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.
The teams particularly 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.
UMR 1089 - THERAPIE GÉNIQUE
Thérapie génique pour les maladies de la rétine et les maladies neuromusculaires
EP : EP16305681.5 - filed on the 06-09-2016
WO - CA,EP,JP,US,US