IMMUNSUPPRESSIVE PROTEIN GOALS

Modulators of alpha-1 antitrypsin

Novel compounds, compositions, and methods of using and preparing the same, which may be useful for treating alpha-1 antitrypsin deficiency (AATD).

IMMUNSUPPRESSIVE PROTEINZIELE

TEST AND REAGENTS FOR THE IDENTIFICATION OF ANTI-PROLIFERATING MATERIALS

Cell-cycle regulatory proteins from human pathogens, and use s related thereto

Reagents and method for modulating Dkk-mediated interactions

The present invention provides reagents, compounds, compositions, and methods relating to novel interactions of the extracellular domain of LRP5, HBM (a variant of LRP5), and/or LRP6 with Dkk, including Dkk-1. The various nucleic acids, polypeptides, antibodies, assay methods, diagnostic methods, and methods of treatment of the present invention are related to and impact on Dkk, LRP5, LRP6, HBM, and Wnt signaling. Dkk, LRP5, LRP6, HBM, and Wnt are implicated in bone and lipid cellular signaling. Thus, the present invention provides reagents and methods for modulating lipid levels and/or bone mass and is useful in the treatment and diagnosis of abnormal lipid levels and bone mass disorders, such as osteoporosis.

TEST UND REAGENTIEN ZUR IDENTIFIZIERUNG VON ANTIPROLIFERIERENDEN STOFFEN

IMMUNOSUPPRESSIVE PROTEIN GOALS

Assays and reagents for identifying anti-fungal agents, and uses related thereto

Assay and reagents for identifying anti-proliferative agents

Cyclin activation kinase (cak) from human pathogens, and uses related thereto

MODULATORS OF ALPHA-1 ANTITRYPSIN

Novel compounds, compositions, and methods of using and preparing the same, which may be useful for treating alpha-1 antitrypsin deficiency (AATD).

Immunosuppressive Proteinziele

Assays and reagents for identifying anti-fungal agents, and uses related thereto

IMMUNOSUPPRESSANT TARGET PROTEINS

The present invention relates to the discovery of novel proteins of mammalian origin which are immediate downstream targets for FKBP/rapamycin complexes.

ASSAY AND REAGENTS FOR IDENTIFYING ANTI-PROLIFERATIVE AGENTS

The present invention makes available assays and reagents for identifying antiproliferative agents, such as mitotic and meiotic inhibitors. The present assay provides a simple and rapid screening test which relies on scoring for positive cellular proliferation as indicative of anti-mitotic or anti-meiotic activity, and comprises contacting a candidate agent with a cell which has an impaired cell-cycle checkpoint and measuring the level of proliferation in the presence and absence of the agent. The checkpoint impairment is such that it either causes premature progression of the cell through at least a portion of a cell-cycle or inhibition of normal progression of the cell through at least a portion of a cell-cycle, but can be offset by the action of an agent which inhibits at least one regulatory protein of the cell cycle (e.g., cdc25) in a manner which counterbalances the effect of the impairment.

Immunosuppressant target proteins

CONDENSED TRYCICLIC PYRROLES AS ALPHA-1 ANTITRYPSIN MODULATORS

The disclosure provides compounds useful for treating alpha-1 antitrypsin deficiency (AATD), according to formula (I), tautomers thereof, pharmaceutically acceptable salts of the compounds, pharmaceutically acceptable salts of the tautomers, deuterated derivatives of the compounds, deuterated derivatives of the tautomers, and deuterated derivatives of the salts, solid forms of those compounds and processes for making those compounds.

Modulators of alpha-1 antitrypsin

Novel compounds, compositions, and methods of using and preparing the same, which may be useful for treating alpha-1 antitrypsin deficiency (AATD).

Immunosuppressant target proteins

Immunosuppressant target proteins

Immunosuppressant target proteins

ASSAY AND REAGENTS FOR IDENTIFYING ANTI-PROLIFERATIVE AGENTS

The present invention makes available assays and reagents for identifying antiproliferative agents, such as mitotic and meiotic inhibitors. The present assay provides a simple and rapid screening test which relies on scoring for positive cellular proliferation as indicative of anti-mitotic or anti-meiotic activity, and comprises contacting a candidate agent with a cell which has an impaired cell-cycle checkpoint and measuring the level of proliferation in the presence and absence of the agent. The checkpoint impairment is such that it either causes premature progression of the cell through at least a portion of a cell-cycle or inhibition of normal progression of the cell through at least a portion of a cell-cycle, but can be off- set by the action of an agent which inhibits at least one regulatory protein of the cell cycle (e.g., cdc25) in a manner which counter- balances the effect of the impairment.

Assays and reagents for identifying anti-fungal agents, and uses related thereto

IMMUNOSUPPRESSANT TARGET PROTEINS

The present invention relates to the discovery of novel proteins of mammalian origin which are immediate downstream targets for FKBP/rapamycin complexes.

