Rocket Pharmaceuticals Announces Publication of Data from Phase 1/2 Trial of First-Generation RP-L102 for Fanconi Anemia in Nature Medicine
Rocket Pharmaceuticals, Inc. (RCKT)
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Source: Business Wire
—First Demonstration of Successful Engraftment of Gene Corrected Hematopoietic Stem Cells Without the Use of Conditioning— NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a leading U.S.-based multi-platform clinical-stage gene therapy company, today announces the publication of long-term data from the ongoing Phase 1/2 trial of RP-L102, the Company’s lentiviral vector (LVV)-based gene therapy for Fanconi Anemia (FA) in the journal Nature Medicine. The data included in the manuscript are from the first four patients treated with RP-L102 in the Phase 1/2 FANCOLEN-I trial that utilized first-generation “Process A” without the use of any conditioning regimen. Follow-up for each of the initial four patients was 18-30 months from administration of RP-L102.“Data from our first trial of RP-L102 demonstrate increasing levels of bone marrow engraftment, leading to stabilization and restored bone marrow function. These data highlight the natural selective ad
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RCKT
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- 11/7/24 - Beat
RCKT
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