 |
Accelerate your gene therapy development with validated bioanalytical methods that meet FDA and EMA standards.
Accelevir delivers comprehensive gene therapy bioanalytical services built on extensive expertise in AAV testing, immunogenicity monitoring, and vector characterization. Our pre-validated assay panels provide the regulatory-ready data your gene therapy programs require for successful IND and BLA submissions.
Pre-validated panels for AAV, immunogenicity, and vector characterization.
IND & BLA ready.
AAV gene therapy programs face a distinct set of bioanalytical challenges that require specialized expertise, validated methods, and regulatory experience to navigate successfully:
Quantifying vector genomes across diverse tissues — including gonads, CNS, and liver — to demonstrate safety margins and meet FDA and EMA biodistribution guidance.
Detecting vector release into body fluids over extended timepoints to assess environmental transmission risk for regulatory submissions.
Confirming the therapeutic transgene remains intact, correctly packaged, and free from truncations, rearrangements, or ITR alterations that compromise efficacy.
Measuring host cell DNA and plasmid DNA carryover in final product against indication-specific regulatory thresholds.
Monitoring both pre-existing neutralizing antibodies and induced adaptive immune responses to AAV capsids and transgenes to inform patient selection and dosing strategies.
Bringing cell and gene therapies to patients demands rigorous bioanalytical testing at every stage. Proper bioanalytical support ensures your therapeutic development meets regulatory expectations while generating the safety and efficacy data needed for clinical trials.
FDA’s ICH S12 guidance and EMA ATMP requirements outline expectations for comprehensive biodistribution and shedding studies, with specific study designs determined by product type, route of administration, and indication. Our regulatory expertise ensures your submissions align with current ICH principles and agency expectations.
AAV biodistribution assay data reveals off-target transduction patterns, tissue accumulation, and potential long-term toxicity risks across multiple species and timepoints.
Genome integrity sequencing identifies truncations, chimeric species, and ITR alterations that can compromise therapeutic efficacy and patient safety.
Immunogenicity testing monitors both pre-existing and induced immune responses to viral vectors, informing patient selection and dosing strategies for your gene therapy research.
We provide comprehensive vector biodistribution analysis using state of the art qPCR ddPCR platforms for absolute quantification of vector genomes across tissues. Our multiplex dPCR delivers precision at low copy numbers with tolerance to PCR inhibitors common in complex biological matrices.
Our dPCR viral vector quantification methods enable sensitive detection of AAV genomes in serum, urine, saliva, and feces. Longitudinal tracking captures shedding duration and magnitude to assess environmental and transmission risks required for regulatory submissions.
Understanding immune responses to gene therapy modalities is critical for both safety and efficacy. Our dedicated team delivers comprehensive immunogenicity testing spanning innate, humoral, and cellular immunity.
Absolute dPCR viral vector quantification without standard curves, achieving sensitivity approaching single-digit copies per microgram of input DNA. Limit of detection reflects a combination of dPCR platform sensitivity, sample input volume, and tissue matrix effects.
Panel-based assessment of vector DNA distribution with cross-species mapping capabilities.
Long-read sequencing platforms detect truncations, rearrangements, and ITR alterations affecting therapeutic efficacy.
Individual viral genome analysis for variant discovery and population heterogeneity assessment. This approach resolves mixed genome populations — including truncated, chimeric, or rearranged species — that bulk sequencing methods may mask through averaging across the sample population.
Quantification of host cell DNA and plasmid DNA contaminants against regulatory thresholds.
Validated cellular response assays with peptide pools and established cut-point determination.
Sensitive dPCR methods tracking genome copies across matrices over time.
Cell-based NAb assays capturing functional inhibition of vector transduction.
Multiparameter phenotyping with activation markers and tetramer staining.
Multiplex immunoassays for pro-inflammatory markers including IL-6, TNF-α, and IL-1β.
Multiplex immunoassays for pro-inflammatory markers including IL-6, TNF-α, and IL-1β.
Size profiling and quantification meeting indication-specific regulatory standards.
