Factor ix variants with clotting activity in absence of...

C - Chemistry – Metallurgy – 12 – N

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C12N 9/64 (2006.01)

Patent

CA 2729501

The present invention relates to variants of a vitamin K-dependent serine protease of the coagulation cascade, preferably variants of factor IX (F.IX), wherein the variant is characterized in that it has clotting activity in absence of its cofactor. The present invention furthermore relates to the use of these variants for the treatment and/or prophylaxis of bleeding disorders, in particular hemophilia A and/or hemophilia B or hemophilia caused or complicated by inhibitory antibodies to F.VIII. The present invention also relates to further variants of factor IX (F.IX) which have desired properties and can, thus be tailored for respective specific therapeutic applications.

La présente invention concerne des variants d'une sérine protéase vitamine K-dépendante de la série des réactions en cascade de la coagulation sanguine, de préférence des variants du facteur IX (F. IX), lesdits variants étant caractérisés en ce qu'ils présentent une activité coagulante en l'absence de leur cofacteur. La présente invention concerne, en outre, l'utilisation de ces variants pour le traitement et/ou la prophylaxie de troubles hémorragiques, en particulier de l'hémophilie A et/ou de l'hémophilie B ou, encore, de l'hémophilie provoquée ou compliquée par des anticorps inhibiteurs dirigés contre le F. VIII. La présente invention concerne, en outre, d'autres variants du facteur IX (F. IX), présentant des propriétés recherchées et pouvant, donc, être adaptés à diverses applications thérapeutiques particulières.

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