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New therapeutic peptide for Huntington’s Disease

Reference

04195-01

Patents status

Priority patent application n° FR1153193 filed on April 12, 2011 entitled “Composés thérapeutiques contre la chorée de Huntington”

Inventors

Florence MASCHAT
Marie-Laure PARMENTIER
Nathalie BONNEAUD
Yoan ARRIBAT

commercial status

Exclusive or non-exclusive licenses, Option agreement, Collaborative agreement

Laboratory

Institut de génétique humaine, (IGH, UPR 1142), Montpellier, France. 

Description

CONTEXT

Huntington’s disease (HD) is a neurodegenerative genetic disorder that usually becomes noticeable in middle age. It affects muscle coordination and provokes cognitive decline and dementia. HD is caused by an autosomal dominant mutation of a gene called Huntingtin (Htt) leading to an abnormal repeat number of the amino acid Glutamine (Q) in the protein (PolyQ-Htt). This mutant form of the protein has altered activities and is prone to form protein aggregates. It is toxic to certain types of cells, especially in the brain where neurons are particularly affected.
There is at the present time no cure for HD, and the available treatments aim at reducing the severity of some of its symptoms.

TECHNICAL DESCRIPTION

One therapeutic strategy that is explored is the prevention of aggregates caused by the polyQ domain in mutant Huntingtin. The invention is based on the discovery published in 2008 that the aggregation of PolyQ-Htt may be prevented by its wild-type counterpart.
Considering the size of the wild-type protein, around 600 amino acids, the inventors have looked for smaller but still active peptides isolated from Htt. One of them, pep42, turned out to be particularly interesting.

BENEFITS

The small size of the peptide is more appropriate for a use as a therapeutic compound than the Htt whole protein, while it retains its full anti-aggregates activity.

   Phenotype rescue in a Drosophila HD model

INDUSTRIAL APPLICATIONS

This peptide may be used as a treatment of HD, alone or in combination with other strategies, and administered as a peptide alone or integrated in a lentivirus allowing its expression.

DEVELOPMENT STAGE

In vitro and in vivo data have been obtained and are still in progress. Vectorisation strategies for the peptide are studied. In particular, its fusion with the TAT penetrating sequence has been very efficient to allow its tranbsport trough the blood-brain barrier.
In vivo experiments on R6/2 mouse model shown very promising results.
This work is financially supported by the programme “Emergence of high potential products or services” of the Agence Nationale pour la Recherche.
An Orphan Drug Designation is programmed in 2014.

PUBLICATION

A huntingtin peptide inhibits polyQ-huntingtin associated defects.
Arribat Y, Bonneaud N, Talmat-Amar Y, Layalle S, Parmentier ML, Maschat F.
PLoS One. 2013 Jul 4;8(7):e68775.

For further information, please contact us (Ref 04195-01)

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