C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 9/88 (2006.01) A61K 38/51 (2006.01) A61P 3/00 (2006.01) C07K 14/375 (2006.01)
Patent
CA 2580679
The present invention pertains to the use of the protein phenylalanine ammonia- lyase, as well as the biologically-active derivatives of the said protein for preventing or treating diseases associated with a phenylalanine imbalance in a human or animal body. More particularly, the present invention relates to the therapeutic use of the above-cited molecules for preventing or treating a phenylalanine imbalance in vivo. This invention also deals with therapeutic compositions comprising a pharmaceutically active amount of the above- described therapeutic molecules as well as with therapeutic methods using the said therapeutic compositions. Finally, the present invention relates to processes for selecting more therapeutically-effective variants of said protein as well as to the selected variants themselves.
La présente invention a trait à l'utilisation de la protéine phénylalanine ammonia-lyase, ainsi qu'à des dérivés biologiquement actifs de ladite protéine pour la prévention ou le traitement de maladies associées à un déséquilibre de phénylalanine dans un organisme humain ou animal. Plus particulièrement, l'invention a trait à l'utilisation thérapeutique desdites molécules pour la prévention ou le traitement d'un déséquilibre de phénylalanine in vivo. L'invention a également trait à des compositions thérapeutiques comportant une quantité pharmaceutiquement active desdites molécules thérapeutiques ainsi qu'à des procédés thérapeutiques mettant en oeuvre lesdites compositions thérapeutiques. L'invention a trait enfin à procédés pour la sélection de variants d'efficacité thérapeutique supérieure de ladite protéine ainsi qu'aux variants sélectionnés eux-mêmes.
Fitzpatrick Paul A.
Gamez Alejandra
Heft Robert
Kakkis Emil
Kim Woomi
Biomarin Pharmaceutical Inc.
Smart & Biggar
The Scripps Research Institute
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