Moving Beyond the Vanilla: Employing Intelligent Therapeutic Design and Manufacturing Automation to Develop Multiple Cell and Gene Therapy Flavors

CHAIR: Matthew Hewitt, PhD, Charles River Laboratories, United States

SPEAKERS:

• Dan Getts, PhD, MBA, Myeloid Therapeutics, United States
• Aaron Foster, PhD, Outpace Bio, United States

Attendees will learn about several high interest topics within the space of automation and CGT. The first, intelligent cell therapy therapeutic design enables cells to adapt and thrive in challenging environments such as the solid tumor microenvironment. Next, the session will address the shift from ex vivo cell therapeutics to in vivo using LNPs and RNA. One key consideration is how to achieve the desired level of tropism and access to impart significant clinical benefit. Lastly, as demand continues to outpace supply in the commercial cell therapy market, decentralized manufacturing will be discussed as a potential solution. Critical to this is understanding what is needed to establish a decentralized manufacturing network from an automation and digital perspective.

SESSION OBJECTIVES:

1. Discuss how therapeutic developers are employing synthetic biology to improve cell therapy functionality in oncology.
2. Understand the potential shift as well as benefits from ex vivo cell therapies to in vivo and how this may affect therapeutic access.
3. Understand how therapeutic developers can leverage automation in manufacturing to launch a decentralized manufacturing network to increase therapeutic access for patients.

Advanced Analytics: Harnessing AI and Cutting-Edge Technologies to Accelerate Cell Therapy Development

CHAIR: Marinna Madrid, PhD, Cellino, United States

SPEAKER:

• Dalip Sethi, PhD, MBA, MS, Terumo Blood and Cell Technologies, United States

This session brings together pioneers in advanced analytics to address the most pressing challenges in cell and gene therapy development. Attendees will gain insights into state-of-the-art imaging techniques, AI-driven data analysis, and genomic profiling methods that are reshaping the CGT landscape. The session will showcase how these technologies can be leveraged to enhance product characterization, streamline manufacturing processes, and develop more robust potency assays. 

SESSION OBJECTIVES:

1. Explore cutting-edge analytical technologies to enhance characterization and quality control of cell and gene therapies, addressing critical bottlenecks in product development and manufacturing.
2. Discuss how AI and machine learning can revolutionize analytics, leading to faster and more cost-effective product development.
3. Provide practical insights on implementing novel assays to improve product consistency, efficacy assessment, and regulatory compliance across all phases of CGT development.

New Approach Methodologies (NAMs) and Non-Animal Models: How does CGT move forward along with FDA Modernization Act 2.0 and 3.0?

CHAIR: Shannon Eaker, PhD, Xcell Biosciences, United States

SPEAKERS:

• Ludovico Buti, MSc, PhD, Charles River Laboratories, Netherlands
• Ankur Singh, PhD, Georgia Institute of Technology, United States

Most researchers want to minimize the use of animals in scientific experiments. Whether from the vantage point of a research, medical or federal funding organization, the reasons to discover alternatives are numerous. Over the last two years, the US has reached a tipping-point where all are accountable to reduce animal use within life science research.  The focus of this session is on 3D culture models (Spheroids/Organoids/Organ-on-a-chip/Tumoroids) as Non-Animal Models or New Approach Methodologies (NAMs). This is a sustainability initiative to reduce the need for animals and vivarium’s in life science research (FDA Modernization Act 2.0 and 3.0).

SESSION OBJECTIVES: 

1. Perspectives from Industry, Academia and Government on the current situation and strategies for using new approach methodologies and non-animal models.
2. Provide real-world examples of the new approach methodologies and non-animal models.
3. Highlight the inclusion and applications of AI and machine learning in new approach methodologies and non-animal models.

Building the Future: Multidimensional Strategies in Organ Regeneration

CHAIR: Julie Allickson, PhD, Michael S. and Mary Sue Shannon Director of Mayo Clinic’s Center for Regenerative Biotherapeutics; Otto Bremer Trust Director, Biomanufacturing and Product Development, Center for Regenerative Biotherapeutics; Associate Professor of Regenerative Medicine, USA

SPEAKER:

• Harald Ott, MD, MBA, United Therapeutics, United States

Attendees will gain insights into the transformative potential of organ regeneration through a multidisciplinary lens, featuring presentations from leading experts who are at the forefront of this exciting field. The session will address critical issues such as the challenges of scaling tissue engineering solutions, the regulatory landscape for new regenerative products, and the integration of novel technologies into clinical practice. By examining successful studies and emerging research, participants will leave with a deeper understanding of the current state of organ regeneration and the future possibilities it holds. This engaging session will not only inform but also inspire attendees, making it a must-see session at the International Society for Cell and Gene Therapy in New Orleans in 2025.

