Scientific Symposia

AI Models of Cell State for the Design of Next-Generation T Cell Therapies

This session will explore how leveraging big data and artificial intelligence can inform the design of the next generation of cellular therapies. Adoptive T cell therapies have transformed the treatment landscape for hematologic malignancies, but durable benefit remains limited to a subset of patients. A central challenge is that current engineering strategies still depend heavily on iterative preclinical testing that only incompletely captures patient-specific biology, cellular heterogeneity, and the tumor microenvironment. Recent advances in artificial intelligence and single-cell biology are creating a new framework for therapy design. Models trained on patient-resolved and perturbation-resolved single-cell datasets can now identify outcome-associated cellular programs, predict responses to genetic perturbations, and nominate engineering strategies expected to improve function. These developments shift the field from descriptive analysis toward data-driven design of therapeutic T cell states.

Chair:

Zinaida Good, PhD
Stanford University
Stanford, CA

Speakers:

Zinaida Good, PhD
Stanford University
Stanford, CA
Clinically Informed AI Models for Predicting and Engineering CAR T Cell Function

Yusuf Roohani, PhD
Arc Institute
Palo Alto, CA
State-of-the-Art Single-Cell Foundation Models for Future Cell Design

Rahul Satija, PhD
New York Genome Center
New York, NY
Scalable Frameworks for Multimodal Data Analysis and Next-Generation Cell Models

Designing Microbiome-Directed Interventional Trials in Hematologic Malignancies

This session will provide a practical framework for designing microbiome-directed interventional trials in hematologic malignancies. The gut microbiome has emerged as an important determinant of clinical outcomes, with growing evidence linking microbial composition and function to treatment response, survival, immune reconstitution, and toxicity. Although much of this evidence remains observational, advances in high-resolution sequencing, metabolomics, and immune profiling now enable integrated mechanistic studies that can move beyond correlation toward causality. While these mechanistic studies are evolving, their insights are informing therapeutic strategies.

Despite growing interest, few rigorously designed microbiome-directed interventional trials have been conducted in hematologic malignancies and their precursor conditions. This session will equip attendees with a practical framework for designing microbiome-directed clinical trials by highlighting lessons learned from completed and ongoing studies, key considerations for selecting interventions and endpoints, integrating microbiome and immune biomarkers, addressing important confounders, and identifying opportunities to develop novel microbiome therapies.

Dr. Urvi Shah will discuss dietary and prebiotic interventions as broad ecological approaches to microbiome modulation. She will review the rationale and summarize evidence linking diet, the microbiome, and clinical outcomes including NUTRIVENTION trial results. She will highlight key principles and practical challenges in designing rigorous dietary and prebiotic intervention trials.

Dr. Florent Mallard will discuss fecal microbiota transplantation (FMT) as an approach to restore disrupted microbial communities. With a focus on allogeneic hematopoietic cell transplantation (alloHCT), he will review the mechanisms and clinical evidence for FMT in the prevention and treatment of graft-versus-host disease, including the pooled allogeneic fecal microbiota (MaaT013), and discuss its potential to reduce infections.

Dr. Doris Ponce will focus on defined live biotherapeutic products as targeted microbial therapeutics. She will review the rationale for using defined bacterial consortia to selectively modulate microbial communities and host–microbe interactions, present clinical data from SER-155 to reduce infections in alloHCT and discuss adaptive trial design implementation in microbiome-directed clinical development

Chair:

Urvi Shah, MD
Memorial Sloan Kettering Cancer Center
New York, NY

Speakers:

Urvi Shah, MD
Memorial Sloan Kettering Cancer Center
New York, NY
Dietary and Prebiotic Intervention Strategies

Florent Malard, MD, PhD
Sorbonne University, Hôpital Saint-Antoine, AP-HP
Paris, France
Fecal Microbiota Transplant Intervention Strategies

Doris M Ponce, MD, MS
Memorial Sloan Kettering Cancer Center
New York, NY
Live Bacterial Therapeutic Intervention Strategies

