Analytical Development: Nonclinical Evaluation of Candidate Potency Assay
Potency assays should reflect efficacy-associated in vitro and in vivo activity. The client requested assistance from DHC with the design and use of nonclinical studies to demonstrate biological relevance of their lead candidate in vitro potency assay for a clinical stage cell therapy product. This interaction demonstrates a common industry overlap between the service areas of analytical and preclinical/nonclinical development.
- The goal of this project was to obtain proof-of-concept in vivo data to support the validity of the client’s in vitro potency assay and to set assay acceptance criteria that could be used to evaluate the lot-to-lot consistency of the client’s drug product.
- Dark Horse’s extensive experience in nonclinical modeling and in vivo mechanistic studies provided the necessary know-how to identify a viable strategy for establishing the physiological relevance of the client’s in vitro potency assay.
- The first step was to evaluate existing nonclinical data and identify potential strategies to demonstrate causality between the cell therapy product’s activity in the candidate in vitro potency assay and the product’s in vivo therapeutic activity.
- Once an optimal strategy had been selected, DHC provided the client with a detailed design of the proof-of-concept in vivo nonclinical study, including key outcome measures and a sampling plan that would allow the program to advance beyond correlation and demonstrate a causative role of the product’s hypothesized therapeutic mechanism of action.
The outcome of this engagement was the identification of the strategy necessary to verify the physiological relevance of the lead candidate potency assay for a clinical stage cell therapy product.
- Process Development/Comparability Studies
- Regulatory Support
- Manufacturing Support