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.