In Vivo Gene Modification Approaches for Classical and Malignant Hematologic Disorders

This session will address viral and non-viral delivery methods and technologies (including their respective advantages and limitations) for in vivo gene therapy for classical and malignant hematological disorders. Ex vivo gene therapies are powerful treatment approaches for classical and malignant hematological disorders. Despite the clinical success of ex vivo hematopoietic stem cell (HSC)-based gene therapy, this procedure is costly and difficult to implement for large numbers of patients, particularly in countries with limited healthcare infrastructure. Furthermore, the gene therapy protocol (including cell culture) can affect HSC yield and engraftment, and myeloablation (required to make room for transplanted HSCs) causes short- and long-term complications. In parallel, chimeric antigen receptor (CAR) T-cell therapies have revolutionized the treatment of B-cell malignancies. However, their broader use is constrained by complex ex vivo manufacturing requirements and the need for lymphodepleting chemotherapy, which limits patient access. Viral and non-viral delivery approaches of in vivo gene editing aim to substantially reduce complexity, toxicity, and costs, making gene therapies more accessible and applicable across additional clinical indications.

Chair:

Annarita Miccio, PhD
Imagine Institut, Inserm, Universite' Paris Cite'
PARIS, France

Speakers:

Hans-Peter Kiem, MD, PhD
Fred Hutchinson Cancer Center
Seattle, WA
Viral Delivery to Hematopoietic Stem Cells in Vivo

William Peranteau, MD
Children's Hospital of Philadelphia
Philadelphia, PA
Lipid Nanoparticle (LNP) Delivery to Hematopoietic Stem Cells in Vivo

Michael Birnbaum, PhD
Massachusetts Institute of Technology
Cambridge, MA
In Vivo Car T Cell Generation to Treat Cancer and Autoimmune Disorders

Lab-Grown Red Blood Cells: From Scalable Manufacturing to Clinical Translation

The production of lab-grown red blood cells has advanced rapidly, creating new opportunities to address persistent challenges in transfusion medicine. However, major barriers remain before cultured red cells can be produced reproducibly, economically, and at the scale required for routine diagnostic and therapeutic use. These include the need for robust erythroid progenitor sources, efficient differentiation and enucleation, high-density culture systems, standardized product characterization, and clinically compatible manufacturing processes. This session will examine recent progress across the translational continuum, from scalable bioprocessing to unmet clinical needs and first-in-human studies.

Dr. Eric Bouhassira will discuss the metabolic and bioprocessing constraints that limit large-scale production of lab-grown red blood cells. He will describe self-renewing erythroblasts carrying KIT and JAK2 signaling modifications that can proliferate without exogenous cytokines while retaining the capacity for terminal differentiation and enucleation. He will also present hollow-fiber and stacked-membrane bioreactors designed to support ultra-high-density erythroid culture through improved nutrient delivery, waste removal, and process control.

Dr. Stella Chou will address the unmet needs of patients with red cell alloimmunization and rare blood types, including difficulties in antibody identification and access to compatible blood. She will discuss how genetically engineered lab-grown red blood cells could provide standardized, antigen-defined reagent cells and, ultimately, customized transfusion products.

Dr. Joanne Mountford will review the clinical translation of lab-grown red blood cells, including first-in-human studies evaluating their safety, survival, and function. She will discuss manufacturing consistency, regulatory requirements, product characterization, and the early evidence supporting cultured red cells as a future transfusion product for patients with complex transfusion needs.

