Successfully bringing a gene therapy to market requires a deep understanding not only of the science, but also of the specific regulatory needs unique to gene therapies. Precision delivers leadership in regulatory strategy, specialization in immunogenicity studies and rare disease trials, and the expertise to effectively coordinate both a therapeutic and companion diagnostic strategy in parallel.
Industry-leading expertise in biomarker assays. Our tailored assays include immunogenicity, cell-based neutralizing antibody assays, and viral tissue distribution via ddPCR. We also offer AAV-based assay support from preclinical through clinical trials under all applicable quality systems.
Full support for rare disease trial execution: Gene therapies often target small patient populations, presenting unique challenges. We design trials with a patient-centric approach and utilize advanced statistical methods to reach small populations and meet complex endpoints. Our expertise is reflected in a 100% data submission acceptance rate.
End-to-end companion diagnostics and regulatory process experience: We develop and execute strategies for assays, trials and CDx development in tandem, an approach necessary to ensure rapid time to market. In addition, we offer fully-compliant (CLIA) cell-based NAb assays for CDx and commercial use.
Delivering evidence at every critical inflection point