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Rocket Pharmaceuticals Announces Publication of Manuscript Evaluating Mosaicism in Fanconi Anemia
- Publication Affirms Rocket’s Approach to Treating Fanconi Anemia - NEW YORK--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n- Publication Affirms Rocket’s Approach to Treating Fanconi Anemia -\n\n NEW YORK--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, announces the recent publication of a peer-reviewed manuscript: the first comprehensive review of somatic mosaicism in Fanconi Anemia (FA) in the journal Annals of Hematology. The review, entitled, “Mosaicism in Fanconi Anemia: Concise review and evaluation of published cases with focus on clinical course of blood count normalization,” summarizes the existing literature regarding mosaicism in FA, a phenomenon previously described as “natural gene therapy.” Mosaicism occurs when a spontaneous second mutation occurs in an (already-mutated) FA gene, resulting in a functional FA protein and restored cellular DNA-repair capacity. When this type of reversion or compensatory mutation occurs in hematopoietic stem cells (HSCs), these cells have at times been capable of restoring a more normalized blood and bone marrow environment, in some cases over multiple decades. The review provides the FA community with an additional resource to better understand the phenomenon and further supports Rocket’s approach of treating patients with RP-L102, the Company’s gene therapy candidate for FA, without a pre-treatment conditioning regimen.\n\n\n“Although mosaicism in FA has been discussed within the physician and research community for more than two decades, uncertainty about the clinical significance of reversion mutations has remained,” said Jonathan Schwartz, M.D., Chief Medical Officer and Senior Vice President of Rocket. “Importantly, findings from this review support the selective growth advantage for gene corrected stem cells over diseased stem cells which potentially obviates the need for conditioning in FA gene therapy. In addition to allowing us to better understand how RP-L102 may best help patients, we hope that bringing these data together into a cohesive review can better help the entire FA community as we work together to optimize treatment options.”\n\n\nThe publication includes a literature-based assessment of FA mosaicism and identification of a cohort of cases that most likely emanated from reversion in long-term repopulating HSC populations. Addition...