New Generation of AAV Vector :rAAV chemically modified capsid

OUEST VALORISATION



12 Juillet 2019

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Fields

Biology / Medical

Sectors

Health

L'innovation


The research team showed that it is possible by chemical coupling to modulate the coupling of a ligand in the surface of the capsid of AAV, for example AAV2 and AAV3b. In particular, the present invention relates to a recombinant Adeno-Associated Virus (rAAV) vector particle having at least one primary amino group contained in the capsid proteins, chemically coupled with at least one ligand L, wherein coupling of said ligand L is implemented through a bond comprising a -CSNH- bond and an optionally substituted aromatic moiety. Particularly, we tested the chemical coupling of mannose ligand on AAV2 for subretinally injection to rats. The present invention further relates to a method for chemically coupling an Adeno-Associated Virus (AAV) vector particle with at least one ligand L and to a Recombinant Adeno-Associated Virus (rAAV) vector particle obtained by said method as well as a pharmaceutical composition comprising it and their corresponding medical use.

Ses bénéfices
- Improvement of therapeutic index,
- Higher specificy (cell or organ)
- Better therapeutic efficiency with lower rAAV doses,
- Limitation of side effects

Ses applications
Gene therapy

Stade de développement
IN VIVO - Preuve de concept

Laboratoire de recherche
UMR 1089 - THERAPIE GÉNIQUE

Équipe de recherche
Thérapie génique pour les maladies de la rétine et les maladies neuromusculaires

Propriété intellectuelle associée
EP : EP16305681.5 - filed on the 06-09-2016
WO - CA,EP,US

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