SESSION OBJECTIVES: 

1. Highlight Innovative Approaches: To explore and discuss groundbreaking strategies and technologies that are advancing the field of organ regeneration.
2. Showcase Successful Studies: To present case studies and research findings that illustrate the efficacy of current and emerging therapies in regenerative medicine.
3. Foster Collaboration: To create networking opportunities among attendees, encouraging partnerships and collaborations that can drive further innovation in organ regeneration.

Presenting Dendritic Cells: Clinical Trial Insights and Therapeutic Advances

CHAIR: Michael Gustafson, PhD, Mayo Clinic, United States

SPEAKERS:

• Hideho Okada, MD, PhD, University of California San Francisco, United States
• Keith L. Knutson, PhD, Mayo Clinic, United States

Dendritic cells represent a promising direction for cellular immunotherapeutics; however, substantial work remains to adapt them for clinical application. This session will focus on the advancement of autologous dendritic cell therapies, specifically their utilization in cancer treatment. Dendritic cells play a critical role in stimulating anti-tumor T cells to target various antigens, which enhances their effectiveness against the diverse cell populations present in solid tumors. Additionally, the rapid emergence of new technologies is poised to improve the potency and efficacy of dendritic cells for therapeutic purposes.

SESSION OBJECTIVES:

1. Summarize why dendritic cells continue to be a promising focus for cellular immunotherapy.
2. Identify the critical barriers that limit the efficacy of dendritic cell therapeutics.
3. Demonstrate the importance of bench-to-bedside-and back again research to improve dendritic cell therapies.

T Cells at the Forefront: Pioneering New Avenues in Solid Tumor Therapy

CHAIR: Michael Gustafson, PhD, Mayo Clinic, United States

SPEAKERS:

• John Mullinax, MD, FACS, Moffitt Cancer Center, United States
• Udai S. Kammula, MD, FACS, University of Pittsburgh, United States
• Sylvia Lee, MD, Fred Hutchinson Cancer Center, United States

Autologously expanded adoptive T cells and tumor-infiltrating lymphocytes present a significant opportunity to broaden the range of immunotherapeutic cell types that demonstrate strong tumor reactivity. Given the capacity of these cell types to target multiple antigens, their application appears highly promising. This optimism is supported by the recent approvals of lifileucel and afami-cel for non-CAR T cell strategies in treating solid tumors. Therefore, it is essential to address the challenges that hinder T cell potency within the tumor microenvironment to enhance the efficacy of T cell therapies for solid tumors.

SESSION OBJECTIVES:

1. Summarize the current state of ATC/TIL therapy in solid tumors.
2. Evaluate the barriers exhibited by solid tumors  that limit ATC/TIL efficacy.
3. Describe opportunities to enhance ATC/TIL therapy in solid tumors.

From "Bespoke" to "Off-the-Shelf" Stem Cell-Based Therapies: The Next Steps in Clinical Advancement

CHAIR: Katja Weinacht, MD, PhD, Stanford Medicine, United States

SPEAKER:

• Martin Maiers, BA, MS, NMDP, United States

Regenerative cell and tissue therapies from iPSCs offer the unprecedented opportunity to recover the function of diseased organs or tissues otherwise not available for allogenic transplantation. However, manufacturing of “bespoke” products from autologous iPSCs is time and resource-intensive and has to be avoided under specific circumstance such as for treatments of autoimmune disorders or genetically defective cells. This session will explore strategies to expand access, increase histocompatibility, and prevent immune rejection of third-party off-the-shelf cell therapy products.

SESSION OBJECTIVES:

1. How can we advance autologous therapies that are time and resource-intensive to manufacture into available products?
2. What are strategies for promoting immune evasion in donor cells?
3. Can we increase histocompatibility through HLA-editing?
4. What are examples (or visions) for a successful off-the-shelf cell therapies? 

Immunomodulatory Mechanisms and Potency Attributes of MSC Exosomes in Disease Treatment

CHAIR: Sai Kiang Lim, PhD, Paracrine Therapeutics, Singapore

SPEAKERS:

• Shibani Pati, MD, PhD, University of California San Francisco, United States
• Wei Seong Toh, PhD, National University of Singapore, Singapore

Understanding how mesenchymal stem/stromal cell (MSC) exosomes exert their therapeutic effects is crucial for their successful clinical translation. Immunomodulation is a common feature of their efficacy, and delving into this aspect will be key to elucidating the mechanisms of action of MSC exosomes. This session offers the latest insights into MSC exosome-mediated immunomodulation across diverse disease models, detailing potential modes and mechanisms of action as well as candidate potency CQAs. While identifying robust potency CQAs remains a significant challenge in clinical translation, focusing on immunomodulation provides a promising path forward.

