Gene Therapy

Regulatory Consideration for the Nonclinical Safety Assessment of Gene Therapies
October 4th, 2022
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy
October 4th, 2022
Overview of Analytics Needed to Support a Robust Gene Therapy Manufacturing Process
January 19th, 2022
2020 White Paper on Recent Issues in Bioanalysis: Vaccine Assay Validation, qPCR Assay Validation, QC for CAR-T Flow Cytometry, NAb Assay Harmonization and ELISpot Validation (Part 3 – Recommendations on Immunogenicity Assay Strategies, NAb Assays, Biosimilars and FDA/EMA Immunogenicity Guidance/Guideline, Gene & Cell Therapy and Vaccine Assays)
March 18th, 2021
AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction
August 3rd, 2020
Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease
December 6th, 2019
Genome editing in mitochondria corrects a pathogenic mtDNA mutation in vivo
December 6th, 2019
Axonal transport of AAV9 in nonhuman primate brain.
August 24th, 2018
State of the art: gene therapy of haemophilia.
August 24th, 2018