Performing In-Depth Analysis of Lentiviral Cell Therapies: Luca Biasco, PhD

“We have developed a complex pipeline that starts from the bone marrow of the patients, and goes all the way to cellular processing, molecular processing, sequencing and computational analysis to deliver a comprehensive overview on how the engineered cells behave, and where they reside once we infuse them into the patient.”

AVROBIO is prioritizing molecular follow-up of lentiviral cell therapies in patients with lysosomal disease with the use of their new analytical pipeline that elucidates cell therapy destiny and distribution. Luca Biasco, PhD, director, research and development, AVROBIO, presented an overview of the pipeline at the 18th Annual WORLDSymposium, February 7-11, 2022 in San Diego, CA.

As part of the analytical pipeline, Biasco and colleagues conducted high-resolution cellular and molecular studies on bone marrow and peripheral blood before and after lentiviral cell therapy treatment. They analyzed subpopulations of bone marrow and peripheral blood cells via digital PCR-vector copy number and integration site analysis, as also used clonal tracking data to elucidate the dynamics and destiny of cell populations after gene therapy.

CGTLive spoke with Biasco to learn more about the analyses of the analytical pipeline. He discussed their methods and the importance of the analysis.

REFERENCE

Biasco L, Baricordi C, Loperfido M, et al. High throughput monitoring of safety, potency and stability of gene therapy cell products in lysosomal disease patients. Presented at: 18th Annual WORLDSymposium. February 7-11, 2022; San Diego, CA. Poster #26