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The AUMsilence platform

Designed against your transcript, and self-delivering

The AUMsilence platform designs the antisense oligonucleotides and makes them self-delivering. The chemistry is what lets the cell take them up, with no transfection reagent.

Designed, not chosen

Sequence design by machine learning

The platform reads the transcript for the gene and the species named. Machine learning scores every candidate window along it, and several sequences are designed against different regions of that transcript.

  1. Step 1: Every window scored by machine learning

    Each candidate sequence along the target transcript is scored for predicted activity and specificity, and the highest-scoring sequences are selected for synthesis.

  2. Step 2: Synthesized, purified and shipped

    The selected sequences are synthesized, purified to the grade you choose, and shipped lyophilized. Add them to culture medium or inject them in vivo: no transfection reagent, no carrier.

FigureThe platform scoring every window of a transcript
score5′transcript3′each tick a candidate windowchosenchosenchosen
A schematic of the method, not data from any transcript: the transcript as a ruler, each tick a candidate window, the line the score along it, the lit windows the ones chosen.

Self-delivering

The cell takes them up, so the workflow is one step

Our AUMsilence platform uses chemical modifications that enable self-delivery.

  • Transfection-free delivery

    AUMsilence sdASOs are self-delivering. They are added directly to cell culture or injected in vivo, with no lipid transfection reagent, electroporation, or viral vector.

  • Broad compatibility

    The same AUMsilence sdASO works from cell lines to primary patient cells, three-dimensional cultures, organoids and animal models.

  • Specificity by design

    Each sequence is scored against the transcriptome before it is chosen, and antisense silencing does not pass through the RNA-induced silencing complex, so there is no seed-region off-targeting to account for.

What it powers

One platform, every RNA class

  • Self-delivering technology

    No transfection reagent, electroporation, or viral vector. Add to cell culture or inject in vivo.

  • Compatible cell types

    Works in primary cells, neurons, immune cells, stem cells, 3D cultures, organoids, and animal models.

  • Targeted RNA classes

    Target mRNA with AUMsilence sdASO, microRNA with AUMantagomir sdASO, long non-coding RNA with AUMlnc sdASO, and viral RNA with AUMsilence V+ sdASO.

One platform, every stage of drug discovery: from target ID to preclinical studies

Evidence

Published in scientific journals

  • Nature
  • Science
  • Circulation
  • Nature Immunology
  • Nature Genetics
  • Blood
  • Cellular and Molecular Immunology
  • Journal of Extracellular Vesicles
  • Cell Stem Cell
  • Nature Communications
  • Science Immunology
  • Science Translational Medicine
  • Science Advances
  • Neuro-Oncology
  • Acta Neuropathologica
  • PNAS
  • The EMBO Journal
  • Cell Reports
  • Blood Advances
  • Aging Cell
  • Frontiers in Immunology

Every study on the publications page says what was silenced, in which cells or animals, and what was found, with the paper's own numbers.

See the publications

Name a target, and the platform designs against it

For research use only. Not for use in diagnostic or therapeutic procedures.