Cell and Gene Therapies

Three topics are being presented throughout three sessions.

Viral Vector R&D and Manufacturability

Sunday, August 13 from 8 am – 12 pm PST in Foothill D (hybrid)

Gene & cell therapies have gained significant interest and increased momentum in the biopharmaceutical industry as viable and effective treatments for a wide array of diseases. With recent and upcoming clinical readouts and commercial launches, ongoing clinical trials, growing involvement from larger biopharmaceutical companies, increasing industrial collaborations with academia, and the formation of new start-ups, the field of gene & cell therapy promises to be a major player and potential game-changer for 21st-century medicine. While demonstrating great promise, these therapies are still relatively new to the biopharmaceutical manufacturing industry and require further study to reach the depth of understanding typically associated with established biologic therapeutics. Similarly to what was done 20 years ago for biologics, we are starting to see the emergence of platform processes for gene therapies and viral vectors that hold great promise in improving scalability and reducing time-to-clinic for these groundbreaking therapies. In that spirit, this session calls for abstracts focused on the development and production of Gene Therapies and Viral Vectors, with a focus on platform technologies in various phases of development from both academia and industry.

CGT R&D and Manufacturability

Sunday, August 13 from 2 – 6 pm PST in Foothill D (hybrid)

Cell therapies have demonstrated their efficacy to treat or be curative in oncology and non-oncology applications. Multiple avenues have been explored in the cellular therapy space to increase the potency but also simplify the process of generating cell therapy products, for example, CAR-T, TCR-based therapies, and other gene-edited cells such as engineered NKs. This session will encompass various aspects of cell therapy process development including new rapid manufacturing processes in the autologous and iPSC approaches in the allogeneic cell therapy manufacturing space. This would include advances in the isolation, expansion, gene modification, media optimization, process scale-up, analytics, and characterization of cell-based therapy products. Topics covering the impact of clinical pre-treatments and the cellular heterogeneity of the starting material on drug product potency and some of the control strategies to reduce this impact are invited. Additionally, novel drug products, cryopreservation, and delivery strategies for in vivo use along with improved or novel viral vector production systems will be included in this session. Papers relevant to these topics are highly encouraged, including those focusing on novel process improvements, control and optimization strategies, equipment and reagents design, and characterization.

Cell Therapy R&D and Manufacturability

Monday, August 14 from 8 am – 12 pm PST in Foothill E (hybrid)

Gene-modified cellular therapies achieved through viral transduction, such as CAR-T cells and gene-modified CD34 cells, have reached commercialization stages (e.g., Kymriah, Yescarta, Tecartus, Zynteglo, Strimvelis) with unprecedented clinical response rates and durability in oncology and complete correction of severe hematologic diseases. The early success of these therapies inspired a deluge of investment in the field, including affinity-modified TCRs for targeting solid tumors and intracellular antigens, along with gene-editing approaches via CRISPR/Cas for allogeneic adaptive T cells and hematologic diseases and gene therapies based on an AAV viral vector. Production of these therapeutic cells present diverse challenges in the manufacturing process. In this session, we will cover cell therapy chemistry, manufacturing, and control (CMC) and regulatory challenges regarding product characterization with in-process, lot release specification establishment, comparability studies, process validation, and clinical safety and efficacy.