.
- Danon disease is a rare genetic disorder characterized by weakening of the heart muscle, muscles for movement (skeletal muscles), and intellectual disability. The condition is caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2).
- "RP-A501 is the first cardiac gene therapy to receive RMAT designation from the FDA," said Rocket CEO Gaurav Shah.
- The company expects to start a phase 2 in Q2 2023 of the adeno-associated virus (AAV)-based gene therapy RP-A501.
- RCKT +4.23% to $21.20 premarket Feb. 7
For further details see:
Rocket rises on FDA regenerative medicine tag for cardiac gene therapy