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Regenerative Medicine and Advanced Therapies
Summary
Advanced therapies, particularly those under the Regenerative Medicine and Advanced Therapies (RMAT) designation within the U.S. or as part of the Advanced Therapy Medicinal Products (ATMP) in many other regions, have shown promising clinical efficacy and are changing the paradigm for treating diseases and injuries. Clinical translation and patient access to this broad class of therapeutics requires better defined and characterized products and more robust, reliable, and cost-effective manufacturing processes.
The NIST Advanced Therapy Program supports the growing industry as well as the broader ecosystem by addressing manufacturing, characterization, and testing challenges. Our efforts include 1) developing measurement assurance strategies and innovative measurement solutions, 2) convening stakeholders, including regulatory agencies, to identify industry-wide challenges and pre-competitive solutions, and 3) leading and contributing to the development of global documentary standards and reference materials. Together, we aim to accelerate research and development to commercialization through biometrology, data, and globally harmonized practices and standards.
DESCRIPTION
RMAT Laboratory Programs – Our programs leverage BBD core capabilities in biometrology, engineering biology, as well as state-of-the-art automation, machine learning (ML) and artificial intelligence (AI) to address unique measurement challenges associated with advanced therapies.
Our team is focused on quantitative measurement of quality attributes for cells, cellular systems, and other biological entities (e.g., DNA/RNA, viral vectors and other delivery vesicles, and genome editing tools), and their complex and dynamic interactions.
Our laboratory programs provide the scientific foundation and data underpinning the development of documentary standards and reference materials.
- Click here for an example of how NIST is supporting cell therapy
- Click here for an example of how NIST is supporting gene therapy
- Click here for an example of how NIST is supporting tissue engineering
Measurement Assurance – A major focus at NIST has been the application of quality tools to improve the performance of complex biological measurements. As part of this focus, we recently launched the NIST Prototype Cell Assay Measurement Platform (P-CAMP) for high quality assay validation and innovation. This unique platform technology enables automated multimodal analysis of large parameter spaces and was designed to guide the development of measurement assurance strategies for assays used for characterization and testing of biological products and processes. The system design is centered on automation that can set up thousands of experiments for the purpose of identifying sources of variability in bioassays, designing fit-for-purpose reference materials, and generating specifications that assure confidence in an assay measurement system.
Next Generation Metrics for Cell-Based Therapies and Regenerative Medicines – One of the most challenging aspects of cell-based therapies is the characterization of cell products vis a vis their functional activity, and how those might be influenced by handling and manufacturing processes. One approach we are taking involves the creation of reporter cell lines which are engineered to produce fluorescent reporter molecules when important control pathways are induced in cells. This approach will allow real time monitoring of a manufacturing protocol.
Standards Development – Standards are critical in the development and commercialization of new technologies and facilitate national and international commerce. Standards also have an important role in ensuring product quality and consistency as well as streamlining regulatory approval.
Under the previsions enacted under the 21st Century Cures Act (U.S. Public Law 114–255), NIST continues to work with the U.S. FDA, the Standards Coordinating Body, the NIH, and other stakeholders to develop standards for advanced therapy manufacturing and testing. NIST is developing documentary standards as well as reference materials to support advanced therapy.
- Click here for an overview of standards development principles
- Click here to access SCB’s Regenerative Medicine Standards Landscape report
- Click here to access FDA’s Standards Development Guidance for Industry
- Click here to access FDA's Potency Assurance for Cellular and Gene Therapy Products Guidance
Stakeholder Engagement – NIST works with industry, academia, and other agencies to identify and develop measurement solutions. Learn more about our strategic partnerships, interagency coordination via MATES, collaboration with the U.S. FDA, and the NIH Regenerative Medicine Innovation Project.
Other collaborators include: