Entering text into the input field will update the search result below

Rocket Pharma to launch pivotal mid-stage study of gene therapy for rare anemia

Sep. 04, 2019 1:49 PM ETRocket Pharmaceuticals, Inc. (RCKT) StockBy: Douglas W. House, SA News Editor
  • Based on FDA feedback, Rocket Pharmaceuticals (NASDAQ:RCKT -3.8%) will launch a Phase 2 clinical trial next quarter that, it says, should support a U.S. marketing application for gene therapy RP-L102 for Fanconi anemia, a rare inherited disorder characterized by bone marrow failure.
  • The primary endpoint will be resistance to mitomycin-C (MMC), a DNA-damaging chemo agent, in bone marrow cells at a minimum time point of one year. The company says MMC resistance may also serve as a surrogate endpoint for accelerated approval.
  • Another Phase 2 is currently underway in Europe with an estimated completion date of January 2023. The results will support both U.S. and European marketing applications.

Recommended For You

More Trending News

About RCKT Stock

SymbolLast Price% Chg
Market Cap
PE
Yield (TTM)
Rev Growth (YoY)
Prev. Close
Compare to Peers

Related Stocks

SymbolLast Price% Chg
RCKT--
Rocket Pharmaceuticals, Inc.