A year of science, momentum, and real steps toward treatments for STXBP1.

2025 was our busiest and most productive research year yet.

Across laboratories in Israel, the UK, Europe, and the US, we pushed forward on multiple therapeutic strategies, expanded our scientific ecosystem, and reached milestones that only a year ago felt out of reach.

Most importantly, we made meaningful progress toward real treatments for children living with STXBP1-related disorders.

Three Competitive Research Grants Awarded

In 2025, we funded three major scientific programs designed to accelerate therapeutic development across small molecules, RNA therapeutics, and disease modeling.

1. Small-Molecule Discovery (Prof. Dan Kaganovich and Dr. Triana Aman)

This project combines computational chemistry and high-throughput biology to identify drug candidates for STXBP1.

• Computational work: Led at the Weizmann Institute by Dr. Efrat Ben-Zeev, who generated and screened a pipeline of promising compounds.
• Biological validation: Conducted at the University of Southampton, where we developed a new cellular assay and identified our first encouraging biological hits.
  

2. ASO Therapeutics Program (Dr. Nofar Mor and Dr. Omer Revah)

This grant supports our push toward a targeted RNA-based therapy for STXBP1.

• Development of isogenic cell lines
• Functional assays to evaluate ASO rescue potential
  We expect initial data very soon, and this program is a core pillar of our path to clinical translation.
  

3. Mouse Model Development (Prof. Illana Gozes, Tel Aviv University)

We funded the creation and characterization of a new R292C mouse model, carrying the same mutation as our son, Raphael.
This model is essential for testing promising therapeutics and deepening our understanding of the disorder.

2.2 Million Dollars in New Grant Funding Secured

This year, we successfully raised 2.2 million dollars in external scientific grants for our collaborators:

• 400,000 dollars for Dr. Omer Revah to advance the STXBP1 organoid platform
• 1.8 million dollars in two additional grants for new therapeutic programs that will be announced soon
  

This influx of support strengthens Israel as an emerging global hub for STXBP1 research and enables us to move faster than ever.

Four New Disease Models Developed

To increase our therapeutic testing power, we built a robust modeling infrastructure:

• Human brain organoids
• Brain-implanted organoid chimeras in rats
• Recombinant STXBP1 protein platform
• Advanced computational models
  

These models are already being used to evaluate drugs, ASOs, and future gene therapies.

A Major Leap Toward Clinical Trials

2025 brought us to the doorstep of a milestone.
We are preparing to launch the first STXBP1 clinical trial in Israel.
This is a remarkable step forward for our children and for the global community.

Growing the Scientific Community

This year, we put tremendous energy into raising awareness, educating, and mobilizing researchers:

• More than 15 scientific and inspirational talks across rare disease conferences, hospitals, academia, and tech companies
• Participation in the STXBP1 European Scientific Conference in Heidelberg, with a 10-person Israeli delegation and four scientific presentations from Israeli researchers
• Growing our LinkedIn community to more than 1,000 followers
  

Researchers, physicians, and industry partners worldwide are now reaching out to collaborate. This growing ecosystem is exactly what we set out to build.

Expanding Preclinical Capabilities

• We successfully established a living colony of R292C mouse models and began phenotyping.
• We launched a biomarker discovery program focused on tremor and movement signatures as objective indicators of drug response.

Raising Global Awareness

Through storytelling, scientific updates, and continuous outreach, we strengthened the global presence of STXBP1 research. This year brought:

• More collaborations with biotech and pharma
• More labs are joining the field
• Increased public awareness in Israel and internationally
• A stronger and clearer Rafa’s Moonshot identity and mission
  

Every new partnership accelerates the path to treatments.

Looking Ahead to 2026

We are entering the new year with momentum and big goals.

In 2026, we aim to:

• Launch our first clinical trial in Israel
• Advance each therapeutic program toward preclinical readiness
• Expand our researcher network and strengthen international collaborations
• Build new industry partnerships
• Continue cracking the scientific mysteries of STXBP1
• Move closer to therapies that can help the 1,500 diagnosed children worldwide
  

Rafa’s Moonshot started as a personal mission.
Today, it is a growing global movement grounded in science, collaboration, and determination.

Thank you to every scientist, donor, partner, and supporter who helped make this year possible.
We are grateful for the dedication and expertise of our Scientific Advisory Board, who work closely with us, provide invaluable guidance, challenge us, and push our programs forward. We look forward to expanding this collaboration in the coming year.

Together, we are transforming knowledge into treatments and hope into action.

Sagi and Ella