Medesis Pharma has received a grant of 300,000 euros from AFM-Téléthon to fund the first phases of preclinical development of its RNA interference treatment for Huntington’s disease, the company announced.
Huntington’s disease is a rare, hereditary neurodegenerative disease that causes motor, cognitive and psychiatric dysfunction. Affected individuals eventually become bedridden and develop severe intellectual deterioration, the company said in a press release. Current treatments are symptomatic.
The fatal disease is caused by mutations within an increased number of CAG trinucleotide repeats in the HTT gene, which is essential in the creation of a protein called huntingtin. Alleles with low numbers of repeats are wild type, while those with larger numbers of repeats are mutant.
According to the release, patients with Huntington’s disease are heterozygous, meaning they have a mutant, dominant allele and a wild-type allele. Because the mutant allele is dominant, efficient therapies must be repressive.
The Medesis NanosiRNA-HD treatment in development will use small interfering RNA, which will specifically target a different nucleotide on the mutated gene and make it possible to reduce the production of the mutated protein. The first targeted nucleotide could be used in 50% of patients, the release stated.
The prevalence of Huntington’s disease is eight cases per 100,000 individuals globally, the company said. It is estimated that nearly 100,000 people have the disease, and approximately 200,000 people carry the abnormal gene.
