Wakerbio offers specialized analytical services for gene therapy development, providing precise integration site analysis and quantitative off-target detection. Our platform supports critical safety evaluations by assessing vector integration patterns and unintended genetic modifications, aiding regulatory submissions.
Funding
Funding not disclosed
Founders
Product
Problem
The development of gene therapies necessitates rigorous safety evaluations to mitigate risks associated with vector integration and potential off-target effects. Ensuring the safety profile of these advanced therapeutics requires precise analysis of insertion sites and quantitative assessment of unintended genetic modifications.
Solution
Wakerbio provides specialized analytical services for gene therapy drug development, focusing on integration site analysis (ISA) and quantitative off-target detection. Our platform supports the critical safety evaluation phase by precisely assessing vector integration patterns and insertion mutation risks. We offer end-to-end solutions, managing the process from sample processing through to the final regulatory report submission. Our expertise is built upon validated methodologies, including LTA-PCR and TES systems, which have been instrumental in the successful regulatory submissions of multiple gene therapies to agencies like the FDA and EMA.
Target Audience
Our primary clients are biotechnology and pharmaceutical companies engaged in the development of cell and gene therapies (CGT), as well as academic research institutions and contract research organizations (CROs) requiring specialized safety and characterization services.
Features
- Integration Site Analysis (ISA) utilizing validated LTA-PCR and TES methodologies.
- Quantitative off-target detection for gene editing technologies such as CRISPR/Cas9.
- Vector copy number (VCN) quantification using probe-based qPCR assays.
- Comprehensive vector quality control services, including characterization, impurity quantification, and replication competency assessment.
- Next-Generation Sequencing (NGS) based assays, including AAVSEQ for AAV vector analysis and LENTISEQ for lentiviral vector analysis, to identify and quantify residual nucleic acid impurities and assess genome integrity.
- Dedicated bioinformatics analysis pipeline for genomic and transcriptomic data.
- GLP-compliant laboratory facilities with advanced instrumentation for robust data generation.
- End-to-end service offering from sample preparation and library preparation to data analysis and reporting.
- Expertise in supporting regulatory submissions with data packages compliant with FDA, EMA, and NMPA guidelines.