An oral, brain-penetrant splicing modulator targeting ELP1. Incorrect splicing of the Elongator complex protein 1 (ELP1) gene causes Familial Dysautonomia (FD), a neurodegenerative disease that results in uncoordinated movement, blindness through retinal degeneration, and risk of unexplained sudden death, among other symptoms. A single T-to-C [...]

Induction of fetal hemoglobin expression by a PRC-targeting small molecule. Pociredir (FTX-6058) is an oral, allosteric polycomb repressive complex 2 (PRC2) inhibitor that binds embryonic ectoderm development protein (EED) within this complex, ultimately boosting fetal hemoglobin (HbF) expression . Increasing HbF levels has been shown to [...]

The Galapagos CFTR potentiator, GLPG2451, is an oral, once-daily clinical candidate for cystic fibrosis (5-80 mg QD). It was advanced into a high-profile Ph. II triple-combo study with the CFTR correctors GLPG2737 and GLPG2222 in partnership with AbbVie, and was seen as a potential competitor to Vertex’s CF franchise, but unfortunately weak [...]

“Compound 19” is a selective brain-penetrant inhibitor of the ceramide galactosyltransferase UGT8, intended for use in substrate reduction therapy for rare lysosomal storage disorders. The compound has good oral exposure in higher species (%F = 52 in dog) and is active in models looking at enzymatic activity using isotopically-labeled [...]

Tyra Biosciences’ lead asset, TYRA-300, is a potent, selective, and orally bioavailable FGFR3 inhibitor designed to mitigate the toxicities commonly seen with pan-FGFR inhibitors. A key objective of the program was to meet the safety standards required for advancing into pediatric populations for the treatment of ACH (achondroplasia) and other skeletal dysplasias. TYRA-300 is currently being evaluated in Ph. I clinical trials for advanced urothelial cancers and other solid tumors and, importantly, has received IND clearance to initiate a Ph. II clinical trial in pediatric ACH patients.