US 11,865,189 B2
Transgenic RPE cells overexpressing OTX2 for the treatment of retinal degeneration
Thierry Leveillard, Maison-Alfort (FR); Christo Kole, Chatenay Malabry (FR); and Jose-Alain Sahel, Paris (FR)
Assigned to SORBONNE UNIVERSITE, Paris (FR); CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE, Paris (FR); and INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE), Paris (FR)
Appl. No. 15/536,725
Filed by SORBONNE UNIVERSITE, Paris (FR); CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE, Paris (FR); and INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE), Paris (FR)
PCT Filed Dec. 17, 2015, PCT No. PCT/EP2015/080288
§ 371(c)(1), (2) Date Jun. 16, 2017,
PCT Pub. No. WO2016/097183, PCT Pub. Date Jun. 23, 2016.
Claims priority of application No. 14307069 (EP), filed on Dec. 18, 2014.
Prior Publication US 2017/0348434 A1, Dec. 7, 2017
Int. Cl. A61K 48/00 (2006.01); C12N 5/079 (2010.01); C12N 15/864 (2006.01); A61K 35/12 (2015.01); C12N 7/00 (2006.01); C12N 5/16 (2006.01)
CPC A61K 48/0058 (2013.01) [A61K 35/12 (2013.01); A61K 48/00 (2013.01); C12N 5/0621 (2013.01); C12N 5/16 (2013.01); C12N 7/00 (2013.01); C12N 15/8645 (2013.01); C12N 2510/00 (2013.01); C12N 2750/14141 (2013.01)] 18 Claims
 
1. A method of treating retinitis pigmentosa in a mammal having retinitis pigmentosa, said method comprising the intraocular administration, by injection into the subretinal space of the eye of said mammal, of mammalian primary retinal pigment epithelial (RPE) cells or induced pluripotent stem cell-derived RPE cells overexpressing mammalian orthodenticle homeobox 2 protein (OTX2 protein), thereby increasing intracellular levels of OTX2 protein in the mammal, wherein the RPE cells overexpress OTX2 protein and comprise an AAV2 viral vector comprising a nucleic acid sequence encoding an OTX2 protein comprising SEQ ID NO: 15 or SEQ ID NO: 16.