IMMUNSUPPRESSIVE PROTEINZIELE

TEST UND REAGENTIEN ZUR IDENTIFIZIERUNG VON ANTIPROLIFERIERENDEN STOFFEN

Assays and reagents for identifying anti-fungal agents, and uses related thereto

Assay and reagents for identifying anti-proliferative agents

Reagents and methods for modulating Dkk-mediated interactions

MODULADORES DE LA a-1 ANTITRIPSINA

Compuestos, composiciones y métodos de uso y preparación de los mismos, que pueden ser útiles para el tratamiento de la deficiencia de a-1 antitripsina (AATD). Un compuesto de la fórmula (1), un tautómero del mismo, una sal farmacéuticamente aceptable de cualquiera de los anteriores, o un derivado deuterado de cualquiera de los anteriores; caracterizado porque: (i) R⁰ es elegido de (a) grupos alquilo C₁₋₈ lineales, ramificados y cíclicos, en donde el grupo alquilo es opcionalmente sustituido con 1 - 4 RA; y (b) anillos aromáticos de 5 a 10 miembros opcionalmente sustituidos con 1 - 4 RA, en donde cada RA es elegido independientemente de halógenos, ciano, hidroxi, tiol, ácido sulfúrico, sulfonamida, sulfinamida, amino, amida, anillos aromáticos de 5 a 10 miembros, y grupos C₁₋₆ lineales, ramificados y cíclicos, en donde los grupos C₁₋₆ lineales, ramificados y cíclicos son elegidos de alquilo, alcoxi, tioalquilo, alquilsulfóxido, alquilsulfonilo, alquilsulfonamida, alquilsulfinamida, aminoalquilo, y alquilamida, y en donde los anillos aromáticos de 5 a 10 miembros y los grupos C₁₋₆ lineales, ramificados y cíclicos son opcionalmente sustituidos con 1 - 4 sustituyentes seleccionados de halógenos y metoxi; (ii) R¹ es elegido de (a) grupos alquilo C₁₋₈ lineales, ramificados y cíclicos, en donde el grupo alquilo es opcionalmente sustituido con 1 - 4 sustituyentes elegidos de halógenos, ácido carboxílico, ciano, y grupos C₁₋₆ lineales, ramificados y cíclicos, en donde los grupos C₁₋₆ lineales, ramificados y cíclicos son elegidos de grupos alquilo y alcoxi, y en donde los grupos C₁₋₆ lineales, ramificados y cíclicos son opcionalmente sustituidos con 1 - 4 halógenos, (b) grupos alcoxi C₁₋₈ lineales, ramificados y cíclicos opcionalmente sustituidos con 1 - 4 sustituyentes elegidos de halógenos, ciano, y grupos alquilo C₁₋₆ lineales, ramificados y cíclicos que son opcionalmente sustituidos con 1 - 4 halógenos; (c) heterociclos C₁₋₈, y (d) grupos alquenilo C₂₋₆ lineales, ...

Modulators of alpha-1 antitrypsin

The disclosure provides compounds useful for treating alpha-1 antitrypsin deficiency (AATD), according to formula (I): tautomers thereof, pharmaceutically acceptable salts of the compounds, pharmaceutically acceptable salts of the tautomers, deuterated derivatives of the compounds, deuterated derivatives of the tautomers, and deuterated derivatives of the salts, solid forms of those compounds and processes for making those compounds.

Assays and reagents for identifying anti-fungal agents, and uses related thereto

The present invention relates to rapid, reliable and effective assays for screening and identifying pharmaceutically effective compounds that specifically inhibit the biological activity of fungal GTPase proteins, particularly GTPases involved in cell wall integrity, hyphael formation, and/or other cellular functions critical to pathogenesis. Another aspect of the present invention relates to novel Candida genes and gene products.

REAGENT AND METHOD FOR MODULATING Dkk-MEDIATED INTERACTION

PROBLEM TO BE SOLVED: To provide an additional research tool for elucidating the molecular mechanism of bone modulation, for screening and development of a candidate new drug, and for treatment of bone developmental and bone loss disorders. SOLUTION: A reagent, a compound, a composition, and a method related to new interactions of the extracellular domain of LRP5, HBM (a variant of the LRP5), and/or LRP6 with Dkk, including Dkk-1 are provided. A specific nucleic acid, a polypeptide, an antibody, an assay method, a diagnostic method, and a treating method are related to and impact on Dkk, LRP5, LRP6, HBM, and Wnt signaling. The Dkk, LRP5, LRP6, HBM, and Wnt are implicated in bone and lipid cellular signaling. Thus, the reagent and the method for modulating lipid levels and/or bone mass are useful in treating and diagnosing abnormal lipid levels and bone mass disorders such as osteoporosis. COPYRIGHT: (C)2009,JPO&INPIT ...

Immunosuppressant target proteins

The present invention relates to the discovery of novel proteins of mammalian origin which are immediate downstream targets for FKBP/rapamycin complexes.

