Patent classifications
A61P21/04
COMBINATION OF IAP INHIBITORS AND PARP OR MEK INHIBITORS OR OTHER CHEMOTHERAPEUTIC AGENTS
A pharmaceutical composition comprising: a) an effective amount of an Inhibitors of Apoptosis Proteins (IAP) inhibitor, wherein the IAP inhibitor is represented by formula (I): or a pharmaceutically acceptable salt thereof, the definitions of each variable are provided herein; b) an effective amount of a second inhibitor, wherein the second inhibitor is a poly ADP ribose polymerase (PARP) inhibitor or a mitogen-activated protein kinase kinase (MEK) inhibitor; and a pharmaceutically acceptable carrier or diluent.
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METHODS OF USING ZSCAN4 FOR REJUVENATING HUMAN CELLS
The present disclosure relates to methods for increasing telomere length in one or more human cells and/or increasing genome stability of one or more human cells, for example by contacting one or more human cells with an agent that increases expression of Zscan4 in the one or more human cells. Methods of treating a subject in need of telomere lengthening, treating a disease or condition associated with a genomic and/or chromosome abnormality, of rejuvenating one or more human cells, of rejuvenating tissues or organs, and of rejuvenating a subject in need thereof, for example by contacting one or more human cells in the subject with an agent that increases expression of Zscan4, or by administering to a subject in need thereof, an agent that increases expression of Zscan4 are also provided.
Indole compounds and analogues thereof
Disclosed are compounds of formula I, and pharmaceutically acceptable salts thereof. The compounds are inhibitors of the complement system. Also provided are pharmaceutical compositions comprising a compound of formula I, and methods involving use of the compounds and compositions in the treatment and prevention of diseases and conditions characterized by aberrant complement system activity. ##STR00001##
Indole compounds and analogues thereof
Disclosed are compounds of formula I, and pharmaceutically acceptable salts thereof. The compounds are inhibitors of the complement system. Also provided are pharmaceutical compositions comprising a compound of formula I, and methods involving use of the compounds and compositions in the treatment and prevention of diseases and conditions characterized by aberrant complement system activity. ##STR00001##
Methods for modulating RNA splicing
In one aspect, described herein is an intronic recognition element for splicing modifier (iREMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains an intronic REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises an intronic REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising an intronic REMS, and uses of those artificial gene constructs to modulate protein production. In another aspect, provided herein are methods for altering endogenous genes to comprise an intronic REMS, and the use of a compound described herein to modulate protein produced from such altered endogenous genes.
COMPOSITIONS FOR AN ORALLY ACTIVE 1,2,4-OXADIAZOLE FOR THE TREATMENT OF DISEASE
The present invention relates to specific doses of and dosing regimens for using a 1,2,4-oxadiazole benzoic acid compound in treating or preventing diseases associated with nonsense mutations. In particular, the invention relates to specific doses and dosing regimens for the use of 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid in mammals having diseases associated with nonsense mutations.
BENZOPYRAZOLE COMPOUNDS AND ANALOGUES THEREOF
Disclosed are compounds of formula I, and pharmaceutically acceptable salts thereof. The compounds are inhibitors of the complement system. Also provided are pharmaceutical compositions comprising a compound of formula I, and methods involving use of the compounds and compositions in the treatment and prevention of diseases and conditions characterized by aberrant complement system activity.
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BENZOPYRAZOLE COMPOUNDS AND ANALOGUES THEREOF
Disclosed are compounds of formula I, and pharmaceutically acceptable salts thereof. The compounds are inhibitors of the complement system. Also provided are pharmaceutical compositions comprising a compound of formula I, and methods involving use of the compounds and compositions in the treatment and prevention of diseases and conditions characterized by aberrant complement system activity.
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Methods and compositions for immunomodulation
Provided are cells containing exogenous antigen and uses thereof.
BORON CONTAINING SMALL MOLECULES AS ANTI-INFLAMMATORY AGENTS
Methods of treating anti-inflammatory conditions through the use of boron-containing small molecules are disclosed.