Rocket Pharma has ‘encouraging’ data from anemia gene therapy trial

Rocket Pharmaceuticals announced the presentation of updated data from the ongoing Phase 1/2 clinical trial of RP-L102, the company’s lead lentiviral vector-based gene therapy, for Fanconi Anemia. The data were highlighted today in an oral presentation during the distinguished Presidential Symposium at the ASGCT 2018 Annual Meeting, by Dr. Juan Bueren, Head of the Hematopoietic Innovative Therapies Division at the Centro de Investigaciones Energeticas, Medioambientales y Tecnologicas in Spain / CIBER-Rare Diseases / IIS-Fundacion Jimenez Diaz, and program principal investigator of the RP-L102 trial. “Several important observations are emerging from our ongoing Phase 1/2 trial in FA. First, even without myeloablative conditioning, there are increasing levels of bone marrow engraftment following administration of RP-L102. Second, the improvement of chromosomal stability in corrected FA cells indicates that RP-L102 is reversing the FA phenotype. Third, the natural progression of bone marrow failure in these patients is reversed. In fact, the bone marrow cells of the two patients who received higher doses demonstrate conversion to a somatic mosaic status that is sustained over the course of several months. Finally, the progressive increases of corrected, versus non-corrected, peripheral blood leukocytes indicate that RP-L102 is restoring functionality of bone marrow hematopoietic stem cells. This translates to a stabilization in peripheral blood cell counts which would otherwise continue to decline in the absence of treatment. Based on these encouraging results, I believe that RP-L102 has the potential to be a transformative and minimally toxic prevention of bone marrow failure for FA,” said Dr. Bueren.

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