Pipeline
TDP-43 accumulation is found in diseased neurons of many neurodegenerative disorders (ND), such as amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), Alzheimer's disease (AD), hippocampal sclerosis of aging (HS-Aging), chronic traumatic encephalopathy and limbic predominant age-related TDP-43 encephalopathy (LATE). The causautive role of TDP-43 in neuronal death has been firmly estabolished.

As a critical regulator of the CFTR protein, TDP-43 also play an important role in cystic fibrosis (CF). High TDP-43 activity not only reduces full-length CFTR protein levels but also keeps the protein from being transported to cell surface where it functions. Inhibiting TDP-43 can increase CFTR or dF508CFTR on cell surface,thus is a promising strategy that can lead to a cure for CF.

Our compounds remove TDP-43 toxicity by blocking its nucleic acid binding activity.
Candidates
Targets
Indications
Discovery
Pre-clinical
Phase 1
Phase 2
Phase 3
ALTA-808
TDP-43
ALS

ALTA-808
TDP-43
AD

ALTA-808
TDP-43
FTD

ALTC-819
TDP-43
CF

Diagnosis
Biomarker
ND