A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 38/47 (2006.01) A61K 9/50 (2006.01) A61P 19/08 (2006.01)
Patent
CA 2715379
Summary Formulation and method for the prevention and treatment of skeletal manifestation of Gaucher's disease The invention relates to the prevention and treatment of skeletal manifestation of Gaucher's disease. It relates to a formulation for introducing glucocerebrosidase into the lysosomal compartment of macrophages and/or Gaucher cells in the bone marrow, the for-mulation comprising glucocerebrosidase encapsulated in erythrocytes. Said erythrocytes may have been subjected to a chemical treatment with an agent so as to promote the targeting of macrophages and/or Gaucher cells in the bone marrow. The treatment may be carried out with a bis(sulphosuccinimidyl) suberate (BS3) solution. The invention also describes drugs and methods for prevention and treatment. No figures.
La présente invention concerne un procédé pour la prévention et le traitement de manifestation squelettique de la maladie de Gaucher. L'invention concerne une formulation permettant d'introduire la glucocérébrosidase dans le compartiment lysosomal de macrophages et/ou de cellules de Gaucher dans la moelle osseuse, la formulation comportant de la glucocérébrosidase encapsulée dans des érythrocytes. Lesdits érythrocytes peuvent avoir été soumis à un traitement chimique avec un agent afin de favoriser le ciblage de macrophages et/ou des cellules de Gaucher dans la moelle osseuse. Le traitement peut être réalisé à l'aide d'une solution de bis(sulfosuccinimidyl) suberate (BS3). L'invention concerne également des médicaments et des procédés pour la prévention et le traitement.
Bourgeaux Vanessa
Dufour Emmanuelle
Godfrin Yann
Blake Cassels & Graydon Llp
Erytech Pharma
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