AMP Cell-Based Therapy Consortium

Overview

The AMP Cell-Based Therapy concept proposes a new public-private partnership effort focused on cellular therapies, leveraging the model of the ongoing AMP Bespoke Gene Therapy Consortium. Given the similarity of goals for technology development of cellular therapies and gene therapies, of the industry and academic partners, and of the diseases themselves, launching a cellular therapies effort using the framework of the existing BGTC research plan infrastructure will accelerate the AMP design phase and allow overlapping of industry and scientific expertise between the programs. Like the BGTC’s efforts, a future Research Plan for an AMP in cell-based therapies for regenerative medicine is expected to detail the overall scientific and strategic objectives and present the basis for operationalizing a parallel effort that will focus on translational biology, manufacturing, regulatory processes, and clinical trials design for regenerative medicine technologies and therapies. The development of a public-private partnership will facilitate access to potentially transformative cell-based treatments, develop a regulatory pathway that leverages non-clinical and manufacturing data from one application to another. These efforts would streamline product development, reduce costs, and encourage investment in future therapies.

The AMP – CBTC will address key unmet needs through establishing a precompetitive partnership to address 2 general areas of interest: 1) Basic biology translational implications for the targeted therapeutics and 2) Advancing access to regenerative cell therapy technologies for clinical applications. The current knowledge gap in finding, characterizing and growing a cell type owing to insufficient understanding of its basic biology is limiting clinical translation. Investing in robust clinical trials held to a high standard of safety and efficacy, and the opportunity to develop streamlined preclinical and clinical testing processes will provide the necessary evidence to support clinical use and scale up production. A key focus of this consortium will be manufacturing given the gaps and challenges that still persist despite the significant advances in the field. These gaps include overcoming roadblocks in QC (quality and potency) for end-to-end closed manufacturing systems, developing/optimizing AI methods for automated production and quality control with set parameters to predict safety and efficacy. Additionally, there is a need to shift to open-source manufacturing with IP on the constructs and not the processes, to identify ways to shorten CMC, and to standardize qualification of raw materials and consumables.

The AMP CBTC Design Phase launched on October 27, 2023.