Discover a therapeutic approach

We aim to pursue the following strategies to find treatments:

  1. Target AGO2 protein: repurposing existing drugs or finding novel small-molecule drugs

  2. Target AGO2 RNA: splicing antisense oligonucleotides

  3. Target AGO2 DNA: gene replacement therapy and gene editing

A better understanding of a) disease mechanisms and b) how the disease presents itself gained from our earlier phases will strongly guide the development of therapeutic approaches.

Learn more on this page about the three approaches and why we believe in them.

As we don't know yet strategy will work best, we will go after these strategies in parallel.

The science and medicine are so complex that it's just impossible to know in advance which therapies will be safe and effective. I would suggest a portfolio approach, even for a single disease.

Andrew Lo, Prof. of Finance at MIT and rare disease advocate

Therapeutic strategies

Protein therapy

Small molecule drugs to increase production or activity of AGO2, starting with screening already approved or developed drugs for off-target effects


RNA therapy

Upregulate production of healthy AGO2 with a splicing antisense oligonucleotide (ASO) or regulatory element ASO


DNA therapy

Deliver a functional copy of AGO2 (AAV-mediated gene therapy) or correct the mutation (CRISPR base editing)