The third and final risk to consider would be in terms of the regulatory filings and reviews of gene therapy RP-L102 for the treatment of patients with Fanconi Anemia [FA]. The risk here is that ...
Rocket’s lentiviral vector-based hematology portfolio consists of late-stage programs for Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure and ...
CRANBURY, N.J., September 17, 2024--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic ...
After additional testing, it was discovered that she has a rare blood disorder, ‘Fanconi Anemia’ which has led to bone marrow failure. Now, Dylan and her father are admitted to St.
RP-L102, Rocket's gene therapy in treatment of Fanconi Anemia, will likely have a BLA filing in Q4, 2024. In Pioneer, I pointed to the potential baked into its late-stage pipeline, while warning ...