named programs · 2 · from sources
What they call their work
Safety Roadmap for PURA Syndrome Gene Therapy
Funded and directed preclinical research with The Jackson Laboratory to establish a safety framework for gene therapy dosing in PURA Syndrome, including gene overexpression studies in mouse models
Tools for Researchers Portal
Provides access to scientifically developed mouse models and other research tools for PURA Syndrome to accelerate therapeutic development by the global research community
activities · 4 groups
What they do
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Rare Genetic Disease Research and Advocacy 3 activities
- Conducting and directing preclinical research for PURA Syndrome gene therapyConducts strategic evaluation of translational research and directs preclinical research to determine safe levels of PURA protein expression for gene therapy development.
- Developing and distributing research models for PURA SyndromeDevelops and provides animal models (including mouse models) and induced pluripotent stem cell (iPSC) lines for use by researchers worldwide, in partnership with The Jackson Laboratory, to advance studies on PURA Syndrome.
- Sharing scientific progress through advocacy briefingsHosts scientific briefings to disseminate findings and updates on funded research toward a cure for PURA Syndrome, engaging the scientific and patient communities.
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Genetic and Neurological Disease Research Funding 1 activity
- Funding research to develop treatments and a cure for PURA SyndromeFunds scientists and research initiatives aimed at developing treatments and ultimately a cure for PURA Syndrome, supporting cutting-edge research through targeted grants.
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Charity Golf Tournament Fundraiser 1 activity
- Hosting fundraising events to support PURA Syndrome researchOrganizes annual fundraising events including golf outings, spin classes, pickleball clinics, and dinner programs that have raised over $250,000 in a single year to support research initiatives.
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Uncategorized 1 activity
- Presenting research findings at scientific conferencesPresents research results, including on PURA gene therapy dosage sensitivity, at major scientific meetings such as the ASGCT Annual Meeting.
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financials · form 990 · fy2024
revenue
Total revenue$258K
Contributions & grants$227K88%
Program service revenue$00%
Investment income$30K12%
Other revenue$0
expenses
Total expenses$257K
Program expenses99%
Admin / overhead1%
Fundraising0%
Salaries & benefits$0
Grants paid out$130K
Largest expense lineProfessional Fees
balance sheet
Total assets$653K
Cash$58K
Investments$595K
Liabilities$0
Net assets$653K
Liquid reserves30.4 mo
4 years on record · 2021–2024 · YoY revenue -26.4%
leadership · form 990 part vii · fy2024
Who runs it
board members · 4
- ADAM CAPON — DIRECTOR
- JEAN WEINBERG — CO-FOUNDER & PRESIDENT
- KYLE CZEPIEL — CO-FOUNDER & DIRECTOR
- RAPH POSNER — DIRECTOR
relationships · 14
Who they work with
- Andrew Steinsapir Partner — Scientific Advisory Board member providing guidance on research evaluation for PURA syndrome.
- BTIG Partner — Putting Green Sponsor for the 2026 Swing for a Cure event
- Benjamin T. Cocanougher Partner — Scientific Advisory Board member contributing clinical and research expertise to inform efforts to find a treatment and cure for PURA syndrome.
- BraunHagey & Borden LLP Partner — Hole Sponsor for the 2026 Swing for a Cure event
- Goldman Sachs Partner — Gold Sponsor for the 2026 Swing for a Cure event
- Mezzanine Distributors Partner — Silver Sponsor for the 2026 Swing for a Cure event
- New York State Early Intervention Government — State program that provided early therapeutic support for Jack
- Rare Disease Translational Center Partner — Partnered with JACKS TOMORROW INC through The Jackson Laboratory to provide research tools for studying PURA Syndrome.
- Ripple Partner — Sponsor for the 2026 Swing for a Cure event
- Scientific partners conducting PURA Syndrome research Partner — Collaborates with research partners to advance studies toward a cure for PURA Syndrome.
- The Jackson Laboratory Partner — Collaborates on preclinical gene therapy research and development of mouse models for PURA Syndrome.
- The Jackson Laboratory Partner — Collaborates with JACKS TOMORROW INC to develop and distribute mouse models and iPSC lines for PURA Syndrome research.
- The Jackson Laboratory Partner — Collaborates with Jack's Tomorrow on developing a mouse model to study PURA syndrome.
- Westhampton Country Club Partner — Host venue for the "Swing for a Cure" fundraising event.
strategies · 4
How they approach the work
Named approaches extracted from this org’s sources. Where others share an approach, follow it to see the full set of orgs running it.
- Direct Research Funding with Full Cost Absorptionmethodology: direct-research-fundingBy restricting grants to projects where institutions absorb indirect costs, we maximize the impact of every dollar on direct research activities, because eliminating overhead spending accelerates progress toward treatments and a cure.
- Patient-Directed Research Modelmethodology: patient-directed_researchBy empowering patient advocacy organizations to fund and guide early-stage translational research, we accelerate rare disease innovation, because patient-led research initiatives increase focus, urgency, and alignment with clinical needs in underfunded disease areas.
- Research Tool Development for Scientific Accessmethodology: research_tool_developmentBy developing and sharing genetically engineered mouse models and iPSC lines, we lower barriers to entry for researchers, because open access to high-quality disease models accelerates therapy development for ultra-rare conditions like PURA Syndrome.
- Research-Driven Cure Developmentmethodology: research-driven_cure_developmentBy funding and directing scientific research — including disease modeling, therapy development, and gene therapy safety studies — we accelerate treatment discovery and advance toward a cure for PURA Syndrome, because targeted, mission-aligned research is the most effective path to clinical solutions for ultra-rare disorders.