Rajni Agarwal-Hashmi, MD, on Safety Advantages of Gene Therapy in Fanconi Anemia Over SOC


The professor of pediatrics at Stanford Cancer Institute discussed recent data from clinical trials of the lentiviral gene therapy RP-L102.

"The patient experience is quite different when patients go to transplant: the length of hospitalization, the immediate complications, and the follow ups, which are longer after transplant, and more intense. While, with the gene therapy protocol, a lot of it is avoided just by the absence of all the immunosuppressive and myeloablative agents that we have to give them.”

The gene therapy RP-L102(Rocket Pharma) demonstrated phenotypic correction, including increased resistance to mitomycin-C in bone marrow-derived colony forming cells, concomitant genetic correction and hematologic stabilization in at least 6 of 10 participants with Fanconi anemia with a trend of improvement in a seventh participant, meeting the primary endpoint of its phase 2 study (NCT04069533).

RP-L102 had a favorable safety profile, although 1 participant did experience a grade 2 transient infusion-related reaction which resolved. Based on the positive data, Rocket plans to complete regulatory filings for the therapy in the second half of 2023.

CGTLive spoke with Rajni Agarwal-Hashmi, MD, professor of pediatrics, stem cell transplantation, Stanford Cancer Institute, to learn more about the trial and the recent data presented on RP-L102. She also stressed the advantages of gene therapy over traditional stem cell transplant treatment, especially with regards to safety and the lack of myeloablative conditioning in the treatment course.

Rocket Pharmaceuticals presents positive clinical data from fanconi anemia, pyruvate kinase deficiency and severe leukocyte adhesion deficiency-I programs at the 64th American Society of Hematology (ASH) Annual Meeting. News release. Rocket Pharmaceuticals. December 12, 2022. https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-presents-positive-clinical-data-fanconi-0
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