Lung Fibrosis - Collagen I translation inhibitors
Program
DISCOVERY
OPTIMIZATION
IND ENABLING
Fibrosis
Lung Fibrosis
Fibrosis represents a large group of diseases that remain without effective treatment. It is caused when a wound healing process goes out of control and progresses into continued scarring in the affected organs. The common denominator across fibrotic processes in different organs is over-production of Collagen Type I.
Using our platform, we discovered and validated highly selective Collagen-1 translation inhibitors. Our compounds are lung selective and do not act on other tissues nor on Collagen-1 basal production.
- High throughput screening using Anima’s proprietary platform
Three chemically diverse series identified - Dose-response activity shown with activity at the nM levels in cell-based assays
Compounds demonstrated selectivity toward lung tissue
- Do not affect global translation
- Do not affect transcription
- Selective to lung cells (no effect on skin/liver/bone) - Compounds optimization
Mode of action of translation modulation elucidated for the three series
Molecular target of the three series confirmed to be part of the novel target space of mRNA translation regulation - Efficacy in Animal models
Compounds effectively reduce Collagen I in animal models Superior to an approved Idiotypic Pulmonary Fibrosis drug and Phase III drug candidate - Next IND-enabling studies