CONTEXT
Menkès disease is a rare pathology of genetic origin which occurs at a rate of one in 300,000 births, i.e. two to three births each year in France.
It is characterized by a deficiency in ATPase 7A, which allows copper to pass through the intestine and the blood-brain barrier. This leads to a deficiency of cuproproteins, responsible for the severe brain damage of these patients.
Without treatment, their lifespan is less than 3 years and current treatments do not allow the passage of copper to the brain.
DESCRIPTION
The laboratory has developed new copper carriers immobilised on a support in order to make them biocompatible and stable.
Their stability has been tested and is more than 10 weeks.
In vivo, subcutaneous administration of these transporters reverts the Menkès phenotype in models of Menkès mice. At this stage, no toxicity is observed.
COMPETITIVE ADVANTAGES
MARKETS AND APPLICATIONS
Phamaceuceutical
DEVELOPMENT STAGE
TRL 3 in vivo studies in progress
INTELLECTUAL PROPERTY
In progress
TARGET PARTNERSHIP
Patent licensing
CONTACT
Thomas BLUM
Business Developer
+33 (0)6 17 06 68 07
thomas.blum@sayens.fr