Connecting ASXL research to what comes next
Progress in rare disease research does not happen in isolation. It happens when the right people, ideas, and opportunities come together.
From March 2-5, 2026, Dr. Karen Ho, Chief Scientific Officer at the ASXL Rare Research Endowment (ARRE) Foundation, attended an international scientific meeting focused on epigenetics and gene regulation. This Keystone Meeting, organized by Karim-Jean Armache (NYU) and Cigall Kadoch (Harvard) was entitled, “Epigenetics and Gene Regulation in Health and Disease: Linking Basic Mechanisms with Therapeutic Opportunities,” held in Geneva, Switzerland. The highly focused meeting centered on the latest, cutting-edge research in epigenetics, which is the research that will help us understand how genes like ASXL1, ASXL2, and ASXL3 function, and what happens when there is a change (mutation) in those genes.
At the meeting, Karen connected with leading researchers studying the same, or related, biological pathways that are affected in ASXL-related disorders. These connections are important because they bring new expertise, ideas, and potential collaborations into our field.
She also met with biotechnology and pharmaceutical companies working on therapies that target gene regulation. These are the types of approaches that could one day be relevant for ASXL-related disorders.
Just as importantly, Karen advocated for the development of better research tools, including specific antibodies for ASXL1, ASXL2, and ASXL3. These tools are essential for scientists to study how these genes function. Without them, research is slower and more limited.
Putting our 2026 strategy into action
At the ARRE Foundation, we are not waiting for progress to happen. We are actively building it.
This includes funding foundational research, supporting key studies like the ASXL-Related Disorders Natural History Study and Biobank and the ASXL Behavioral Phenotyping Study (ALPS), and creating opportunities for collaboration across the scientific and medical communities.
Karen’s participation in meetings like this is one example of how we are putting that strategy into action. It reflects our commitment to ensuring that ASXL-related disorders are part of the broader scientific landscape, and that our community benefits from advances happening across the field.
Building what comes next
This is how progress happens. Step by step, connection by connection, we are building the foundation for what comes next.
And while there is still much work to do, each of these efforts brings us closer to a future with better understanding, better care, and ultimately, future treatments for individuals with ASXL-related disorders.
Be part of what comes next
If you believe in accelerating research for ASXL-related disorders, we invite you to become part of the Discovery Society by making a monthly donation to the ARRE Foundation. Ongoing support allows us to invest in the foundational science, partnerships, and research leadership that move this work forward every day. Your monthly gift helps ensure that efforts like Karen’s continue — building the knowledge, connections, and momentum needed to advance understanding, improve care, and prepare for future treatments.