Loading...

AAV Gene Therapy

Regulatory-Ready AAV Gene Therapy Assay Services

AAV biodistribution testing, shedding analysis, AAV genome integrity sequencing, and immunogenicity assays are essential for regulatory approval of adeno-associated virus (AAV) gene therapies. 

Accelevir offers GLP AAV testing CRO capabilities, pre-validated assay panels, and low-input workflows for vector copy number analysis in preclinical and clinical programs.

Challenges in AAV Gene Therapy Development

  • Biodistribution mapping: Quantifying vector genomes in diverse tissues to meet FDA/EMA guidelines.
  • Shedding analysis: Detecting vector release into body fluids to assess transmission risk.
  • Genome integrity: Ensuring the therapeutic transgene remains intact and correctly packaged.
  • Residual impurities: Measuring host cell or plasmid DNA carryover in final product.
  • Immune responses: Monitoring adaptive and innate immunity to AAV capsids and transgene.

How Accelevir Supports AAV Programs

  1. Biodistribution Studies
  • Multiplex dPCR/qPCR for vector genome copy number in multiple tissue types and species.
  • GLP-compliant workflows aligned with FDA/EMA expectations.
  1. Vector Shedding Analysis
  • Sensitive detection of AAV genomes in serum, urine, saliva, feces.
  • Longitudinal sample tracking for shedding duration and magnitude.
  1. Genome Integrity Assessment
  • Long-Read Sequencing to identify truncations, rearrangements, or partial genomes.
  • Single-Viral-Genome Sequencing for variant discovery.
  1. Residual DNA Testing
  • dPCR assays for plasmid DNA or host cell DNA contaminants.
  • Validation against regulatory thresholds.
  1. Immunogenicity Monitoring
  • ELISpot/ICS for capsid- and transgene-specific T cells.
  • Multiparameter Flow Cytometry for immune subset phenotyping.
  • Cytokine Multiplex for inflammatory biomarker profiling.

Why Partner with Accelevir

  • Pre-validated AAV assay library shortens timelines.
  • Integrated molecular + immune endpoints from a single lab.
  • CLIA-certified, GLP-ready environment for clinical and preclinical studies.
  • Experienced in low-volume and multi-species sample workflows.
Accelevir | © 2023