Rocket Pharma's lead candidate shows positive effect in Fanconi anemia


From Seeking Alpha: Rocket Pharma's lead candidate shows positive effect in Fanconi anemia – 

  • Results from a Phase 1/2 clinical trial evaluating Rocket Pharmaceuticals' (NASDAQ:RCKT) lead gene therapy candidate, RP-L102, in patients with Fanconi anemia (FA) showed a treatment effect. The data were presented at ASGCT in Chicago.
  • Five patients have been treated to date. All demonstrated continued improvement following treatment with RP-L102, designed to restore the functionality of bone marrow hematopoietic stem cells.
  • Patient #1 experienced a 44% improvement in peripheral blood vector copy number (VCN), a measure of the transduction (transfer of genetic material) of hematopoietic progenitor cells.
  • No serious treatment-related adverse events have been observed.
  • Fanconi anemia is an inherited disorder characterized by impaired bone marrow function that leads to irregular skin coloring and physical abnormalities. It occurs in 1 in 160K people globally.


Claudine Prowse, Ph.D.
SVP Corporate Development and IRO
Rocket Pharmaceuticals, Inc.
The Alexandria Center for Life Science
430 East 29 Street, Suite 1040
New York, NY 10016


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