公开(公告)号：US20240115502A1

公开(公告)日：2024-04-11

申请号：US18274631

申请日：2022-01-26

申请人： CONSEJO SUPERIOR DE INVESTIGACIONES CIENTIFICAS (CSIC) ,  NANOMOL TECHNOLOGIES, S.L. ,  LEANBIO

发明人： Leonor VENTOSA RULL ,  Jaume VECIANA MIRÓ ,  Miriam ROYO EXPOSITO ,  Judit TOMSEN MELERO ,  Ibane ABASOLO OLAORTUA ,  Simón SCHWARTZ NAVARRO ,  Jose Luis CORCHERO NIETO ,  Daniel PULIDO MARTINEZ ,  Edgar CRISTÓBAL LECINA ,  Elisabet GONZÁLEZ MIRA ,  Santiago SALA VERGÉS ,  Alba CÓRDOBA INSENSÉ ,  Josep MERLO MAS ,  Andreu SOLDEVILA FÁBREGA ,  Albert FONT INGLÉS

IPC分类号： A61K9/127 ,  A61K38/47 ,  A61K47/26 ,  A61P3/06

CPC分类号： A61K9/1272 ,  A61K9/1277 ,  A61K38/47 ,  A61K47/26 ,  A61P3/06 ,  C12Y302/01022

摘要： The present invention refers to a liposome comprising: a) a phospholipid; b) cholesterol (chol); c) a conjugate comprising a cholesterol moiety, a polyethylene glycol (PEG) moiety and a peptide moiety comprising a RGD sequence, wherein the PEG moiety is covalently attached to the cholesterol moiety by one end via a bond of the type alkyl ether and is covalently attached to the peptide moiety comprising the RGD sequence by the other end: d) a non-lipid cationic surfactant present in a percentage of less than 30% mol in respect to the total mol of the components of the liposome a), b), c) and d); and e) alpha-galactosidase (GLA) enzyme present in a ratio of micrograms of GLA in respect to the total milligrams of the components of the liposome a), b), c) and d) of between and including 2 to 35. It also refers to a pharmaceutical composition that comprises it and to the liposome or the pharmaceutical composition for use as a medicament, in particular for use in the treatment of Fabry disease. It also refers to a process for the production of the liposome.