Project description
A novel molecular therapy against myotonic dystrophy
Myotonic dystrophy type 1 is an inherited muscular dystrophy that is characterised by muscle weakness and wasting as well as prolonged muscle tensing. Recent evidence indicates altered microRNA expression such as upregulation of the microRNA-23b which plays a key role in the translation of muscle-related proteins. Funded by the European Innovation Council, the fight-dm1 project will bring forward a novel molecular therapy based on synthetic single-stranded oligonucleotides called antimiRs. These molecules have been designed to be complementary to that of microRNA-23b so that they can block its function or degrade it. Following promising preclinical efficacy, the patented technology will be validated in a patient study.
Objective
Myotonic dystrophy type 1 (DM1) is a clinically and genetically heterogeneous disorder with more than 1 million diagnosed patients worldwide, making it the most common adult muscular dystrophy. Available treatments only relief symptoms. Given this need, we offer ATX-01, a first in class miRNA therapy that inhibits miR-23b without changing the DNA, which rescues DM1 pathogenic mis-splicing. We have demonstrated improved target engagement effects in skeletal and respiratory muscle in in-vitro and in-vivo studies, granting us an FDA pre-IND approval and orphan drug designation. Our approach, antimiRs conjugated to fatty acids, constitutes Arthex's technology platform, which guarantees efficient drug delivery to target muscular and extra-muscular tissue at low doses. This patented technology will be validated through a Phase I/IIa clinical trial and chronic preclinical studies, to then be extended to applications as other myotonic dystrophies, Fuchs dystrophy, cachexia, among other opportunities.
Fields of science
Programme(s)
- HORIZON.3.1 - The European Innovation Council (EIC) Main Programme
Funding Scheme
EIC-ACC - EIC-ACCCoordinator
46980 Valencia
Spain
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.