CONNECTOMICA

About us

We are a company focused on the development of drugs to treat connexin-related pathologies with special interest in Duchenne Muscular Distrophy (DMD). Our research is focused on AI-driven developement of small molecules to block Connexin hemichannels and prevent the diseases born from its uncontrolled expression.

Duchenne Muscular Distrophy (DMD)

DMD is a progressive disorder triggered by a mutation in the dystrophin gene which leades to muscular atrophy. This disease affects mainly males as it’s X-linked, and affects around 16-20 per 100,000 of them making it an orphan disease.

The disease first manifests with walking problems around the age of 3 and progresses to the need of a wheel chair by the age of 10. At 15-years old kids start to loose arms movility and 3-5 years lates a mechanical ventilator is needed. Life expectancy of DMD patients is of 25 years.

At the moment there’s no cure for DMD, only paliative treatments are available. Actual care practices consists of anti-inflammatories, which have long term issues, and RNA therapies that are only effective in a very small percentage of the population. All this treatments have an estimate cost of US$19,000 per pacient in USA only, with an approximately worldwide expenditure of US$2 billions.