Fibrosis - Collagen I mRNA biology modulators
| Indication | mRNA modulation | Discovery |
Optimization |
IND enabling |
|
|---|---|---|---|---|---|
Immunology |
|||||
| Lung fibrosis | Collagen-1 | ||||
| Multiple fibrosis | |||||
| Multiple fibrosis | |||||
| Area | Indication | mRNA modulation | Discovery |
Optimization |
IND enabling |
||
|---|---|---|---|---|---|---|---|
| Immunology | Lung fibrosis | Collagen-1 | |||||
| Multiple fibrosis | |||||||
| Multiple 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 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 I mRNA modulators.
Our compounds inhibit newly synthesized Collagen I protein that is over produced during Lung, liver, kidney or skin fibrosis while sparing Collagen I basal production across tissues.
- High throughput screening using Anima's TranslationLight technology
Three chemically diverse series identified - Dose-response activity shown with activity at the nM levels in cell-based assays
Compounds demonstrated selectivity toward newly synthesized Collagen I in primary Lung, Skin and Liver cells
- Compounds do not affect global translation
- Compounds do not affect transcription
- Compounds do not affect basal collagen translation in animal studies in Lung, Liver, kidney, heart and bone
- Prevent differentiation of fibroblasts into diseased myofibroblasts - Compounds optimization
Mode of action of collagen-selective mRNA modulation elucidated for the three series
The molecular target of the three series confirmed to be part of the novel target space of mRNA modulators - Efficacy in Animal models
Compounds effectively reduce Collagen I in animal models of fibrosis
Superior to an approved Idiotypic Pulmonary Fibrosis drug and an Autotaxin inhibitor - Candidate Selection
- Next IND-enabling studies