The Need for Analysis in Lentiviral Cell Therapies: Luca Biasco, PhD

“As you would study the pharmacokinetics of chemical drugs, you need to also monitor the biodistribution and dynamics of these cells, with the added complexity that these cells are a much more complicated vehicle for therapies."

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

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

CGTLive spoke with Biasco to learn more about the analytical pipeline. He discussed the advantages of analyzing subpopulations of peripheral blood and bone marrow over analyzing whole blood and bone marrow samples.

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