SESSION OBJECTIVES:

1. What are the key immunomodulatory modes of action for MSC exosomes?
2. What are the mechanisms underlying these modes?
3. Which key attributes of MSC exosomes drive these mechanisms?

Pushing the Frontiers of First in Human Gene Modification Therapies

CHAIR: Tami John, MD, Stanford University, United States

SPEAKERS:

• Fraser Wright, PhD, Kriya Therapeutics, United States
• Jason Skowronski, BS, Kamau Therapeutics, United States

There is a pressing need to address the substantial expenses associated with research and development for the treatment of rare diseases with stem cells. It is essential that these discussions include the early implementation of Chemistry, Manufacturing, and Controls (CMC) and process development methodologies well ahead of the first administration of treatments to human subjects. The advancements made in the treatment of sickle cell disease offer valuable insights for overcoming the known challenges associated with the further development of therapies for rare diseases. By focusing on this topic and integrating the lessons learned, the stem cell therapeutic developers can ensure the continued progression of clinical trials and facilitate a sustainable transition to commercial care in the context of ongoing scientific advancements.

SESSION OBJECTIVES:

1. Discuss a first in human, high-cost and high-profile gene therapy trial including patient experience and manufacturing changes.
2. Highlight strategies for progression of trials from first in human to the next phase with considerations such as cost, manufacturing updates, scientific improvement.
3. How to plan for large scale translation of gene modification therapies to clinics.

Extending the Potential of Genetically Manipulated Cellular Therapies to Patients with Rare Disease

CHAIR: Susan Prockop, MD, Boston Children's Hospital, United States

SPEAKERS:

• Christine Duncan, MD, Boston Children's Hospital, United States
• Hilary Longhurst, MBBS, PhD, Auckland District Health Board, New Zealand

SESSION OBJECTIVES:

1. Models for academic and industry to advance cell and gene therapies given anticipation of a small market.
2. How to compare gene based therapeutics to standard approaches.
3. How to assess risk benefit of cell and gene therapies in rare disease.

Beyond Cancer: Cell Therapy for Autoimmune

CHAIR: William Hwang, MBBS, MBA, National Cancer Center, SingHealth, Singapore

SPEAKERS:

• Dominique Farge, MD, PhD, St. Louis Hospital, Assistance Publique-Hôpitaux de Paris, Université Paris-Cité, France
• Sonja Schrepfer, MD, PhD, Sana Biotechnology, Inc., United States
• David Porter, MD, University of Pennsylvania, United States

The session will present how CGT have successfully treated autoimmune diseases. Researchers and companies will learn how these cells can be better engineered for this purpose and clinicians will learn how to better apply these for breakthrough therapies for their patients. 

SESSION OBJECTIVES:

1. Present latest results for using mesenchymal stem/stromal cells (MSC) to treat systemic lupus erythematosus (SLE).
2. Present results of a Phase 1 study evaluating a hypoimmune allogeneic CD19-directed CAR T cell therapy, for severe r/ r B-cell mediated autoimmune diseases.
3. Overview of the promise of cell therapy for the treatment of autoimmune disease.

In Vivo Cell Engineering: Breaking Through the Manufacturing Bottleneck

CHAIR: Zlatibor Velickovic, PhD, Royal Perth Hospital, Australia

SPEAKERS:

• Yosef Rafeli, PhD, Ascensus Therapeutics, United States
• David Peritt, PhD, Lupagen, United States
• David Bishop, BMedSc(Hons), MBBS, PhD, FRACP, FRCPA, Westmead Hospital, Australia

Explore the next frontier in cell and gene therapy: transforming the human body into its own manufacturing bioreactor. This session will unveil groundbreaking advances in in vivo reprogramming, where targeted gene modification of cells occurs directly where they reside, eliminating the complexities of ex vivo manufacturing. Attendees will witness breakthrough data on novel delivery systems that enable precise genetic modification of cells within the body. Leading experts will present never-before-shared case studies on targeting strategies that have achieved remarkable results. This approach marks a paradigm shift in the development of personalised gene modified cell therapies.

