C - Chemistry – Metallurgy – 07 – C
Patent
C - Chemistry, Metallurgy
07
C
C07C 69/68 (2006.01) A61K 31/22 (2006.01) C07C 69/347 (2006.01)
Patent
CA 2233606
This invention relates to butyrate prodrugs derived from lactic acid and pharmaceutical compositions and methods employing them, either alone or in combination with other agents, for increasing gamma globin and fetal hemoglobin in a patient. These compounds, compositions and methods are particularly effective in treating .beta.-hemoglobinopathies, including sickle cell syndromes and .beta.-thalassemia syndromes. In addition, this invention relates to the use of these prodrugs, alone or in combination with other agents, to stimulate cell differentiation which prevents proliferation of malignant cells. These methods are particularly useful in treating cancer, especially malignant hematological disorders.
Promédicaments à base de butyrate dérivé d'acide lactique, compositions pharmaceutiques et méthodes d'utilisation de ces compositions, soit seules soit en combinaison avec d'autres agents, pour accroître la gamma globine et l'hémoglobine foetale chez la patiente. Ces composés, ces compositions et ces méthodes sont particulièrement efficaces pour le traitement des hémoglobinopathies bêta, notamment des drépanocytoses et de la thalassémie bêta. L'invention porte en outre sur l'utilisation de ces promédicaments, seuls ou en combinaison avec d'autres agents, pour stimuler la différenciation cellulaire afin de prévenir la prolifération de cellules malignes. Ces méthodes sont particulièrement utiles pour le traitement du cancer, notamment des affections hématologiques malignes.
Li Biqin
Tung Roger D.
Smart & Biggar
Vertex Pharmaceuticals Incorporated
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