Precision Bioanalytics for Next-Generation CRISPR and Gene Editing Programs
CRISPR-Cas9, Cas12, TALENs, and other advanced gene editing technologies hold immense potential to correct genetic diseases at the source. Success in clinical development depends on CRISPR assay development, on-target editing verification, off-target detection assays, and immune response profiling for gene editing tools and delivery vectors such as AAV or LNP.
Accelevir provides world-class digital PCR (dPCR), qPCR, and NGS gene editing assays to move programs from preclinical proof-of-concept to IND/BLA submission.
Challenges in CRISPR & Gene Editing Development
- On-target precision: Regulatory bodies require proof that editing occurred exactly at the intended site.
- Off-target safety: Unintended edits can raise safety concerns and must be quantified at ultra-low frequencies.
- Efficiency optimization: Determining the percentage of cells successfully edited is key for dose selection and process development.
- Immune response management: Delivery vectors (AAV, LNP) and editing enzymes (Cas9, Cas12, TALENs) can trigger innate or adaptive immune reactions.
- Limited sample availability: Preclinical models and early clinical trials often yield small sample volumes, demanding low-input, high-sensitivity workflows.
How Accelevir Supports CRISPR Programs
- On-Target Editing Verification
- Targeted Deep Sequencing: Short- and long-read NGS to confirm intended genome modifications.
- Allele-Specific PCR/dPCR: Quantitative detection of edited vs. unedited alleles.
- Off-Target Detection
- Custom Off-Target Assays: Based on GUIDE-seq, CIRCLE-seq, or predictive bioinformatics.
- High-Sensitivity NGS: Detect rare off-target events down to 0.1% allele frequency.
- Editing Efficiency Quantification
- Digital PCR (dPCR) & qPCR: Absolute quantification of editing frequency in cell and tissue samples.
- Low-Input Capabilities: Robust workflows for scarce clinical or preclinical samples.
- Immune Response Profiling
- T-Cell Assays (ELISpot, ICS): Detect immune responses to nucleases (Cas9, Cas12) and delivery vectors.
- Multiparameter Flow Cytometry: Phenotype T, B, NK cells post-treatment.
- Cytokine Multiplexing: Assess innate immune activation patterns.
- Vector-Associated Testing (AAV, LNP, LV)
- Biodistribution & Shedding Studies: qPCR/dPCR for vector genomes in tissues and fluids.
- Residual DNA Testing: Detect plasmid or host cell DNA per regulatory guidelines.
Why Partner with Accelevir
- Integrated Molecular + Immunology Workflows: Genotypic and phenotypic endpoints from a single, small-volume sample.
- Regulatory Alignment: CLIA-certified, GLP-capable laboratory delivering IND- and BLA-ready data.
- Deep Virology & Vector Expertise: Proven track record in AAV, LV, and other vector systems.
- Cross-Species Capability: Support for mouse, NHP, and human samples.
- BSL-2+ Containment: Safe handling of infectious samples and viral vectors.