公开(公告)号：US20200318138A1

公开(公告)日：2020-10-08

申请号：US16956894

申请日：2018-12-21

申请人： SPARINGVISION ,  INSERM (Institut National de la Santé et Recherche Médicale) ,  SORBONNE UNIVERSITE

发明人： Thierry LEVEILLARD ,  Najate AÏT-ALI MAAMRI ,  Fréderic BLOND ,  José-Alain SAHEL ,  Géraldine PUEL ,  Emmanuelle CLERIN

IPC分类号： C12N15/86 ,  C07K14/435

摘要： The present invention relates to improved constructs comprising the short and long Rod-Derived Cone Viability Factors and to methods for treating retinal degenerative diseases.

公开(公告)号：US20240207451A1

公开(公告)日：2024-06-27

申请号：US18491138

申请日：2023-10-20

申请人： SPARINGVISION ,  RUHR-UNIVERSITÄT BOCHUM ,  INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE ,  CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE ,  SORBONNE UNIVERSITE

发明人： Deniz DALKARA

IPC分类号： A61K48/00 ,  A61K38/17 ,  C07K14/47 ,  C12N15/86

CPC分类号： A61K48/0058 ,  A61K38/1709 ,  A61K38/177 ,  C07K14/47 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145

摘要： The present invention concerns a new gene therapy approach to increase light-sensitivity in degenerating cones in advanced stages of rod-cone dystrophy (RCD) mediated by G-protein-gated inwardly rectifying potassium channel (GIRK), in particular GIRK4 S143T, activated by G proteins recruited by cone opsin expressed in degenerating cones.

公开(公告)号：US20240197913A1

公开(公告)日：2024-06-20

申请号：US18287758

申请日：2022-04-20

申请人： INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) ,  SPARINGVISION ,  CENTRE NATIONAL DE LA RECHERCHE SCIENFITIQUE - CRNS - ,  SORBONNE UNIVERSITE

发明人： Deniz DALKARA ,  Hanen KHABOU ,  Florence LORGET ,  José SAHEL ,  Simon CARDILLA-JOE

IPC分类号： A61K48/00 ,  A61K9/00 ,  A61K38/17 ,  C12N15/86

CPC分类号： A61K48/005 ,  A61K9/0048 ,  A61K38/1709 ,  A61K48/0075 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2830/48

摘要： The present invention relates to the combination of a nucleic acid encoding a short isoform of rod-derived cone viability factor (RdCVF), a nucleic acid encoding a long isoform of rod-derived cone viability factor (RdCVFL) and a nucleic acid encoding a G protein-activated Inward Rectifier potassium channel 2 (GIRK2), expressed through one, two or three viral vectors, said vectors may be within a single pharmaceutical composition or within several different pharmaceutical compositions (two or three). It also deals with the treatment of a retinal degenerative disease, in particular the retinitis pigmentosa, with said viral vectors or pharmaceutical compositions.

公开(公告)号：US20230338581A1

公开(公告)日：2023-10-26

申请号：US18045657

申请日：2022-10-11

申请人： SPARINGVISION

发明人： Deniz DALKARA ,  Cardillia-Joe SIMON ,  Stefan HERLITZE ,  José-Alain SAHEL ,  Isabelle AUDO ,  Serge PICAUD ,  Stéphane Bertin

IPC分类号： A61K48/00 ,  C12N15/86 ,  A61P27/02 ,  A61K38/17

CPC分类号： A61K48/0058 ,  C12N15/86 ,  A61P27/02 ,  A61K38/17 ,  A61K48/0041 ,  C12N2750/14143 ,  C12N2750/14171

摘要： The present invention concerns a new gene therapy approach to increase light-sensitivity in degenerating cones in advanced stages of rod-cone dystrophy (RCD) mediated by G-protein-gated-K+ channel (GIRK), in particular GIRK2, activated by G proteins recruited by cone opsin expressed in degenerating cones for use in the treatment of patients with RCD.