Cell and Gene Therapies

Five sessions are available for abstract submission.

Platform Approaches & Technologies

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.

Process Development & Optimization

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 CMC & Regulatory Challenges

Gene-modified cellular therapies (CT) 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, durability in oncology and complete correction of severe hematologic diseases. These early successes pave the way for exploration of other CT approaches such as affinity modified TCRs for targeting solid tumors, intracellular antigens and CRISPR/Cas gene edited allogeneic adaptive T cells etc. With the maturation of CT field, the chemistry, manufacturing and control (CMC) perspectives of these cell therapeutics still presents diverse challenges, and the regulatory landscape has been tightened by global regulatory agencies compared to those days when CTs were still in the proof-of-concept stages. In addition, the global regulations are not aligned thus adding additional complexity. In this session, the presentations will focus on the current and exciting progress and associated challenges of Advanced Therapies from both CMC and regulatory perspectives. The relevant topics include but are not limited to product specification setting and release, analytical characterization and comparability, process characterization and validation, raw and starting materials management, clinical safety and efficacy, fast to market (F2M) CMC strategies and considerations.

Viral & Non-Viral Vector Gene Therapy CMC & Regulatory Challenges

Viral and non-viral vector based gene therapies, although promising, are still in their early stages of development. Our understanding of these novel modalities remains limited compared to more established therapeutic approaches. This knowledge gap poses challenges for manufacturers aiming to create well-characterized, controlled manufacturing processes that meet regulatory standards.
In that spirit, this session is calling for abstracts from both industry and academia. Topics of interest include various CMC (Chemistry, Manufacturing, and Controls) strategies to efficiently bring these therapies to patients, phase-appropriate development strategies, process characterization (including critical process parameters and quality attributes covering full, empty, and partial capsid content, residual HCPs, nucleic acids), strategies and analytical techniques for early circumvention of safety concerns in clinical trials, process intensification and continuous processing to reduce costs, process validation, control strategy including the use of PAT and other in-process controls, approaches to select starting materials and reduce cross contamination. The session is also interested in big data (omics), mechanistic modeling, machine learning and AI driven process understanding.

Advanced Therapies Medicinal Products

Advanced Therapy Medicinal Products (ATMPs), comprising cell therapies, gene therapies, and engineered tissues, have emerged as powerful tools for patients with unmet medical needs. Significant research and investment in certain cell and gene therapy modalities has led to exciting clinical results and successful approvals, as well as substantial definition of platform manufacturing processes for those product types. However, this highly innovative field has also supported the advancement of many alternative modalities, which require widely varying manufacturing approaches and present unique CMC challenges. This session invites abstracts that focus on the development and production of novel ATMP technologies that don’t fit neatly into the existing cell and gene therapy frameworks, especially different drug delivery vehicles (novel viral and cell modalities, non-viral vectors, LNPs, exosomes etc.), engineered tissues, and other modalities where there is not yet an established manufacturing platform. We also encourage case studies illustrating unique challenges which must be overcome to develop and manufacture these innovative product types. Abstracts that highlight innovative solutions for characterization, manufacturing, and scalability of ATMPs as well as clinical trial, safety and regulatory framework and guidelines are also encouraged.