Fast Forward to Impact

Strategic Plan 2026-2028

Our 2026–2028 Strategic Plan outlines how the STXBP1 Foundation will accelerate research, strengthen clinical trial readiness, build biopharma interest, and support families today. The plan reflects input from families, researchers, clinicians, industry partners, and Foundation leadership, and is designed to guide our work over the next three years.

The STXBP1 Foundation’s 2026–2028 Strategic Plan builds on the progress of our previous plan and outlines the next phase of our work. Developed with input from families, clinicians, researchers, industry partners, and Foundation leadership, this plan will guide how we accelerate therapeutic progress while continuing to support individuals and families impacted by STXBP1-related disorders today.

For families, this plan means continued investment in resources, education, community connection, and readiness for future clinical trials. For researchers and clinical partners, it means stronger data infrastructure, clearer collaboration pathways, and a Foundation committed to accelerating progress. For biopharma partners, it means a more organized, prepared, and engaged STXBP1 ecosystem.

Download Strategic Plan
Download Strategic Plan

Why We Believe Progress is Possible

The role of STXBP1 in disease was only discovered in 2008, and the STXBP1 Foundation established in 2017. Despite the newness and rarity of our disorder, research is progressing quickly and we are optimistic that viable therapeutics are possible because of these four important learnings:

  1. The root cause is known - changes in the STXBP1 gene cause STXBP1 disorder

  2. We have a strong understanding of STXBP1’s biological function in the brain

  3. We have a much better understanding of the clinical picture of STXBP1 disorders now than we did even 3 years ago.

  4. Restoring levels of the wildtype STXBP1 protein reverses some disease-relevant symptoms in mice

  • Goal 1: Activate Biopharma Partnerships and Capital

    Goal 2: Build Trial Readiness Infrastructure

    Goal 3: Advance STXBP1 Programs from Discovery to Clinical Readiness.

  • Goal 1: Extend and Enhance the STARR Natural History Study

    Goal 2: Launch a Family Trial Preparedness Program

  • Goal 1: Deliver a High-Quality Annual Summit

    Goal 2: Equip Families Through Clear, Transparent, Actionable, and Two-Way Communication

    Goal 3: Reduce Barriers to Participation

  • The Impact Fund would provide a potential mechanism for catalyzing early-stage therapeutics, biomarkers, and enabling technologies, with any financial returns reinvested into STXBP1 research

  • Strengthen collaboration across the international STXBP1 ecosystem through aligned messaging, shared tools, and voluntary brand coordination — formalized progressively as engagement deepens.

See Therapeutic Pipeline
See Therapeutic Pipeline