1,600,000

people live with Charcot-Marie-Tooth Disease Type 1A (CMT1A)

(Pronounced Shark - Oh - Marie - Tooth, hence our company name)

CMT1A is a well-understood mono-genetic disease.

Gene duplication leads to protein overproduction

A duplication of the PMP22 gene leads to a 150% overproduction of the PMP22 protein, disrupting proper myelin formation in the Peripheral Nervous System (PNS). The resulting myelin thinning impairs the transmission of signals from the brain to the muscles, causing muscle weakness, loss of sensation, pain, and atrophy. Over time, these effects can lead to a significant loss of independence.

Half of the problem is solved

We "just" need to solve the delivery

There are many reasons to be hopeful

New delivery mechanisms

There's an explosion of companies with platform technologies to deliver RNA payloads.
CMT1A is a low-hanging fruit to test if their proprietary technology could deliver to the PNS.

New RNA chemistries

RNAi technology (ASO & siRNA) have gone through several generations of improvements since 2017.
The improvements affect the robustness and the binding affinity.

Inherent lower toxicity

Both of the improvements above will inherently reduce the toxicity of a treatment for CMT1A.
We believe the treatment for CMT1A is already out there, we just need to find it.