Advanced therapy medicinal products (ATMPs) and cell and gene therapies (C&Gs) are based on genes, cells, or tissues delivered to patients to provide a therapeutic benefit, based on a specific target of interest. For ATMPs, the therapy is a cell, engineered tissues, or the manipulation of the patient’s genome. This is in contrast with current manufacturing processes for compounds that are synthetically derived (i.e., small molecule) or proteins or peptides expressed by cellular systems (i.e., large molecule biopharmaceuticals). The development, regulatory path, facility design, qualification, and manufacture of ATMPs present significant challenges to manufacturers, engineers, and suppliers. The GMP regulations are evolving as novel processes are presented and manufacturing paradigms are being tested. This fuels a need for sharing experiences and good practices for ATMPs to help our members find solutions that enable patient health.
New Community of Practice
ISPE has elected to form an ATMP Community of Practice (CoP) to expand the industry’s and ISPE’s body of knowledge. The new community of practice will leverage the manufacturing, analytical, and regulatory knowledge of recognized subject matter experts (SMEs) to raise issues and identify solutions that result in:
- Manufacturing (from vial to vial) processes and techniques that reduce cost of goods with consistent product quality. This includes the development of a robust control framework to ensure product quality, including CMC/GMP and unique equipment challenges.
- Robust analytical and stability methodologies, and strategies for raw materials, in-process, and accelerated product release.
- Identifying and determining solutions for complex regulatory issues that, when resolved, will result in accelerated speed to market.
New Focus On ATMPS
One of the first focus tasks of the community of practice will be the finalization and launch of the new ATMP–Autologous Cell Therapies Guide, focusing on manufacturing facilities development and design. The guide is expected to be published by the end of 2021. The purpose of this guide is to address facility engineering issues and how to provide cost-effective facilities to ensure that products of the highest quality are consistently manufactured. Where nonengineering issues are involved (e.g., microbiological topics and operational issues unrelated to the facility), information will be included to show engineers the importance of such topics and the impact they have on facility design.
The new guide acknowledges that the term ATMP is quite broad, including allogeneic and autologous cell therapies, gene therapies, personalized vaccines, and tissue engineering, and that these are emerging therapies utilizing rapidly evolving technology and equipment. In recognition of this, the guide focuses primarily on autologous cell therapies intended for parenteral use while providing content that may be applicable to other types of ATMPs. For example, segregation principles for individual patient autologous cell therapies, addressed in this guide, may also be useful for a personalized vaccine facility.
The guide’s launch will also provide opportunities for engagement in conferences for both ISPE and other industry organizations with the guide’s content.
Publication of the guide will be the first content contribution from the ATMP community of practice. The next guide about ATMPs will be on the manufacture of allogenic-based cell therapy products. Work will begin on the second guide in the first quarter of 2022.
Another focus area for the community of practice will be on the development of training material for the launch of new ISPE professional training. The initial goal will be a course that focuses on the new guide, targeting autologous cell therapy manufacturing. Future materials will address allogenic manufacturing and potential advances in specific areas such as supply chain management and regulatory compliance.
Since experiences with ATMPs are rapidly growing and evolving, the community of practice will develop webinars, iSpeak blog posts, and conference presentations to build the base of knowledge within the community. The community of practice will be closely collaborating with other ISPE communities of practices where there is a mutual interest. For example, practices around multiproduct facilities handling active viruses are applicable to both the ATMP community of practice for viral vectors and the Biotechnology community of practice for vaccines. The collaboration will also include assisting with developing ATMP tracks at ISPE conferences in 2022.
How to Get Involved
The ATMP Community of Practice is looking for members to join, share, and expand the body of knowledge within ISPE and the industry. Registration for the community of practice is available online by logging into ispe.org, selecting “My account,” choosing the “MY INTERESTS AND COMMUNITIES” tab, then checking “ATMP” under the “Communities of Practice” at the bottom of the page.
For questions about the community of practice, contact Erich Bozenhardt, ATMP community of practice Chair.
