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