Skip to main content

Currently Skimming:

Emerging Technologies and Innovation in Manufacturing Regenerative Medicine Therapies: Proceedings of a Workshop - in Brief
Pages 1-15

The Chapter Skim interface presents what we've algorithmically identified as the most significant single chunk of text within every page in the chapter.
Select key terms on the right to highlight them within pages of the chapter.


From page 1...
... medicine therapies. The discussions focused on the Since 2018 the number of regenerative medicine therapy role of emerging technologies, evolving manufacturing product developers has increased from 900 to at least approaches, regulatory considerations, and diverse 2,700 globally, the number of gene and cell therapy partnerships needed to deliver these therapies to clinical trials has increased from around 1,000 to 1,600, patients, said Scott Steele, senior advisor with the and the number of patients treated with chimeric antigen Center for Biologics Evaluation and Research (CBER)
From page 2...
... Likewise, the build partnerships and promote technology transfer. distributed manufacturing model can introduce further quality control challenges.4 OPENING KEYNOTE AND REFLECTIONS ON REGULATORY AND MANUFACTURING CONSIDERATIONS The hope is that automated and closed system With the growing use of CRISPR-Cas9, the regenerative manufacturing of cellular therapy products can address medicine field is likely to see an increased development some of these current manufacturing obstacles, said of cell-based gene therapies, said Peter Marks, director Marks.
From page 3...
... Academic centers around the country serve as de facto contract manufacturing Marks encouraged developers to take advantage of organizations, producing cellular therapy products for early, nonbinding regulatory meetings with FDA to multiple sites, because of their ability to rapidly modify discuss nonclinical studies, manufacturing, and clinical and revalidate production processes during phase I development plans. Opportunities to do so include Initial clinical trials, said Nikiforow.
From page 4...
... knowledge may limit the field's ability to accelerate Regulatory Perspective on Decentralized Manufacturing translation or decentralize manufacturing. A federated FDA/CBER has not yet established regulatory guidance learning model, like the Machine Learning Ledger or policy for cell and gene therapies manufactured using Orchestration for Drug DiscoverY (MELLODDY)
From page 5...
... said Lombardi. FDA has ongoing initiatives to support advanced DECENTRALIZED MANUFACTURING AS A POSSIBLE STRATEGY TO manufacturing, including the framework for regulatory ADDRESS PRODUCTION AND SUPPLY CHAIN CHALLENGES advanced manufacturing evaluation (FRAME)
From page 6...
... By developing a different set of percent, resulting in increased product quality, said analytical tools, Resilience may both reduce the amount Gerlinghaus. Specifically, throughout 2023, Cellares has of time required to release a product and gain more been manufacturing CAR-T cell therapies representative insights into critical quality attributes of a product, of those currently FDA approved, with critical quality which could correlate with improved patient outcomes, attributes far exceeding the release specifications for he said.
From page 7...
... However, recent advances in AI-powered Addressing Biology through Mathematics to Enable Cell control algorithms, optics for bioprocessing, and Manufacturing fluidics have enabled Cellino to develop an autonomous, The biology behind biomanufacturing processes can be closed, and modular platform for manufacturing these controlled if the mathematics dictating the events in cells. Such a cassette-based manufacturing platform is these processes are better understood, said Jan Jensen, important as manufacturers consider how to provide founder, chief executive officer, and chief scientific local accessibility to patients, said Saklayen.
From page 8...
... functional cells from individual donors. Evolution of ML and AI Technologies and Their Regulatory Impacts The Path to Automation of Manufacturing: A Case Study Considering the recent evolutions of ML and AI and their A case study in which an automated platform was used effect on manufacturing regenerative medicine therapies, to manufacture an implant for retinal pigment epithelial Saklayen acknowledged the role of the self-driving car (RPE)
From page 9...
... One problem confronting the field is IMPLEMENTING NEW MANUFACTURING STRATEGIES THROUGH PARTNERSHIPS AND INNOVATIVE TECHNOLOGY TRANSFER the large spectrum of variants within a single condition, Industrialization of Cell Therapies through Shared Manufacturing making a disorder appear rare and requiring more Platforms tailored gene therapies. For example, at least 114,189 While cellular and gene therapies are more complex than individuals globally have been diagnosed with a specific traditional pharmaceuticals, the regenerative medicine primary immunodeficiency defect such as the 450 known field can learn much from the industrialization of these single-gene inborn errors of immunity (Quinn et al., large molecule therapies, said Jens Vogel, senior vice 2022)
From page 10...
... One pathway is rooted in the optimization of vector generation and gene expression for adeno- Enabling Affordable and Accessible Advanced Medicines associated virus gene therapies. The second pathway The current pricing of approved cell and gene therapies focuses on establishing a publicly available playbook with is unsustainable for insurance companies and health streamlined manufacturing, preclinical, and regulatory systems and unfeasible for low- and middle-income processes to enable safe acceleration of adeno-associated countries, and it restricts access particularly for virus gene therapies.
From page 11...
... BOX 1 Challenges and Opportunities in Manufacturing Regenerative Medicine Therapies as Summarized by Graphic Recorder Wade Forbes and Presented by Individual Speakers Challenges • Lack of data sharing between manufacturing entities. (Lombardi, Marks)
From page 12...
... This list is the rapporteurs' summary of points made by the graphic recorder Wade Forbes and individual speakers identified, and the statements have not been endorsed or verified by the National Academies of Sciences, Engineering, and Medicine. They are not intended to reflect a consensus among workshop participants.
From page 13...
... • Employing decentralized manufacturing models, including distributed and point-of-care models, for autologous cell-based therapies, and centralized manufacturing models for allogenic cell-based therapies. Decentralized manufacturing can streamline the production logistics and reduce vein-to-vein time, while centralized manufacturing can result in economies of scale and having off-the-shelf products available.
From page 14...
... Reducing risks and delays in the translation t cell slot allocation: A multi-institution experience. of cell and gene therapy innovations into regulated Transplantation and Cellular Therapy:225-258.
From page 15...
... Leslie Sim, National Academies of Sciences, Engineering, and Medicine, served as the review coordinator. SPONSORS This workshop was partially supported by contracts between the National Academy of Sciences and Advanced Regenerative Manufacturing Institute; Akron Biotech; Alliance for Regenerative Medicine; American Society of Gene & Cell Therapy; Burroughs Wellcome Fund (Grant No.


This material may be derived from roughly machine-read images, and so is provided only to facilitate research.
More information on Chapter Skim is available.