Discover a therapeutic approach

First, we need to understand which mutations lead to a gain of function of the protein (GOF) and which ones to a loss of function (LOF). This is because the therapeutic strategies are opposite: stop the mutated protein for GOF, and increase the healthy one for LOF. 

We aim to pursue the following strategies to find treatments:

  1. Target AGO1 and AGO2 proteins: repurposing existing drugs or finding novel small-molecule drugs

  2. Target AGO1 and AGO2 RNA: antisense oligonucleotides to upregulate activity for LOF (splicing) or shut down the mutated protein for GOF

  3. Target AGO1 and AGO2 DNA: gene replacement therapy (LOF only) 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 or reduce production or activity of AGO1/2, starting with screening already approved or developed drugs for off-target effects


RNA therapy

For gain of function mutations shut down mutated AGO1/2, and for loss of function mutations upregulate production of healthy AGO1/2 with a splicing antisense oligonucleotide (ASO) or regulatory element ASO


DNA therapy

Deliver a functional copy of AGO1/2 (AAV-mediated gene therapy) or correct the mutation (CRISPR base editing). The former only works for loss of function mutations.