Chair:

Eric Bouhassira, PhD
Albert Einstein College of Medicine
Bronx, NY

Speakers:

Eric Bouhassira, PhD
Albert Einstein College of Medicine
Bronx, NY
Scalable Manufacturing of Lab-Grown Red Blood Cells: Overcoming Metabolic and Bioprocess Constraints

Stella T Chou, MD
Children's Hospital of Philadelphia, University of Pennsylvania
Philadelphia, PA
Unmet Needs in Transfusion Medicine: Alloimmunization, Rare Blood Types, and the Case for Lab-Grown Red Cells

Joanne Mountford, PhD
Scottish National Blood Transfusion Service
Edinburgh, Scotland, United Kingdom
Clinical Translation of Lab-Grown Red Blood Cells: First-in-Human Studies and Early Outcomes

Single-Cell and Spatial Genomics Redefining the Immune Cell Landscape in AML/MDS

This session aims to define how single-cell and spatial genomics can refine disease classification, identify actionable vulnerabilities, and guide the development of the next-generation of immunotherapies for acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). AML and MDS are increasingly recognized as diseases shaped by a complex and dynamic immune microenvironment. Recent advances in single-cell and spatial genomics are transforming our ability to resolve this ecosystem at unprecedented resolution, enabling simultaneous interrogation of cellular identity, functional state, clonal architecture, and spatial organization. 

These technologies have revealed previously unrecognized immune cell states, altered myeloid compartments, and niche-specific interactions that drive immune evasion and therapeutic resistance. Importantly, emerging data link these microenvironmental features to outcomes following effective immunochemotherapy and allogeneic hematopoietic cell transplantation. Despite rapid progress, key questions remain regarding the reproducibility, interpretation, and clinical translation of these findings. This session will bring together leaders applying single-cell and spatial approaches to dissect (1) mechanisms of AML immune escape, (2) spatial organization of the AML/MDS bone marrow microenvironment, and (3) leukemia-associated T cell states. 

Chair:

Catherine Wu, MD
Dana-Farber Cancer Institute
Boston, MA

Speakers:

Livius Penter, MD
Charité - Universitätsmedizin Berlin
Berlin, Germany
Aml Immune Escape Mechanisms Driving Post-Therapy Relapse

Susan DeWolf, MD
Memorial Sloan Kettering Cancer Center
New York, NY
Spatial Niches within the AML/MDS Bone Marrow Immune Microenvironment

Hussein Ali Abbas, MD, PhD
M D Anderson Cancer Center
Houston, TX
Phenotypes and Specificities of AML/MDS-Associated T Cell Populations

Special Symposium on the Basic Science of Hemostasis and Thrombosis (SSBSHT)

This session will highlight cutting edge technologies that are poised to transform future research and therapeutic development in hemostasis, thrombosis, vascular biology, and megakaryocyte/platelet biology. By bringing together diverse, innovative approaches, the session is designed to equip investigators with conceptual and practical tools that can accelerate discovery across these interrelated fields. The invited talks will focus on (1) technologies and computational tools that enable the identification of novel, biologically relevant and potentially druggable targets; and (2) platforms and model systems designed to characterize, validate, and functionally test these targets in translationally meaningful ways, thereby directly informing drug discovery pipelines.

As is customary for this special symposium, the session will also feature a fourth presentation delivered by the winner of the Mary Rodes Gibson Award. This award recognizes the trainee with the highest scoring abstract submitted to the ASH Annual Meeting in the areas of hemostasis and thrombosis, thereby highlighting and elevating outstanding emerging science and future leaders in the field.

The session will conclude with an interactive panel discussion that integrates perspectives from all speakers. This discussion will distill key lessons from the technologies presented, explore how they can be applied or combined to address major unmet needs, and outline concrete opportunities for advancing hemostasis, thrombosis, and megakaryocyte/platelet research and therapeutic innovation. Immediately after the session will be a reception for the hemostasis and thrombosis community. 

Chairs:

Mettine Bos, PhD
Leiden University Medical Center
Leiden, Netherlands

Jaehyung Cho, PhD
Washington University School of Medicine
St. Louis, MO

Vivien Chen, PhD, MBBS
The University of Sydney School of Medicine
Sydney, Australia

Speakers:

Oliver Borst, MD
University of Tuebingen
Tuebingen, Germany
Platelet Lipidomics and the Discovery of Novel Anti-Thrombotic Targets

Lindsey George, MD
University of Pennsylvania School of Medicine
Philadelphia, PA
Rational design of FVIII variants for gene therapy: linking molecular insight to therapeutic performance