What's in YOUR capsids?
They may be full...but full of what?
Gene therapy has revolutionized medicine over the past decade, enabling doctors to offer life-saving treatment options to patients with devastating and rare diseases. As success stories accumulate, there is growing demand for expansion of gene therapy to reach more patients and to address more common conditions.
However, as gene therapy production scales up and becomes increasingly customized, current industry standard quality control measures may be inadequate to fully address product purity. Recent media attention on patients dying while undergoing therapy will likely compel federal regulatory agencies to increase scrutiny of gene therapy products and impose stricter standards.
BioInfoExperts is leveraging our decades of experience in pathogen genetics to develop unique genetic characterization assays that enable an accurate and granular assessment of gene therapy product purity. Our reports include quantification of length variation, contaminating DNA, and the single nucleotide mutation rate. Our goal is to help researchers and CDMOs optimize gene therapy products to improve clinical outcomes.
Single Genome Sequencing
We combine the gold-standard Sanger sequencing with limiting-dilution amplification, which enables us to obtain highly accurate sequences from individual vector genomes. This technique provides the rate and location of single nucleotide mutations along the genome. Our laboratory has used this method for two decades to characterize highly-diverse viruses such as HIV-1.
Next Generation Sequencing
NGS is becoming a more widely-used tool for characterizing vectors. However, NGS sequence data is notoriously difficult to process and interpret, and off-the-shelf analytics are likely to give misleading results. Our team is developing ways to address the challenges of this technology to maximize the accuracy and utility of AAV NGS data for routine quality control.
FDA Funded
We are recipients of the Food and Drug Administration Small Business Innovation Research (SBIR) funding for our AAV work and benefit from feedback from FDA working groups in developing our genetic methods. We are poised to assist the gene therapy industry to meet future regulatory standards that require more in-depth genetic characterization to ensure product safety.
Reporting Includes:
