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LHON Opportunity Map

Philanthropic Opportunities for LHON

LHON Collective engaged the Milken Institute’s Science Philanthropy Accelerator for Research and Collaboration (SPARC) in 2023 to evaluate challenges and opportunities in the Leber hereditary optic neuropathy (LHON) field. Specifically, SPARC assessed where philanthropic investment in research and development could have the greatest impact in transforming the LHON therapeutic landscape. 

SPARC conducted a systems-based review of the current LHON research ecosystem using a detailed analysis that integrated knowledge from the peer-reviewed scientific literature, interviews with nearly 70 subject matter experts, analysis of government and nonprofit funding trends, and a two-day scientific retreat held in February 2024. Through this analysis, we identified four high-priority opportunities where philanthropic investment and partnership could have a transformative impact.

A brief description of each opportunity follows, including information on the resources required (i.e., investment budget, operational considerations, timelines) to unlock that opportunity and achieve its objectives. Advancing any of the priority areas identified will help create favorable conditions to accelerate research and discovery in LHON therapeutics. Some opportunities have overlapping objectives; all are important factors for de-risking translational research and attracting industry partners to bring more and better therapeutics to market.

Select an Opportunity Card to Learn More

Link to Opportunity 1: Collect natural history data and biological samples in open-access repositories.
LHON Opportunity 2: Develop research tools for the field, beginning with models.
LHON Opportunity 3: fund basic discovery and drug repurposing researching to advance the therapeutic dev pipeline.
LHON Opportunity 4: Invest in platform tools and tech to align with other diseases and raise awareness of LHON.
Conclusion and next steps for LHON opportunities.
Opens LHON Opportunity Map PDF document
Diagram of LHON Opportunity Map. The diagram includes 2 strategies, Therapeutic dev ad invest in tech. Opportunities 1-4.
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