PROJECT SUMMARY The main goal of the RUNX1 Research Program 6th Annual Scientific Conference and Patient Meeting is to foster collaboration and provide opportunities for knowledge and data sharing among researchers, clinicians, and patients and their families for a rare disease called RUNX1 familial platelet disorder with predisposition to hematologic malignancies (RUNX1-FPD or FPDMM). Individuals with the disorder have a 35-50% lifetime risk of developing a hematologic malignancy. Of those who develop a malignancy, over 50% are diagnosed with AML. Conference participation has grown each year. We had over 182 attendees for the virtual conference in 2021. In previous years, 50% of research attendees were women, 30% of attendees were post-docs or graduate students, and 15% of independent investigators were 5 years or less in their first faculty position. We plan to continue to engage a diverse audience of investigators. RRP is committed to engaging patients, clinicians and/or researchers from underrepresented racial and ethnic groups to broaden participation from these communities at our conference and beyond. The agenda includes research-specific sessions open to all stakeholders as well as a track dedicated to RUNX1-FPD patients and family members. There are some overlapping sessions designed to encourage engagement between the two communities. The objective is to promote and drive patient-engaged research forward, with direct patient input in the development and planning of the sessions via our 23-member Research Guided by Patients Committee (RGPC). The research focused portion of the meeting will bring together experts from the fields of hematology, immunology, oncology, cell therapy and basic science who are committed to uncovering the mechanisms underlying the pathogenesis of RUNX1-FPD with the goal of developing therapeutic interventions that impact both the bleeding issues and most importantly the predisposition to malignancy. The audience will include investigators devoted to studying RUNX1, leukemia progression, clonal hematopoiesis, gene editing, and hematopoietic stem cell transplants. In 2022, we will continue to expand our network by encouraging more immunologists to join and participate. It has become increasingly clear through the NHGRI-sponsored natural history study of RUNX1-FPD that immune dysfunction and inflammatory disorders are common. RUNX1-FPD includes a unique patient population where there may be a nexus between immunology and oncology based entirely on activity of a single master regulator, RUNX1.

PROJECT NARRATIVE The RUNX1 Research Program 6th Annual Scientific Conference and Patient Meeting fosters collaboration and provides opportunities for knowledge and data sharing among researchers, students, clinicians, and patients and their families for a rare disease called RUNX1 familial platelet disorder with predisposition to hematologic malignancies (RUNX1-FPD - OMIM # 601399FPDMM).