Next-Generation ELP1 Splicing Correctors

About Tikun Therapeutics

Tikun Therapeutics is a private, pre-revenue biotech company operating as a Public Benefit Corporation with an exclusive focus on Familial Dysautonomia (FD), a severe and underserved genetic autonomic nervous system disease. The company is advancing a dual-platform strategy, developing both small molecule correctors of the underlying ELP1 splicing defect and an AAV-based gene replacement therapy, with programs currently in the pre-clinical stage. Its unique PBC structure and deep collaboration with academic leaders and the patient community underscore a stakeholder-aligned model aimed at accelerating development and guaranteeing affordable access.

Therapeutic Areas