Gene therapy can potentially treat or cure a wide range of disease entities and is being used mainly in clinical trials and certain cases as FDA-approved therapies. The starting material for these treatments is often collected via apheresis procedures where a relatively large number of hematopoietic progenitor cells (HPCs) are needed in a short period of time to undergo ex vivo manipulation to generate a therapeutic drug product. This session will provide a brief overview of gene therapy, using sickle cell disease, beta-thalassemia, and active cerebral adrenoleukodystrophy (CALD) as examples to illustrate strategies for gene editing/introduction. We will describe challenges with the mobilization and collection of HPCs and potential solutions for optimization of mobilization/collection of HPCs and discuss the risks and benefits of gene therapy as well as strategies to measure success.The audience for this session includes physicians, laboratory and quality personal involved in HPC mobilization and collection, apheresis centers, and blood banks that may be processing and shipping cells for manufacture.
Cryopreservation preserves hematopoietic progenitor cells (HPC) for future use, but the freezing and thawing processes can impact viability. To ensure sufficient viable cells for downstream applications, post-thaw viability testing is essential. This session focuses on standardizing post-thaw viability testing methods, including thawing procedures and viability testing, with an emphasis on CAP proficiency testing and staff training.
Transfusion physicians, technologists, managers, and support staff need at least a basic understanding of billing and coding procedures. Those responsible for maintaining appropriate billing and coding must keep up with the constantly changing expectations for coding and billing. This session is designed to provide a comprehensive review of current coding and reimbursement information, including critical updates, and share best practices and case studies from hospitals. AABB staff will also discuss Medicare advocacy and resources to help hospitals ensure they receive appropriate payment.
This popular session consistently draws a large audience and provides an opportunity for the regulatory experts with the Food and Drug Administration (FDA) and the Centers for Medicare & Medicaid Services/Clinical Laboratory Improvement Amendments representatives to respond to member questions about new rules, regulations, and guidance. The session also allows the FDA to present their current thinking on policies, regulations, guidance documents, and inspection programs relevant to their oversight of blood and human cells, tissues, and tissue-based products (HCT/P) programs. Questions for the session are submitted in advance to email@example.com.
Oral abstract session featuring the latest research on Emerging Blood and Biotherapy Products
Apheresis and cellular therapy procedures in pediatric patients pose many clinical challenges. Significant modifications to adult apheresis protocols & practices are required while performing pediatric apheresis. Some procedures that may feel routine in adult patients may be daunting to the providers if they do not routinely treat children & infants. Vascular access, anticoagulation, volume variation & priming are just a few of the common challenges faced while performing pediatric apheresis.In this session, speakers will share their institutional experiences & provide insight into procedure modifications & management of complications in pediatric patients. Specific examples for managing stem cells and MNC collections, extracorporeal photopheresis, plasma exchange, and other apheresis procedures, including mitigation of adverse events, in patients as small as 4 Kg will be shared. This session will review 17 patients under 10 Kg who underwent successful stem cell collections. Details of managing tandem apheresis procedures (with Extracorporeal Membrane Oxygenation & Continuous Renal Replacement Therapy) will also be discussed. After attending this session, the audience should have gained knowledge of and comfort with safely performing apheresis in pediatric patients.
This session proposes a comprehensive approach to workforce development in cell processing labs, which includes obtaining the internationally recognized AABB Certified Advanced Biotherapies Professional (CABP) credential. The credential covers seven critical domains in the cell processing field, including biotherapies in patient care, operations and equipment, quality systems, and regulatory environments. Obtaining this certification is crucial to enhancing employees' skills and knowledge in cell processing labs and demonstrating their commitment to the field. Training and Education Need Assessment: The first step in our workforce development plan is to comprehensively analyze the skills and knowledge required in cell processing labs. This analysis will identify areas for improvement and help us to tailor training and education programs to the specific needs of the cell processing field. Development and implementation of Training and Education Programs: Based on the results of our needs assessment, we will develop a range of educational tools and activities, including workshops, online courses, and conferences, to provide targeted training and education programs for employees in cell processing labs. Emphasis will be placed on hands-on training and practical real-world experience, with goals of enhancing skills, knowledge, and access to the latest knowledge and technology. Monitoring and Evaluation: With the scope of the CABP syllabus, regular assessments will be conducted to measure the effectiveness of the training and education programs and identify areas for continual improvement. This will help us refine our approach and ensure that our workforce development plan remains relevant and up-to-date to attract and retain an adequate workforce. This education session provides a comprehensive approach to workforce development in cell processing labs, which highlights the critical role of the AABB CABP credential, leading to increased efficiency and productivity.
Currently, six FDA-approved CAR-T cell therapies are available to patients, and at least two gene therapies for the treatment of rare and prevalent conditions. There are more than 2000 Cell & Gene Therapy (CGT) clinical trials worldwide, with 200 in Phase III, of which as many as 20 could be approved by 2025. For now, manufacturing most CGT drugs depends on the support of qualified academic medical centers, from collecting starting material by apheresis programs to handling, storing, and dispensing by specialized cell processing facilities. As manufacturing of new CGT grows exponentially, clinical centers are being asked, on top of already limited resources, to handle numerous protocols from multiple providers with different processes, documentation, data management systems, and requirements. Moving this number of new CGT drugs from clinical trials to commercialization is beyond the current capability of academic medical programs. This session will review the role of medical centers in the manufacturing of advanced CGT. It will describe the specific challenges faced by cell therapy collection and processing facilities with the increased number of available therapies. Practical solutions, such as using uniform procedures for onboarding and centralization of the oversight of CGT will be presented. We will discuss the need for collaboration with manufacturers and regulatory agencies for the standardization and harmonization of CGT manufacturing and the need for the clinical community to prepare for the approaching CGT tsunami.
Blood banking and product safety testing has evolved over decades to ensure safe products and maximize testing efficiency. Innovations in the space have allowed for qualitative analysis of products and faster analytical processes based on scientific and technical discoveries. The Advanced Therapies development space is newly emerging, with very few licensed products, all in a narrow range of classes. In recent years, we have seen products stall at the licensure finish line because of an inadequate set of characterization and potency assays. In this space, 80% of the questions related to licensure filings are related to the CMC section – production and analytical variables. As the range of product types expands, the complexity of these analytics and the potential roadblocks to product licensure can only be expected to increase. These analytical development and testing challenges affect developers of Advanced Therapies throughout the development and production cycle – first starting materials, moving through in-process testing, and finally, focusing on finished product testing. Further, multiple levels of testing are required, including safety, quality, identity, purity & potency, with potency being especially challenging. This session will cover the critical burden of analytical development and testing in alleviating roadblocks to the licensure and commercialization of Advanced Therapy products. It will also look at the value of a strong analytical partner throughout development and production. Topics covered will include the importance of early analytical development, criticality of potency assays, description of newly developed flexible potency assay platforms, qualification of traditional donor screening assays for use in cell therapy safety testing, impact of a robust analytical development plan on successful licensure discussions with regulators and insights into possible future analytical requirements for cell and gene therapy products.
The Emily Cooley Memorial Award & Lectureship began as a lectureship in 1963 and was designated as a Memorial Award in 1983. The award recognizes an individual who has demonstrated teaching ability and has made a major contribution to the field of transfusion medicine or biotherapies. The 2023 Emily Cooley Memorial Award will be presented to Thomas Spitzer, MD