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.
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
Why there is real reason for optimism
Breakthroughs in delivery and RNA chemistry
New platform technologies are emerging to deliver RNA payloads with far greater precision, while ASO and siRNA chemistries have advanced through multiple generations since 2017. These improvements enhance stability, specificity, and the likelihood of safely reaching the peripheral nervous system.
AI accelerating therapeutic discovery
Modern AI tools now make it possible to design, evaluate, and optimize therapeutic candidates far faster and more accurately than before, helping us identify the most promising approaches faster and at a lower cost.
A uniquely promising moment
The combination of improved delivery, better chemistries, and powerful AI tools expands what is clinically achievable. The solution for CMT1A likely already exists, our mission is to find it and bring it forward.