Impact
Our Track Record
Through grants, partnerships, and matched investments, we have significantly amplified the impact of our research funding:
Impact by the Numbers
$1.6M
Donor Funds Raised
$700k
Funding Deployed in 20+ Grants
$8M
Leveraged Investment
1100%
Return on Investment
We work with world-leading teams. We help validate and refine new technologies. These collaborations drive innovation and magnify our impact.
By providing high-touch involvement across our portfolio of projects, we protect and grow our strategic investments. We collaborate closely with researchers across the lifecycle of a study to maximize the value of these investments for patients and researchers alike.
What We’ve Accomplished:
- A historic breakthrough: The first-ever prime editing rescue of a mouse model of a neurological disorder. Prime editing is the most advanced and safest genomic editing technology.
- Established a central hub for research, connecting a global network of scientists, building international collaborations, and promoting the exchange of resources, expertise, insights, ideas, and experimental findings.
- Recruited world-leading, pioneering scientists with expertise in novel therapeutic modalities, like David Liu and Tim Yu, to collaborate with best-in-field experts like Al George, Kathy Sweadner, and Arn van den Maagdenberg.
- Catalyzed multiple preclinical therapeutics development programs, with one clinical trial already underway.
- Long-standing support of discovery in basic science has enabled novel discoveries of mechanisms and pathology.
- Built a powerful toolkit of research resources, including multiple cell and animal models, biomarker discovery, patient data, wearables, and more. A combination of hypothesis-driven, focused research and unbiased omics-based exploration and discovery.
- Representation of patient priorities in research: a research program designed to target symptoms that matter most to patients. A coalition of patients eager to participate in research and therapeutics development.
Proving What’s Possible
We began by building a prototype research program grounded in patient-centric, therapeutics-focused science. Success in one disease provides a blueprint for tackling others, demonstrating how a high-impact model can be applied across rare diseases.
Building for Scale
Our platform extends beyond a single disorder by enabling cross-disease comparisons and shared discovery. By identifying common tools, pathways, mechanisms, and targets across conditions, we create efficiencies that accelerate innovation.
Driving Systemic Change
Data is essential and research tools are key. We champion the collection, centralization, and standardization of high-quality patient data, and the standardization of research resources like models and biomarkers for all rare diseases.
Bridging Science, Strategy, and Advocacy
At the intersection of research, strategy, and patient advocacy, RARE Hope works to bridge divides, optimize processes, connect stakeholders, break silos, and remove inefficiencies— ensuring that rare disease patients gain faster access to life-changing treatments.
By prototyping, systematizing, and scaling research, RARE Hope is reshaping the future of therapeutic development for rare disease patients.
A Platform for Impact: RARE Hope is building a scalable platform to accelerate therapeutics development and drive systemic change by:
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Designing a comprehensive research infrastructure that centralizes, standardizes, and integrates high-quality patient data while advancing the development of standardized research tools such as biomarkers and animal models.
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Optimizing and systematizing research processes.
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Harnessing AI-powered analysis to interpret complex, multilayered datasets.
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Leveraging cross-disease insights to identify common mechanisms and accelerate discovery.
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Forging global partnerships with leading scientists, labs, patient advocates, and policy-makers to drive innovation and facilitate regulatory pathways.
