Analytical methods are crucial for process development and optimization, and later in supporting product manufacturing under GMP conditions. These methods assist in maintaining product quality, safety, and efficacy in the drug development process. Gene therapy and vaccine development encounter numerous analytical challenges that need to be addressed early in the process development stage.

Cornerstone® Analytics Services can assist with:

1. Comprehensive Toolset
2. Robust Analytical Methods
3. Cost-Effective Analytical Methods
4. Wide Range of Expertise
5. Regulatory Compliance

Process analytics requires a broad set of analytical tools to build the production process understanding and product knowledge necessary to advance gene therapies and vaccines through to the clinical stage.

We provide customized and innovative analytical methods to ensure the quality and safety of gene therapies and vaccines. The PATfix analytical system, which consists of expert software and multiple detectors such as UV, fluorescence, light scattering, pH, and conductivity, provides a platform for the implementation of customized analytical methods. In combination with monolithic columns of various chemistries, the PATfix system enables fast development and implementation of analytical chromatography for any drug substance.

Detailed sample characterization is possible with orthogonal analytical methods, which allow for exploration of both target molecules and sample impurities that need to be removed during the process of final product preparation. Depending on the chosen sample modality, complementary methods include techniques such as dPCR, capillary electrophoresis, mass photometry, flow cytometry, and cell-based assays.

A major hurdle in the development of robust drug manufacturing processes is the lack of rapid and reliable analytical methods. Understanding the impact of process parameters on the product’s critical quality attributes is often limited by the lack of well-established assays for novel therapeutic modalities.

Our analytical chromatographic methods are specifically developed for the identification and quantification of large biomolecules in complex mixtures. The methods are developed with a focus on speed, reliability and identification of a wide range of species in a single analytical run. Typically, the chromatographic methods’ analytical runtime is between five to twenty minutes. Comparison to lengthier established analytical methods assures comparability between results.

The high cost of gene therapies is significantly driven by the lack of rapid and reliable analytics for process development and monitoring in the drug manufacturing process.

Our PATfix system, in combination with CIMac columns, provides cost-effective and time-efficient analytical methods for various biomolecules, including AAVs, pDNA, mRNA, LNPs, and others. It only takes twenty minutes to determine the ratio of empty/full AAV capsids and only eight minutes to quantify all nucleotides, capping reagents, and mRNA in IVT reactions.

Our AEX method for AAVs significantly reduces cost and turnaround time when compared to conventional methods such as ELISA, PCR, ultracentrifugation, AGE, and PAGE.

Effective collaboration among researchers is essential for advancing the understanding of complex gene therapy processes and for developing robust innovative analytical strategies. In large companies, departmental silos often hinder collaboration, making it difficult to integrate diverse analytical methods. Smaller companies, on the other hand, may lack the broad expertise needed.

Our Analytical Development team can bridge collaboration and expertise gaps in gene therapy and vaccine analytics. We have conducted over 100 early-stage feasibility studies and successfully transferred more than 30 processes for pDNA, mRNA, AAV, and other virus to customers’ GMP environment.

Our strong pipeline of analytical methods development, including a recent focus on LNPs, keeps us at the forefront of analytical innovation. We collaborate with universities and the biotech industry to develop and refine our PATfix analytical platforms through extensive testing of a diverse range of samples. With over 200 publications and novel application notes published in the last ten years, we are deeply committed to advancing chromatographic biomolecules analytics.

Regulatory requirements for viral vectors are becoming increasingly stringent. As analytical capabilities improve, regulators demand more effective monitoring and characterization of drug products.

Our innovative methods are verified through validation protocols following FDA and EMA guidelines, which include assessing specificity, confirming linearity, and determining the Limit of Detection (LOD) and Limit of Quantification (LOQ). We ensure the specificity, accuracy, precision, and robustness of the developed methods. Sample stability is rigorously tested to ensure reliable performance for the entire range of process conditions. By adhering to the validation protocols, we provide effective monitoring and characterization of process and product consistency, quality, safety, and meeting regulatory demands.