We assess your regulatory requirements—whether for a nonclinical IND, BLA, or ATMP CTA—and define study objectives, including species, tissues, time points, and sample matrices. Our scientific expertise guides the selection of appropriate bioanalytical methods from our pre-validated library or the identification of custom method development needs.
Our team implements FFP validation per ICH S12 guidance following FDA, EMA, and AAPS guidance. Validation parameters include accuracy, precision, linearity, sensitivity, specificity, dynamic range, limit of quantification, and matrix effects. Pre-validated assays accelerate timelines to weeks rather than months.
Expert execution on validated platforms, including droplet dPCR viral vector quantification, qPCR, multiple sequencing platforms, and multiplex immunoassay systems. Real-time data review ensures quality control through replicate concordance, reference standards, and batch effect monitoring throughout analysis.
We deliver comprehensive solutions including GLP-compliant reports with methodology documentation, raw data, statistical analysis, and interpretation. Our regulatory experience supports IND, BLA, and ATMP submissions with data packages designed for agency review.
"Your assay development and refinement exemplifies the power of our platforms … by thoughtfully leveraging the unique precision, sensitivity, single template multiplexing, and linkage determination capabilities of digital PCR."
"With your support of [our] therapeutic vaccine trial, we were able to quickly generate unbiased data with cutting-edge assays to evaluate the impact of a novel T cell-based therapeutic vaccine in a randomized clinical trial. Given our success working with your group, we have several planned studies testing novel interventions."
"Over the course of our interactions, we have been impressed by the [Accelevir's assays] to support both research studies and clinical trials. We look forward to continuing to work with your group."
"As an in vivo CRO, we rely heavily on strong bioanalytical partners—and working with Accelevir has truly elevated the quality of our studies. They feel like an extension of our team, consistently delivering high-quality, reliable data quickly and giving our sponsors real confidence as they move their programs forward."
Digital PCR provides absolute quantification through partitioning reactions into thousands of individual compartments, eliminating dependence on external standard curves. This approach delivers superior precision at low copy numbers—at moderate to high copy numbers, digital PCR typically achieves coefficients of variation <10%, with variability increasing at the limit of detection due to Poisson sampling statistics—and demonstrates greater robustness against PCR inhibitors present in complex tissue matrices. Select dPCR platforms support multiplexed co-localization analysis to assess whether multiple amplicons derive from the same genome molecule, providing additional information for genome integrity characterization. Platform selection and assay design are critical for reliable co-localization results.
Timeline depends on study complexity including number of tissues, species, and timepoints. For GLP nonclinical AAV biodistribution assays using dPCR, full validation—including linearity, sensitivity, matrix effects, and precision parameters—typically requires 6-12 weeks. Pre-validated assay panels can reduce this to 2-4 weeks. FFP validation will be shorter (maybe 3-6 weeks), and more cost-effective for the client. Novel transgenes or tissue types may require additional development time.
Yes. Our bioanalytical solutions span IND-enabling studies through clinical development. We fit to client needs and FDA guidance without overly burdening projects with one size fits all approach. This includes pre-existing neutralizing antibody screening assays to support patient selection strategies when antibody seroprevalence affects eligibility for gene therapy trials.
Gene therapy programs require a bioanalytical partner with deep scientific expertise, regulatory readiness, and the flexibility to support studies from early preclinical work through clinical trials. Accelevir delivers all of this from a single integrated laboratory.
Molecular quantification and immunological monitoring under one roof eliminates inter-laboratory variability, simplifies logistics, spares precious samples, and ensures data consistency across your regulatory package.
Validated workflows for challenging low-input samples and cross-species studies — including rodent, non-human primate, and human — without compromising sensitivity or reproducibility.
Deep familiarity with FDA S12 guidance, EMA ATMP requirements, and ICH principles means your data packages are designed for agency review from day one.
Our team engages at the study design stage — not just the execution stage — to ensure your bioanalytical strategy aligns with your regulatory pathway and clinical objectives.
Bring your cell and gene therapies forward with tailored solutions from a team with extensive experience across gene therapy modalities. Our integrated molecular and immune endpoint capabilities deliver robust solutions for the unique challenges of AAV biodistribution assay development and vector distribution analysis.