LTPO01 study

Presented at Genetics-inspired Therapeutics for PH by Richard Trembath, King's College London

Summary

This study explores SMURF1 as a therapeutic target for pulmonary arterial hypertension (PAH). It shows how inhibiting SMURF1 can restore BMP signalling, reduce cell growth, and improve disease models. Allosteric findings and preclinical and clinical data support LTP001, a first-in-class SMURF1 inhibitor.

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