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New therapeutic peptide for Huntington’s Disease



Statut des brevets

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


Florence MASCHAT

Statut commercial

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


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



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.


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.


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


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.


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.


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.

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