Rare Genomics Drug Initiative (RGDI)

Advancing treatments for rare diseases.

The mission of the Rare Genomics Drug Initiative (RGDI) is to help advance drugs for rare diseases with limited therapeutic options. RGDI is a science-driven initiative aimed at building a strong foundation of knowledge combined with strategic partnerships to facilitate drug discovery and development.

The initiative uses both internal and external collaboration to define areas of greatest need and determine the best technology platforms to create drugs to address these needs. RGDI is agnostic to therapeutic modality and considers everything from small molecules to macromolecules, CRISPR/gene therapies, and cell therapies.

The RGDI team aims to advance therapies for neglected rare diseases through novel and repurposed drugs by bridging the gap between neglected rare diseases, the scientific/medical communities, and industry partnerships.

RGDI Strategy

Neglected Rare Diseases

Rare diseases with no approved treatments and few or no therapies in development.