Rocket Pharmaceuticals Announces Clearance of IND for RP-L201 Gene Therapy

Rocket Pharmaceuticals (Nasdaq:RCKT) a leading U.S.-based multi-platform gene therapy company, announces the clearance of the Company’s Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for RP-L201.

As quoted in the press release:

“This acceptance marks the second Rocket-Sponsored IND cleared for our ex-vivo lenti platform this quarter. We are excited to achieve this important milestone ahead of schedule, and look forward to working with the medical and patient communities to evaluate RP-L201 next year,” said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. “This IND enables Rocket to initiate a Phase 1/2 clinical trial to assess the safety, tolerability, and efficacy of severe LAD-I in children. Severe LAD-I is a devastating pediatric disease and we believe that we may have an opportunity to significantly improve the lives of very young children suffering from this disease with a potentially curative therapy.”

The planned open-label, single-arm, Phase 1 portion of the clinical trial of RP-L201 is expected to enroll two severe LAD-I patients in the U.S. The Phase 2 portion of the clinical trial of RP-L201 is expected to be a registration-enabling global study with clinical sites in the U.S., U.K. and Spain.

Click here to read the full press release.