Vision & Mission
RARE Hope is dedicated to accelerating the development of therapeutics for rare disease patients through two interconnected strategies. These complementary strategies are designed to transform the rare disease research ecosystem and dramatically accelerate patient access to life-changing treatments.
Mission
- Advancing a patient-centered, therapy-focused prototype to investigate and treat a highly representative genetic neurological disease.
Our successful, scalable program establishes a foundation for expanding therapeutics development and enabling novel cross-disease discovery across a range of neurological disorders.
- Building a platform to optimize and accelerate therapeutics development at scale across neurological disorders.
Scaling rare disease therapeutics requires system-wide infrastructure development that integrates high-quality patient data and standardizes research tools, such as biomarkers and animal models. By developing a research infrastructure and optimizing research processes—while forging global partnerships with leading scientists, labs, patient advocates, and policy-makers, and harnessing AI-powered analysis of multilayered datasets and cross-disease insights—RARE Hope is constructing a scalable platform to accelerate therapeutics development and catalyze systemic change.
Our Vision
More patients. More therapeutics. More quickly.
The benefits of addressing neurological conditions at scale are profound.
A platform approach harnesses economies of scale and process efficiencies to reduce costs and accelerate therapeutics development. By enabling treatments for more patients, it attracts top scientists, leading laboratories, and key capital partners—fueling a powerful, self-sustaining cycle of innovation.
Although this model requires greater upfront investment, it significantly reduces per-patient costs over time—delivering faster, more affordable, and more effective treatments. By focusing broadly on neurological diseases—starting with genetically related disorders—we can advance treatments across multiple conditions and, critically, improve the lives of millions of patients.
