SCIENTIFIC PROGRAM

TRANSLATIONAL PATHWAY PROGRAM

ABOUT THE PROGRAM

The ISCT 2026 Dublin Translational Pathway Program will feature an extraordinary lineup of 4 plenaries and 17 concurrent sessions bringing together leading global experts to present preclinical research with high-impact potential, and promising results across phases of clinical trials.

The Program is segmented into four tracks, mirroring the phases of clinical development from pre-clinical research to clinical trials, and onwards to market access.

Join us in Dublin to dive deep into cutting-edge data, uncover groundbreaking innovations and hear more on the status of the field and where to drive it further.

SCIENCE &
INNOVATION
PRE-CLINICAL RESEARCH TRANSLATION TO CLINIC
REGULATORY & EARLY CLINICAL DEVELOPMENT CLINICAL ACCELERATION
CLINICAL EXPANSION & SCALE-UP LAUNCH
& PATIENT ACCESS
POST-MARKET APPROVAL

THE PRESIDENTIAL PLENARY

Nearly a decade from the groundbreaking FDA approval of Kymriah, ISCT President Miguel Forte, MD, PhD hosts a world-renowned panel of experts to examine key takeaways from the past, present, and future of CAR-Ts, to distill insights from this continued success story that will shape CGT development as a whole.

The past -

A global leader in pediatric cellular immunotherapy and a principal clinical investigator behind CAR-T therapy, Dr. Stephan Grupp will examine CAR-T as a transformational breakthrough modality that propelled the cell and gene therapy field into clinical success and commercialization.

Drawing on the clinical development, regulatory pathway, and approval of Kymriah as a defining case study, Dr. Grupp will showcase how early-stage scientific innovation translated into real-world therapeutic impact, establishing a foundation for today’s CGT ecosystem.

The present -

A leading European clinician-scientist in CAR-T therapy, Dr. Sara Ghorashian will share current realities of CAR-T implementation in clinical practice, drawing from direct experience in the European healthcare environment. She will highlight clinical successes achieved to date, evolving patient access and adoption, and the ongoing clinical and commercial barriers to delivery.

Her discussion will examine where expectations have been met — and where barriers remain — providing practical lessons on how today’s experience is informing the next phase of CAR-T development.

The future -

The pioneer behind Kymriah, Dr. Bruce Levine will look ahead to the future of CAR-T therapies across manufacturing, scientific innovation, and sustainability.

He will discuss emerging strategies to enable efficient and scalable manufacturing, alongside advances such as multiplex editing and the potential of in-vivo approaches. Dr. Levine will also examine how evolving business models can support broader accessibility, long-term viability, and continued innovation in CAR-T therapies.

SCIENCE & INNOVATION

DISCOVERY & PRE-CLINICAL RESEARCH

▾

Karen English, PhD

Maynooth University

Ireland

This segment addresses scientific and technological developments in pre-clinical CGT research that have clinical implications. It will highlight the latest progress in CGT science and platform technologies, including target validation, animal models, and proof-of-concept studies—key drivers of innovation in next-generation treatments.

PLENARY SESSION An Overnight Success, 30 Years in the Making: Cell Therapies at the Forefront of Medicine ▾

This session will explore the safety and low immunogenic profile of MSCs and MSC-EVs, the potential of NK cell therapies to overcome challenges faced by T-cell immunotherapies, and the exciting promise of CAR-macrophages and macrophage-based therapies in advancing cancer immunotherapy and tackling fibrosis.

Each presentation will highlight barriers that have hindered the CGT field for the past 30 years and showcase innovative approaches to overcoming them. Drawing on cutting-edge basic science, the session will provide fresh insights into how cell therapies are being engineered to minimize immunogenicity and toxicity, modulate immunosuppression, and enhance the clearance of fibrotic tissues. Delegates will gain a deeper understanding of the novel strategies shaping the next generation of cell therapies.

Speakers:

Claudia dos Santos, MD, FRCPC, University of Toronto, Canada
Stuart Forbes, MBChB, PhD, FRCP(Ed), FRSE, FMedSci, The University of Edinburgh, United Kingdom
Robin Parihar, MD, PhD, Baylor College of Medicine, United States

CONCURRENT SESSION Reprogramming the Narrative: From Short-Term Effects to Lasting MSC Immune Impact ▾

CONCURRENT SESSION Expanding the EV Horizon: Novel Sources and Bioengineering for CGT Innovation ▾

CONCURRENT SESSION From Concept to Clinic: In Vivo Targeting of HSCs ▾

CONCURRENT SESSION iPSC-Derived Therapies: Engineering the Next Frontier in Regenerative Medicine ▾

TRANSLATION TO CLINIC

REGULATORY & EARLY CLINICAL DEVELOPMENT

▾

Bambi Grilley, RPh, CIP, CCRC, CCRP, RAC,

Cheif Regulatory Officer, ISCT

Baylor College of Medicine
United States

Designing and launching First-In-Human (Phase I) trials requires a strong foundation built through IND-enabling studies, comprehensive CMC file preparation, and early manufacturing considerations to enable translational success. These efforts are complemented by proactive interactions with health authorities and IRB/ethics boards to ensure that all steps effectively support translational success.

