The clinical professor of pediatrics at Stanford Medicine discussed positive interim findings from a phase 1 study of RP-L301.
“We expected some gradual improvement and we expected maybe they feel a little bit better. But we didn't really expect people to come out of this completely normal... [in terms of] side effects, they don't even feel like they had a transplant and... they can't remember this disease anymore.”
Patients with pyruvate kinase deficiency (PKD) treated with RP-L301, a gene-edited lentiviral mediated hematopoietic stem and progenitor cell therapy, reached normal levels of hemoglobin production, improved hemolysis and sustained transgene expression with no need for red blood cell transfusions at 1-year post infusion.
These data, from an ongoing phase 1 study (NCT04105166) presented at the 2022 American Society of Gene and Cell Therapy Annual Meeting, May 16-19, 2022, in Washington DC, by Ami J. Shah, MD, clinical professor of pediatrics, Division of Hematology/ Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford Medicine.
CGTLive spoke with Shah to learn more about the efficacy findings of the trial and the positive patient-reported outcomes. She also discussed the progress of the trial and the new pediatric cohort being enrolled.