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Forty Seven and Rocket Collaborate for Fanconi Anemia Research
Mar. 11, 2020 06:00AM PST
Pharmaceutical InvestingForty Seven and Rocket have entered into a research collaboration to pursue clinical proof-of-concept for Forty Seven’s novel conditioning regimen with Rocket’s stem cell gene therapy.
Forty Seven (NASDAQ:FTSV) and Rocket Pharmaceuticals (NASDAQ:RCKT) have entered into a research collaboration to pursue clinical proof-of-concept for Forty Seven’s novel antibody-based conditioning regimen with Rocket’s stem cell gene therapy.
As quoted in the press release:
The initial collaboration will evaluate this treatment regimen in Fanconi Anemia (FA), a genetic disease that affects patients’ capacity to produce blood cells and is associated with an increased risk of leukemia and other neoplasms. RP-L102, Rocket’s gene therapy approach for FA, involves treatment with patients’ own gene-corrected blood forming stem cells (hematopoietic stem cells, or HSCs).
Gene therapies for monogenic blood disorders have broad potential. One concern associated with these treatments is the toxicity of pre-therapy conditioning regimens that utilize cytotoxic chemotherapy and/or radiation to destroy existing HSCs and facilitate engraftment of gene-corrected HSCs.