PLENARY SESSION The Promise of Allogeneic Islet Cell Therapy for T1D: Emerging Clinical Data and Patient Outcomes ▾

This session will explore the latest developments in allogeneic islet cell therapy as a transformative approach for Type 1 diabetes management. Experts will review Phase I/II clinical trial data for zimislecel (VX-880) and examine outcomes from gene-edited, hypoimmune islet cell transplants.

Discussion will center on safety, efficacy, and quality-of-life improvements, alongside an overview of therapeutic modalities, eligibility criteria, and delivery strategies. Attendees will gain a comprehensive understanding of current progress and its implications for clinical practice.

Speakers:
Hanne Scholz, PhD, University of Oslo, Norway
Trevor Reichman, MD, PhD, FACS, University of Toronto, Canada

CONCURRENT SESSION Engineering Safety: Precision Control Strategies to Mitigate Cell Therapy Toxicities ▾

CONCURRENT SESSION Advancing NAMs Toward Real-World Use: Organoid and Organ-on-a-Chip Systems for Translational Impact & Efficacy ▾

CONCURRENT SESSION Clinical Trial Design in Cell Therapy: Tackling Heterogeneity for Meaningful Outcomes ▾

CONCURRENT SESSION In Vivo Gene Editing: From Delivery Platforms to Personalized Therapies ▾

CONCURRENT SESSION iPSC Therapies at a Crossroads: Autologous vs. Allogeneic Approach ▾

CLINICAL ACCELERATION

CLINICAL EXPANSION & SCALE-UP

▾

Jaap Jan Boelens, MD, PhD

Memorial Sloan Kettering Cancer Centre

United States

This segment explores critical considerations during Phase II and Phase III trials through to marketing authorization, emphasizing strategies for regulatory submission, scale-up and scale-out approaches, and tech-transfer best practices. Presenters will also share practical insights on overcoming clinical and manufacturing bottlenecks, drawing from real-world experience to highlight solutions that enable successful late-stage development.

PLENARY SESSION Increasing Access to CGT Products: Pricing and RCT for Rare Diseases ▾

To demonstrate that randomized controlled trials (RCTs) in rare diseases are feasible, highlighting the strong enrollment, and experience sharing from the OTL-203 (MPS-I) trial. It will also showcase examples from India and Spain, where reduced treatment costs have increased patient access while maintaining comparable outcomes and achieving shorter vein-to-vein times.

Large-scale clinical data and real-world outcomes will be presented to provide a compelling view of the broader impact and effectiveness of these approaches.

Speakers:
Rahul Purwar, PhD, Indian Institute of Technology of Bombay, India
Ashish Gupta, MBBS, MPH, University of Minnesota, United States
Nuria Martínez-Cibrián, MD, Hospital Clínic Barcelona, Spain

CONCURRENT SESSION Rewriting Immunity: Innovative CGT Approaches for Autoimmune Disorders ▾

CONCURRENT SESSION Optimizing Conditioning and Lymphodepletion: PK-Driven, Easy-to-Implement Strategies for Durable Clinical Outcomes ▾

CONCURRENT SESSION Building the Fourth Model: Semiconductor-Inspired Manufacturing for Scalable Cell Therapies ▾

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.

Chair:
Fernanda Masri, PhD, Minaris Advanced Therapies, United Kingdom

Speakers:
Owen Bain, EngD, Royal Free London NHS Foundation Trust, United Kingdom
Eytan Abraham, PhD, Minaris Advanced Therapies, United States

CONCURRENT SESSION Beyond the Infusion: Practical Challenges and Long-Term Risks in Gene Therapy ▾

CONCURRENT SESSION The Next Frontier in AML CAR T: Manufacturing Smarter, Targeting Harder ▾

LAUNCH & PATIENT ACCESS

POST-MARKET APPROVAL

▾

Phil Vanek, PhD

Chied Commercialization Officer, ISCT

Gamma Biosciences
United States

This segment addresses topics centered on the clinical adoption of market-authorized CGT products for patients globally. Expert presenters will present on marketing authorization pathways, reimbursements, and pricing strategy. In addition, get real-world data on global launch strategies, supply-chain logistics, and distribution. Topics will also bridge towards the economics of advanced therapies and proven business models within the field.

PLENARY SESSION From Collection to Care: The Clinical Realities Driving Advanced Therapies Adoption ▾

This session explores the safety and immunogenic profile of MSCs and MSC-EVs, the potential of NK cell therapies, and the promise of CAR-macrophages and macrophage-based therapies. Each presentation highlights challenges that have hindered the CGT field for 30 years and the innovative approaches now overcoming them.

Over the past 10 years, we’ve seen revolutionary new therapies progress though the clinic, companies launched on their future promise, and regulatory approvals throughout the world. We’re now collecting real-world data addressing availability and adoption, and facing the realities and growing pains of bringing a new modality through existing clinical infrastructure. What have we learned, and what can we do to take the friction out of clinical adoption and access.