ALTA

ALTA is a series of small molecule drug candidates developed by Alteron Therapeutics that target TAR DNA-binding protein 43 (TDP-43). These compounds are designed to block the nucleic acid binding of TDP-43 by specifically targeting the guanine pocket within its nucleic acid binding domain. By inhibiting this interaction, ALTA aims to disrupt pathological TDP-43-mediated pathways, eliminate multiple markers of neurodegeneration, and prevent neuronal toxicity. The program is currently in preclinical development for the treatment of amyotrophic lateral sclerosis (ALS), Alzheimer's disease, frontotemporal lobar degeneration (FTLD), and cystic fibrosis, with efficacy demonstrated in hTDP-43 transgenic mouse models.

Other names

ALTA Drug Candidates