公开(公告)号：EP4035658A1

公开(公告)日：2022-08-03

申请号：EP21382062.4

申请日：2021-01-27

申请人： Consejo Superior de Investigaciones Científicas (CSIC) ,  Consorcio Centro de Investigación Biomédica en Red M.P. ,  Fundació Hospital Universitari Vall d'Hebron - Institut de Recerca ,  Nanomol Technologies, S.L. ,  Leanbio

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

IPC分类号： A61K9/127 ,  A61K35/00 ,  A61K47/42 ,  A61K48/00

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.