Preventing Expansion​
Preserving Tomorrow

Triplet is developing transformative therapies that treat repeat expansion disorders at their source.


Patient Driven

We are addressing the underlying cause of repeat expansion disorders, a class which includes Huntington’s disease, myotonic dystrophy and spinocerebellar ataxias. Our focus is driven by patients. Their willingness to challenge these diseases by sharing their clinical needs and participating in genetic research has allowed us to discover novel therapeutic approaches to treat these disorders. We’re inspired by patient stories of perseverance and courage which underscore the importance of our work.

Triplet is committing 1% of its company value into an independently managed patient support fund that will be used to secure access to care and therapies.

Our Story

Triplet was founded at the nexus of a new genetic understanding of repeat expansion disorders. We have brought together this field’s foundational scientific leaders alongside experts in drug discovery and clinical development to reimagine treatment options for repeat expansion disorders. Supported by a syndicate of premier life sciences investors, we are rapidly advancing multiple pipeline candidates toward clinical development.

The Science

Patient data has enabled us to identify that the DNA damage response (DDR) pathway is an underlying driver of repeat expansion disorders. Targeting this pathway therefore holds promise to treat a wide array of repeat expansion disorders at their source.

Top Presentations

CHDI 2022: SHIELD HD – A Natural History Study to Support Clinical and Biomarker Development