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

17-May-2018

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.

CONTACTS

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
cp@rocketpharma.com

RELATED LINKS

http://www.rocketpharma.com

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