Quality Assurance for Biomanufactured Regenerative Medicine Products (Qual-Regen)

The JPC-8/CRMRP, DHA RDA, and OASD(HA) have identified a need for regenerative medicine prototype development efforts and manufacturing technologies. Current Good Manufacturing Practice (cGMP) quality is a requirement by the Food and Drug Administration (FDA) and European Medicines Agency to provide patients with clinical-grade products that are safe and have defined quality characteristics. However, process standardization and robust manufacturing

techniques are lacking in regenerative medicine, which will continue to impede progress in advancing regenerative medicine based technologies and treatments toward the clinic. This is likely due to the immaturity of the regenerative medicine field, which could benefit from the development and advancement of many manufacturing capabilities.

This RPP focuses on quality management of biomanufacturing processes and/or development of a prototype for a universal biomanufactured product or process. Applications should clearly state innovative solutions for the biomanufacturing of regenerative medicine products and regulatory strategies to obtain FDA clearances or approvals, if appropriate. This RPP offers the opportunity for industry funding to join the project as it leads to stronger leveraging of Government contractor collaboration. While not a requirement, Offerors are strongly encouraged to include Cost Share as appropriate.

The major objective of this RPP is to overcome current challenges in biomanufacturing of regenerative medicine products and enable successful cGMP manufacturing and clinical translation of regenerative medicine based therapies. To accomplish this we are seeking solutions for quality management of manufacturing processes or prototypes for cell, tissue, or organ bioengineering technologies. This RPP specifically targets solutions to the following Focus Areas:

  • Processing technologies for quality management of acellular matrices or Stem Cell (SC)-derived cell based therapies
  • Manufactured universal acellular matrix
  • Innovative manufacturing process for SC-derived cell based therapies

The research project award recipients were selected from the Offerors who responded to MTEC’s Request for Project Proposals (20-07-Qual-Regen).

NuCressTM Intelligent Factory in a Box

Project Team: NuShores Biosciences

Award Amount: $2.82M

Project Duration: 34 months

Project Objective: The design, development and demonstration of a fully automated intelligent in-process quality monitoring and control production system for 3D scaffolds that could enable a factory in a box vision (FIAB) is the key goal of this project. We will prove the feasibility of a fully automated closed production system (FIAB) with real-time intelligent system quality monitoring and predictive faults, failures and reporting features for NuCress™ bone filler scaffold in production quantities, sizes and shapes. We will use a top-down system-engineering process for development of our envisioned hands-free, closed environment, automated and intelligent production system for medical devices. Three (3) generations of an automated quality and production system will be developed and an investment grade roadmap to full expression of the FIAB will be produced and presented.

Potency Assay Development for MSCs in Regenerative Medicine

Project Team: Sentien Biotechnologies

Award Amount: $2.39M (additional cost share = $2.23M)

Project Duration: 29 months

Project Objective: A consortium of leading MSC developers, Sentien, GenCure and RoosterBio, aim to create a large-scale biomanufacturing process, culminating in a potency assay qualified with human clinical data. This requires deep characterization of critical process parameters and critical quality attributes. GenCure and RoosterBio will lead the initial effort, with 3D bioreactor expansion of commonly used MSC sources in regenerative medicine: bone marrow, umbilical and adipose. Subsequently, Sentien will test these expanded cells in a high-throughput lab-scale bioreactor in vitro to develop putative potency targets. Work will be completed by driving these learnings through clinical stage. Sentien, which expects to be running human clinical trials in both acute kidney injury and acute respiratory distress syndrome, will contribute biomarker data from the clinical-scale bioreactor and patient samples. Testing the potency hypothesis against the human clinical samples will form the underpinnings of a true potency assay.

Quality Management Processes for hiPSC-derived Vascular Cell Therapies

Project Team: Cedars-Sinai Medical Center

Award Amount: $3.12M (additional cost share = $1.54M)

Project Duration: 36 months

Project Objective: The proposed application will address the aims of the MTEC‐20‐07‐Qual-Regen Request for Project Proposals by (i) developing an automated platform to enable efficient and cost-effective iPSC expansion; (ii) validating an automated and non-destructive imaging system to inspect the quality of these iPSC lines; (iii) advancing iPSC preservation technologies; and (iv) establishing quality controlled manufacturing processes for iPSC-derived endothelial cells (iECs) for vascular regenerative cell therapies.