Scientific Symposia

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

Chimeric antigen receptor (CAR) T cell therapies have transformed outcomes in B cell and plasma cell malignancies and hold promise for other indications in hematology. Yet, response remains variable and difficult to predict, and preclinical models often fail to recapitulate patient biology. Understanding and engineering the cell states that govern therapeutic efficacy is an active area of research. This session will explore how artificial intelligence (AI) models trained on multimodal data and clinical outcomes can move cell therapy design from empirical trial-and-error toward predictive, data-driven optimization and highlights the emerging AI systems for cell design. Altogether, this session charts a path toward rationally engineered next-generation cellular therapies.

Dr. Zinaida Good will present clinically informed AI models that predict patient response directly from single-cell RNA-sequencing of CD19-targeted CAR T infusion products and/or baseline histopathology data in large B cell lymphoma. Using an attention-based multiple instance learning framework, she will show how the approach identifies beneficial transcriptional programs and simulates outcomes for in silico engineered CAR T cells, enabling optimization anchored in clinical data in parallel with experimental validation.

Dr. Yusuf Roohani will describe a platform for AI-guided biological design that engineers cell state for precision therapeutics. Combining foundation models spanning biological scales with AI agents that direct data generation, he will address representation, dynamics, and experimental agency to search hypothesis spaces beyond current experimental reach through simulating cellular systems from whole-organism perturbation atlases to patient stratification of drug response.

Dr. Rahul Satija will present scalable frameworks that turn multimodal single-cell data into predictive models of cell state, describing Seurat for modality integration, scSLIDE for sample- and patient-level representations across large cohorts, and VIPerturb-seq for perturbation-resolved training data at scale, linking molecular state, genetic perturbation, and phenotype.

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 A. Shah, MD
Memorial Sloan Kettering Cancer Center
New York, NY

Speakers:

Urvi A. 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 Vector-Mediated In Vivo Hematopoietic Stem Cell Gene Therapy: Targets, Delivery, and Selection

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

Acute myeloid leukemia (AML) and myelodysplastic neoplasms (i.e. MDS) are increasingly recognized as diseases shaped not only by malignant clones, but also by complex and dynamic interactions with the immune microenvironment. Recent advances in single-cell and spatial genomics are providing unprecedented resolution of these interactions, enabling simultaneous characterization of cellular identity, functional states, clonal architecture, and spatial organization within the bone marrow. These approaches are revealing previously unrecognized immune cell states, disease-associated cellular programs, and specialized microenvironmental niches that contribute to immune evasion, disease persistence, and therapeutic resistance. The presentations in this session will illustrate how high-resolution genomic technologies are reshaping our understanding of the AML/MDS immune ecosystem and enabling the discovery of clinically actionable mechanisms of immune escape and therapeutic response.

Dr. Livius Penter will discuss mechanisms of immune escape that contribute to medullary and extramedullary AML relapse following chemotherapy, allogeneic hematopoietic cell transplantation, and immunotherapy. He will highlight how single-cell approaches are defining the interplay between leukemic clonal evolution, remodeling of the immune microenvironment, and dysfunctional anti-leukemia T-cell responses, with implications for the development of next-generation immunotherapeutic strategies.

Dr. Susan de Wolf will present spatial analyses of the AML/MDS bone marrow microenvironment, integrating in situ gene expression, mutation and RNA isoform profiling, and T-cell receptor characterization. Her presentation will examine spatial interactions between malignant and immune cells, with a focus on disease-associated regulatory T-cell populations and the role of TGFβ signaling in immune escape. Computational approaches to identify disease-defining cellular niches and changes in T-cell immunity across myeloid neoplasms will also be discussed.

Dr. Hussein Abbas will discuss the phenotypic diversity, spatial organization, clonal architecture, and functional states of AML/MDS-associated T-cell populations. He will highlight how single-cell and spatial transcriptomic approaches are redefining our understanding of immune surveillance and dysfunction in AML and informing biomarker development and novel immunotherapeutic strategies

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 M 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