Solution modality / Antisense Oligonucleotides

Reduce a transcript, or change how it is spliced.

The choice depends on whether your program needs less protein or a different protein product. BioTwin’s strategy templates help frame that decision around transcript and isoform context.

Current work Strategy templates

Knockdown and splice correction serve different goals

Identify whether reducing a transcript or changing its processing serves the objective, and define the transcript/isoform context and decisive measurement.

Antisense mechanisms

  • RNase-H antisense knockdown
  • Splice-switching oligonucleotides

Templates for the intervention hypothesis

  • In BioTwin today · Strategy templates represent antisense knockdown and splice switching, linked to the transcript and isoform context.
  • Development and validation · Current coverage supports framing and comparing intervention hypotheses. Sequence design, chemistry, delivery and experimental qualification are further requirements for a complete ASO program.

Define the transcript outcome before sequence design

Specify the intended transcript or splice outcome, the important isoforms and the tissue. Establish the sequence, chemistry, delivery and measurement requirements for the chosen antisense mechanism. An siRNA strategy uses a distinct RNA-interference mechanism.

Related transcript and pathway methods

Relevant methods; exact support depends on the target, inputs and scientific route.

  • Isoform-Specific Target Assessment · Determine whether an isoform distinction changes the target, intervention or assay your program should use. Read the method
  • Resistance Min-Cut and Protein–RNA Co-Targeting · Investigate modeled signaling escape routes before choosing a protein–RNA co-targeting hypothesis. Read the method

Discuss Antisense Oligonucleotides for your program

Start with the biological objective and the decision you need to make. We can establish which existing methods fit and what additional development would be required.