Our Approach

Tissue selective drugs
Specifically decrease or increase
protein translation
Chemically diverse hits against
novel target space
New insight into disease biology

Our platform identifies small molecules that selectively modulate the mRNA translation of target proteins. The novel target space enables the discovery of drugs that can either decrease or increase the production of proteins in a tissue selective manner.

  • Multiple specific mechanisms regulating mRNA translation

    Cells have specific mechanisms that regulate the translation of individual mRNAs. We target proteins that have specific biological roles in coordinating and regulating translation in a selective way, allowing for the discovery of small molecules that modulate the translation of a target protein.

    Read more about mRNA Translation Regulation

  • Monitoring mRNA translation

    Our technology platform enables us to monitor mRNA translation of specific proteins or a group of proteins inside cells, visualizing when, where and how much of a target protein is being made in a disease model, in real time.

    Translation of Collagen I mRNA in Human lung fibroblasts

    Translation of Collagen I mRNA in Human lung fibroblasts

    • A and B. Monitoring of Collagen I mRNA translation in human lung fibroblasts using a pair of tRNA which specifically decodes Collagen I mRNA. (A) Low level of translation of Collagen I in resting, healthy fibroblasts; (B) High levels of Collagen I protein synthesis in activated, disease-model of lung fibrosis.
    • C and D. Accumulation of Collagen I protein after five days. (C) Low levels of Collagen I protein accumulated in healthy lung fibroblasts; (D) High amount of Collagen I protein accumulated in cells in a disease model of lung fibrosis.

    Read more about our Translation Control Therapeutics Platform

  • mRNA Translation and Target ID technologies

    We have gained tremendous expertise, allowing us to build technologies around mRNA translation control. Our screening platform centers on a novel target space, including mRNA processing, transport, stability, localization and protein translation regulation. Compounds act on proteins and small RNAs, which regulate RNA rate and protein translation.

    Translation Technologies

    Target ID Technologies

    Translation Control
    Quality assurance of labeled tRNA
    Analysis of interactions inside cells of mRNA and the proteins that bind to it
    Identification of the compound's effect on mRNA and tRNA modifications
    Analysis of the half-life of target mRNA and protein to compound
    Identification of proteins which are bound by compound
    Specific isolation of mRNA and its associated proteins in response to compound
    RNA sequencing of untreated versus compound treated cells
    A score for compounds' effect on global translation
    Ribosome occupancy on mRNA
    Proprietary algorithm selects for gene-specific codon pair
    Fluorescent probes detect single mRNAs inside cells
  • Chemically diverse hits target multiple regulatory mechanisms

    We are discovering compounds that target mRNA maturation, steady state levels, localization and translation. Unlike other approaches that target the mRNA molecules or mRNA modifiers, our technology provides multiple shots on goal without depending on a single assumption about disease biology.

    Hit compounds reduce Collagen I protein without affecting mRNA

    Hit compounds reduce Collagen I protein levels (upper panel, green), without affecting Collagen I mRNA levels (lower panel, red). Dots in the nucleus are COL1A1 transcription sites. Signal in the cytoplasm, is cytoplasmic mRNA. As a control, a compound which is known to inhibit transcription, reduces both protein and mRNA signals (ActD: Actinomycin D).

  • Detecting translation of a specific mRNA

    Once the specific protein tRNA pair has been selected, we validate the assay by using siRNAs. We have been able to demonstrate Assay Specificity in all of our programs.

    The Assay specificity is biologically validated by using siRNA

    To ensure the FRET signal (light) is coming from Collagen I or c-Myc translation, we transfected cells with siRNA specific to the target mRNA. Most of the PSM signal in the siRNA transfected cells disappeared, emphasizing that the PSM signal has been mainly generated by Collagen I translation.

  • Tissue selectivity: a novel paradigm

    Our small molecule drugs are tissue-specific; this stems from the fact that different cells regulate the production of the same protein in different ways.

    Anima’s compounds for Lung Fibrosis are active in the lungs but not in skin fibroblasts

    Collagen I translation modulators regulate Collagen I translation in a tissue specific manner. A representative hit compound, compound X, inhibits Collagen I protein accumulation in the cell line used for the screen, WI-38 lung fibroblasts (panel A) and in primary human lung fibroblasts (panel B). However, the hit compound does not inhibit Collagen I translation in human primary skin fibroblasts (panel C).

    Anima’s compounds for Lung Fibrosis were also tested in liver fibroblasts

    We are able to identify compounds that are tissue specific in the regulation of Collagen I. We tested the effect of the compounds in liver fibroblasts (panel A) and in lung fibroblasts (panel B). Compounds A, B and C reduce the Collagen I in lung fibroblasts (panel B); compounds B and C do not affect Collagen I in liver fibroblasts, however compound A does show some inhibition of Collagen I in liver fibroblasts (panel A).

  • Safer drugs by design

    The mechanisms regulating mRNA translation are mostly tissue selective. This enables us to discover drugs that work only in the specific tissue where there is disease. Anima's approach has been proven to discover compounds that are tissue selective and uses multiple secondary assays to confirm compounds' selectivity.

    c-Myc specific inhibition

    Our screening platform discovers a set of compounds that can affect only c-Myc protein and no other Myc family members as shown by Immunofluorescence in IF in A549 NSCLC (lung cancer cell line).