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

Video

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.

REFERENCE
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
Recent Videos
Steve Kanner, PhD, the chief scientific officer of Caribou Biosciences
David Dimmock, MBBS, on AI-Guided ASO Development for Ultra-Rare Diseases
Manali Kamdar, MD, on The Importance of Bringing Liso-Cel to Earlier Lines of Lymphoma Treatment
Subhash Tripathi, PhD, on Generating In Vivo CARs With A2-CAR-CISC EngTreg Cells
Luke Roberts, MBBS, PhD, on Challenges in Developing Gene Therapy for Heart Failure
Steve Kanner, PhD, the chief scientific officer of Caribou Biosciences
Paul Y. Song, MD, the chairman and chief executive officer of NKGen
Lisa Nieland on Slowing Tumor Growth in Glioblastoma With Novel AAV Therapy
Manali Kamdar, MD, on Acclimating to Routine CAR T Practice in the Field
Paul Y. Song, MD, the chairman and chief executive officer of NKGen
© 2024 MJH Life Sciences

All rights reserved.