ISTH 2025 Annual Conference Preview – Inherited Bleeding Disorder Sessions

Published: May 30, 2025

The 2025 International Society on Thrombosis & Hemostasis conference is quickly approaching. From June 21 through June 25, in Washington D.C., the ISTH Congress will see 28 state-of-the-art sessions, thousands of attendees from around the world, and plenty of exciting opportunities to network and discuss the latest in thrombosis and haemostasis science.

With five days full of insightful sessions, we took the time to curate a list of sessions focusing on inherited bleeding disorders for your convenience.

Saturday, June 21

Hemophilia Gene Therapy: In Search of a “Cure” – Societal Aspects

11:30 AM – 12:45 PM
Description: This session will delve into the decision-making process surrounding gene therapy, featuring insights from first-hand accounts who will share their personal perspectives, addressing not only clinical considerations but also broader ethical and personal dimensions of gene therapy.

Real-world Data in Hemophilia and Rare Bleeding Disorders

1:00 PM – 2:15 PM
Description: This session will feature a series of presentations on real-world data on disease bleeding and treatment patterns of female hemophilia A patients, gender differences in clinical manifestations and severity of FVII deficiency, variability of bleeding phenotype in people with hemophilia A, and more.

Novel Mechanisms in Hemophilia

1:00 PM – 2:15 PM
Description: This session will feature a series of presentations on how Mim8 enhances procoagulant activity of select hemophilia B-causing Factor IX variants, procoagulant platelets and its impact on bleeding severity in patients with hemophilia A, the role of factor VII as a regulator of angiogenesis and promoter of endothelial barrier stability, and more.

Sunday, June 22

Women and Bleeding Disorders: Being a Young Teenager with von Willebrand Disease

7:00 AM – 7:45 AM
Description: This master-class level session features speaker Mary Mathias, MA, MRCP, FRCPath, MD. Dr. Mathias is the Haemophilia Centre Director at the United Kingdom’s largest children’s bleeding disorders center, Great Ormond Street Hospital. The hospital manages nearly 200 children with von Willebrand disease.

Inhibitors and Immune Responses to FVIII

9:30 AM – 10:45 AM
Description: This session will feature a series of presentations on how the von Willebrand factor D”D3 region in BIVV001 alters to the immune response to factor VIII, dynamic transcriptomic profiling in previously untreated patients with hemophilia A, factor VIII inhibitor titers in people with hemophilia A on emicizumab prophylaxis in ATHN 7, and more.

Joint Health in Hemophilia: Biomarkers

9:30 AM – 10:45 AM
Description: This session will feature a series of presentations on how extracellular vesicles containing microRNA10a attenuate joint bleed-induced hemophilic arthropathy, plasma proteomics and collagen biomarkers in people with hemophilia A receiving emiczumab in STASEY, how proteomic expression profiles may identify hemophilia patients at risk of acute joint pains, and more.

Novel Therapies in Hemophilia – Clinical Trials I

2:45 PM – 4:00 PM
Description: This session will feature a series of presentations on an adjunct therapy for hemophilia B, a study on NXT007 prophylaxis in emicizumab-naive persons with hemophilia A without inhibitor, the treatment of bleeding episodes with efanesoctocog alfa in children, and more.

Monday, June 23

Perioperative Hemostatic Management in Inherited Coagulation Disorders

7:00 AM – 7:45 AM
Description: This session will highlight real-world insights from a leading center experienced in using diverse hemostatic approaches during surgery.

Prediction Outcomes in Inherited and Acquired Hemophilia

9:30 AM – 10:45 AM
Description: This session will feature a series of presentations on predicting joint damage in non-severe hemophilia A using machine learning, epidemiology characteristics and outcomes of acquired hemophilia A (AHA) in the USA, and more.

Joint Health in Hemophilia: Prediction Imaging and Interventions

2:45 PM – 4:00 PM
Description: This session will feature a series of presentations on the quantification of the influence of clinical predictor on the hemophilia joint health score, using methotrexate as an adjunct to FIX prophylaxis, and more.

Novel Therapies for Hemophilia – Basic Science

2:45 PM – 4:00 PM
Description: This session will feature a series of presentations on how a novel FVIII-mimetic trispecific nanobody restores coagulant potential in vitro and in vivo, how long-term use of siRNA antifibrinolytic targeting plasminogen is well tolerated in non-human primates and limits menstrual bleeding, and more.

Tuesday, June 24

The ISTH Hemophilia Guidelines at One Year: A Critical Appraisal

8:00 AM – 9:15 AM
Description: Last June, the ISTH published guidelines for the management of hemophilia. Subsequent to the publication, there were two manuscripts published, one critiquing the guidelines and one providing a “deconstruction” of the guidelines for the clinician. This special session will provide the authors of these publications the opportunity to discuss their papers.

Immune Tolerance Induction in Hemophilia A

9:30 AM – 10:45 AM
Description: This session will feature a series of presentations on a translational approach to ITI therapy, how sublingual immunotherapy induces tolerance to FVIII in hemophilia A mice, and more.

Gene Therapy – Fundamental Research

9:30 AM – 10:45 AM
Description: This session will feature a series of presentations on base editing correction of hemophilia A in hPSC-derived liver sinusoidal endothelial cell, CRISPR-Cas9 gene editing modalites for correction of canine VWD type 3 model ex-vivo, and more.

Von Willebrand Disease: Studies in Diagnosis and Management

9:30 AM – 10:45 AM
Description: This session will feature a series of presentations on the value of VWF platelet and factor VII-binding assays vs. genetic testing in diagnosing type 2 VWD, clinical phenotype and pathophysiological mechanism underlying qualitative low VWF, and more.

Real-world Data in Hemophilia: Thrombotic Risk and Surgical Procedures

2:45 PM – 4:00 PM
Description: This session will feature a series of presentation on major surgical outcomes with efanesoctocog alfa, optimizing surgical approaches for patients with inherited factor VII deficiency, thrombotic rates in bleeding disorder individuals treated with concentrate or bispecific antibody, and more.

Novel Therapies in Hemophilia – Clinical Trial II

2:45 PM – 4:00 PM
Description: This session features presentations on the association of antithrombin levels with efficacy of fitusiran prophylaxis in people with hemophilia A or B with and without inhibitors, favorable nonclinical safety profile of HMB-002 for prophylactic treatment of von Willebrand disease, and more.

Wednesday, June 25

Novel Therapeutic Approaches in Hemophilia

8:30 AM – 9:45 AM
Description: This session will discuss gene therapy for tolerance induction, the use of bispecific antibodies for select hemophilia B mutations, and more.

Mechanisms and Novel Treatments in Rare Bleeding Disorders

10:15 AM – 11:30 AM
Description: This session features topics such as the mathematical analysis of emicizumab, characterizing FXI deficiency by in-silico and plasma-based proteotyping, the effects of emicizumab on human monocytes/macrophages through a single-cell transcriptome analysis, and more.

Gene Therapy – Pre-clincal and Clinical Outcomes

10:15 AM – 11:30 AM
Description: This session discusses topics such as safe multi-year bleeding reduction by AAV gene therapy in neonatal and juvenile hemophilia B dogs, gene transfer with BBM-H901, steady musculoskeletal health after gene therapy in severe hemophilia, and more.

For a complete look at all the planned schedules for the 2025 ISTH annual conference, visit the event’s official website.