RTW Charitable Foundation: Therapeutics development for Ultrarare Diseases (Letters of Intent)

Aim: to power rare-disease research, medical innovation, and humanitarian collaborations to improve the health of underserved communities. Grants will be awarded to support projects exploring diseases that affect fewer than 2,000 patients worldwide (monogenetic, life-threatening conditions affecting fewer than 300 patients worldwide preferred), diseases for which no commercial drug-development programs exist, projects in any therapeutic area; projects that involve any clinically validated therapeutic modality (gene-replacement and antisense oligonucleotide-based therapies preferred), and projects for which there is evidence that the proposed therapeutic modality works in an animal model (or in another suitable system).

Funding: up to $150,000 for one year.

https://www.rtwcf.org/research-grant-cycle/