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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.

How a sequence is designedOrder against a target

Designed, not chosen

Sequence design

The platform reads the transcript for the gene and the species named. Every candidate window along it is weighed against the scientific literature and the design parameters, using algorithms, machine learning and AI, combined with our extensive experience in design. Several sequences are designed against different regions of that transcript.

  1. Step 1: Candidate scoring

    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 chosen sequences are synthesized, purified to the selected grade, checked, and shipped lyophilized. No transfection reagent, electroporation, or viral vector is required. Add AUMsilence sdASOs to the culture medium, or inject them in vivo.

How a sequence is designed, in detail
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.

Where it worksThe technologyAgainst RNAi and gene editing

  • 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

See the publications

Name a target, and the platform designs against it

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