SESSION OBJECTIVES:

1. Explore innovative technologies and delivery systems for targeted in vivo gene modification of cells, transforming the human body into its own bioreactor for cell therapy manufacturing.
2. Examine safety, efficacy, and regulatory considerations in in vivo cell engineering, including strategies for controlled gene modification and monitoring mechanisms.
3. Discuss clinical development strategies for in vivo gene-modified therapies, focusing on delivery efficiency, scalability, analytical challenges, and patient selection to advance the next generation of personalized cell and gene therapies.

Treating the Untreatable: Innovative Cell & Gene Therapy Approaches for Difficult Diseases

CHAIR: Zlatibor Velickovic, PhD, Royal Perth Hospital, Australia

SPEAKERS:

• He Huang, PhD, The First Affiliated Hospital - Zhejiang University School of Medicine, China
• Donald O'Rourke, MD, University of Pennsylvania School of Medicine, United States
• Qi Jing Li, PhD, A*STAR, Singapore

Meet the future CAR T cell therapies where antigen escape becomes an opportunity, not a roadblock. Check out novel 'smart' CARs that can navigate the hostile tumor microenvironment and transform CAR T cell therapy from a one-time intervention into an evolving defense system. Unlock the potential to treat solid tumors and resistant diseases effectively.

SESSION OBJECTIVES:

1. Explore innovative approaches in CGT for complex, difficult-to-treat diseases, with a focus on novel disease targets, case studies of successful indication expansion, and target validation in complex diseases.
2. Examine cutting-edge delivery technologies and multi-targeting strategies aimed at previously 'undruggable' tissues and targets.
3. Evaluate next-generation CAR T designs and T cell engineering solutions, emphasizing multi-targeting approaches to prevent antigen escape, novel sensing and switching technologies for adaptive responses, strategies to enhance CAR T-cell persistence, and overcoming immunosuppressive environments, while challenges remain in tumor penetration and cell survival.

Beyond the Bottleneck: Manufacturing and Supply Chain Overhaul for Real-World CGT Impact

CHAIR: Olga Bukatova, MSc, Azenta Life Sciences, Germany

This session will bring together key stakeholders from both non-profit and industry sectors, to evaluate between centralized and decentralized approaches and to put in the spotlight the actors closest to the patients.

SESSION OBJECTIVES:

1. Discuss the root causes for patient access challenges even for approved products (30% for CAR T, only a fraction for CRISPR) and the role of manufacturing and logistics.
2. Identify the right pathways to optimize manufacturing and reduce COGs for various advanced therapies, considering the trade-offs between centralized and decentralized approaches.
3. Explore the infrastructural updates and lessons learned through “cellular pharmacy”, point-of-care manufacturing, and revised distribution.

Bridging the Gap: Enabling Equitable Patient Access to Advanced Therapies

CHAIR: Rebecca Lim, PhD, CTMC, United States

SPEAKERS:

• Rodabe N. Amaria, MD, MD Anderson Cancer Center, United States
• Sebastian Kluboch, MD, Netherlands Cancer Institute, Netherlands

This session will delve into enablement and advocacy for patient access to advanced therapies regardless of geography or socioeconomic standing, including the ethics and economic role of medical tourism for healthcare access. 

SESSION OBJECTIVES:

1. Understand global efforts to bring options for approved advanced therapies to patients, and alternatives where advanced therapies are not available or approved.
2. Discuss the double-edged sword of medical tourism – when enabling access meets hope mongering.
3. Hearing the patient’s voice through the economic noise.

Designing Evidence Strategy Beyond Regulatory Approval:  Accelerating Reimbursement, Market Access and Patient Adoption

CHAIR: Katy Spink, PhD, Dark Horse Consulting Group, United States

SPEAKERS:

• Julie England, MD, Emerging Therapy Solutions, United States
• Doug Danison, BS, MBA, Bayer, United States

With many CGT products receiving accelerated approval with the hope of capturing value through meaningful long term clinical benefit; we often see patient and payer skepticism in the form of slow adoption, commercial failures, and product withdrawals. To avoid these challenges, it is essential to carefully plan your evidence strategy throughout development to ensure your data package at BLA/MAA submission supports not only regulatory approval but also reimbursement, market access, and patient adoption. In this session we will hear from speakers representing both the sponsor and the payer perspective regarding best practices for evidence development during clinical trials to support rapid access and adoption of therapies designed to offer transformational clinical outcomes.

SESSION OBJECTIVES:

1. Gain insights from experienced industry executives on how to design an evidence strategy that supports reimbursement and market access in addition to regulatory approval.
2. Share Payer perspective on value.
3. Discuss best practices for evidence development strategy.