This session explores strategies to mitigate toxicity in CAR T-cell and other cellular therapies by embedding safety mechanisms into Phase I product design.

Discussions will focus on conditional switches, cytokine blockade, and suicide-gene systems as tools to de-risk first-in-human trials and build regulatory confidence. Speakers will highlight how rational design, preclinical validation, and manufacturing integration enable the safe translation of next-generation immune effector cell products.

This session details the cutting-edge use of non-animal models to advance human medicine. Leaders in Organ-on-a-Chip, 3D tumor organoids, and Cell & Gene Therapy testing will discuss how these human-relevant platforms improve predictability, reduce animal use, and accelerate the development and manufacturing of next-generation therapeutics.

This session will explore Heterogeneity of Treatment Effect (HTE), a critical challenge in precision medicine. Experts in T-cell therapies, TCR engineering, and gene editing will discuss how their work identifies biological factors driving varied patient responses.

The session will examine innovative strategies for rigorous patient selection and therapeutic tailoring to maximize efficacy while ensuring the safest application of cutting-edge, individualized treatments.

This session will focus on current developments in in vivo gene editing based on the LNP delivery platform for the treatment of various non-malignant diseases.

The discussion will explore pre-clinical data requirements for first-in-human studies, early Phase1/2 data of ongoing trials with emphasis on safety, durability and regulatory requirements, especially for ‘platform’ designation for treating ultra-rare diseases with gene-editing approaches.

Stem cell-based therapies for Parkinson’s disease are advancing rapidly, with two distinct strategies emerging: allogeneic, off-the-shelf products such as raguneprocel and bemdaneprocel, and autologous, patient-specific approaches using iPSC-derived neurons.

This session will compare these paradigms, exploring clinical trial progress, immunological considerations, manufacturing challenges, and long-term outcomes.

Experts will share insights into the promise and limitations of each approach, highlighting how these innovations could transform treatment for neurodegenerative disorders. Join us for a deep dive into the science, clinical experience, and future directions of cell-based therapies for Parkinson’s disease.

Autoimmune diseases remain among the most challenging conditions to treat, demanding approaches beyond conventional immunosuppression. This session explores cutting-edge cellular strategies aimed at reprogramming immunity.

Experts will share insights from immune resetting through bone marrow transplantation, to the use of mesenchymal stromal cells in progressive multiple sclerosis and the early CAR-T applications in autoimmune disorders.

Attendees will gain a deeper understanding of the scientific rationale, safety considerations, and translational pathways shaping the future of cell-based therapies for autoimmune disease.

Conditioning (including lymphodepletion) is a critical determinant of success in cell and gene therapy, yet high variability in the clearance will result in highly variable drug exposures and immune responses that can compromise outcomes.

This session brings together experts to explore PK-PD principles guiding conditioning regimens, such as busulfan and fludarabine exposure as a model for optimizing myelo-ablation and lymphodepletion, and additional strategies to prevent rejection.

Attendees will gain practical insights and learn about 'easy to implement' strategies into tailoring conditioning for safety, efficacy, and durability across diverse indications.

Challenges to the cell and gene therapy industry’s status quo take center stage with the introduction of a bold “Fourth Model” for manufacturing scale-out—drawing inspiration from the semiconductor sector.

Instead of incremental fixes, we explore a radical blueprint to achieve true mass-market access. Through an interactive stress-test “game,” panelists and audience will evaluate this model against cost, risk, and regulatory hurdles.

Attendees will leave with a clear understanding of why cross-industry innovation is essential, the limitations of current approaches, and a new framework to guide future manufacturing strategy debates.

Ex vivo gene therapy success depends on more than the infusion. This session examines critical challenges across the treatment continuum—from cell collection hurdles in sickle cell disease to lessons from long-term effect of residual disease.

Experts will also address emerging concerns around malignancy risk after gene therapy, exploring monitoring strategies and the impact of vector design and prior chemotherapy.

Attendees will gain actionable insights into optimizing patient selection, improving global implementation, and safeguarding long-term outcomes.

Acute myeloid leukemia (AML) presents unique challenges for CAR T-cell therapy including manufacturing complexity, antigen heterogeneity, and intrinsic resistance mechanisms.

This session brings together leading experts to discuss the innovations shaping the next generation of AML-targeted CARs. Topics include lessons learned in production and toxicity management, strategies for multi-antigen targeting to prevent relapse, and the design of armored CARs to overcome the hostile tumor microenvironment.

Attendees will gain insights into how these approaches aim to improve safety, efficacy, and scalability for AML patients.