Candida geranylgeranyl-protein transferase polypetide, compositions and methods related thereto

The present invention relates to rapid, reliable and effective assays for screening and identifying pharmaceutically effective compounds that specifically inhibit the biological activity of fungal GTPase proteins, particularly GTPases involved in cell wall integrity, hyphael formation, and/or other cellular functions critical to pathogenesis. Another aspect of the present invention relates to novel Candida genes and gene products.

Assays and reagents for identifying anti-fungal agents, and uses related thereto

The present invention relates to rapid, reliable and effective assays for screening and identifying pharmaceutically effective compounds that specifically inhibit the biological activity of fungal GTPase proteins, particularly GTPases involved in cell wall integrity, hyphael formation, and/or other cellular functions critical to pathogenesis. Another aspect of the present invention relates to novel Candida genes and gene products.

Nucleic acids for identifying anti-fungal agents, and uses related thereto

The present invention relates to rapid, reliable and effective assays for screening and identifying pharmaceutically effective compounds that specifically inhibit the biological activity of fungal GTPase proteins, particularly GTPases involved in cell wall integrity, hyphael formation, and/or other cellular functions critical to pathogenesis. Another aspect of the present invention relates to novel Candida genes and gene products.

Reagents and method for modulating Dkk-mediated interactions

The present invention provides reagents, compounds, compositions, and methods relating to novel interactions of the extracellular domain of LRP5, HBM (a variant of LRP5), and/or LRP6 with Dkk, including Dkk-1. The various nucleic acids, polypeptides, antibodies, assay methods, diagnostic methods, and methods of treatment of the present invention are related to and impact on Dkk, LRP5, LRP6, HBM, and Wnt signaling. Dkk, LRP5, LRP6, HBM, and Wnt are implicated in bone and lipid cellular signaling. Thus, the present invention provides reagents and methods for modulating lipid levels and/or bone mass and is useful in the treatment and diagnosis of abnormal lipid levels and bone mass disorders, such as osteoporosis.

MODULATORS OF ALPHA-1 ANTITRYPSIN

The disclosure provides compounds useful for treating alpha-1 antitrypsin deficiency (AATD), according to formula (I): 8. Compound 33 in substantially crystalline form , wherein the crystalline form is selected from Compound 33 Form A , Compound 33 Form B , Compound 33 DCM Solvate Form A , Compound 33 Hydrate Form A , Compound 33 MeOH/HO Solvate/Hydrate Form A , Compound 33 Form C , Compound 33 Form D , Compound 33 Form E , Compound 33 Form F , Compound 33 Form G , Compound 33 Form H , Compound 33 Form I , Compound 33 THF Solvate Form A , Compound 33 Form J , Compound 33 Form K , Compound 33 Form L , Compound 33 2-MeTHF Solvate Form A , Compound 33 Form M , Compound 33 Form N , Compound 33 Form O , Compound 33 Potassium Salt Form A , Compound 33 Potassium Salt Form B , and Compound 33 Potassium Salt Form C.9. Substantially amorphous Compound 33.10. A solid dispersion comprising substantially amorphous Compound 33 and a polymer.11. The solid dispersion comprising substantially amorphous Compound 33 according to claim 10 , wherein the polymer is selected from polyvinylpyrrolidone/vinyl acetate (PVPVA) claim 10 , hydroxypropylmethylcellulose (HPMC) claim 10 , and hydroxypropylmethylcellulose acetate succinate (HPMCAS).12. The solid dispersion comprising substantially amorphous Compound 33 according to claim 10 , wherein substantially amorphous Compound 33 is present in an amount from 50-80%.13. The solid dispersion comprising substantially amorphous Compound 33 according to prepared as a spray-dried dispersion.14. Spray-dried neat amorphous Compound 33.15. A pharmaceutical composition comprising the compound claim 1 , tautomer claim 1 , salt claim 1 , or deuterated derivative according to .16. A pharmaceutical composition comprising the substantially crystalline Compound 33 according to .17. A pharmaceutical composition comprising the substantially amorphous Compound 33 according to .18. A pharmaceutical composition comprising the spray-dried neat amorphous Compound 33 ...

Assays and reagents for identifying anti-fungal agents, and uses related thereto

The present invention relates to rapid, reliable and effective assays for screening and identifying pharmaceutically effective compounds that specifically inhibit the biological activity of fungal GTPase proteins, particularly GTPases involved in cell wall integrity, hyphael formation, and/or other cellular functions critical to pathogenesis. Another aspect of the present invention relates to novel Candida genes and gene products.

Assay and reagents for identifying anti-proliferative agents

The present invention makes available assays and reagents for identifying anti-proliferative agents, such as mitotic and meiotic inhibitors. The present assay provides a simple and rapid screening test which relies on scoring for positive cellular proliferation as indicative of anti-mitotic or anti-meiotic activity, and comprises contacting a candidate agent with a cell which has an impaired cell-cycle checkpoint and measuring the level of proliferation in the presence and absence of the agent. The checkpoint impairment is such that it either causes premature progression of the cell through at least a portion of a cell-cycle or inhibition of normal progression of the cell through at least a portion of a cell-cycle, but can be off-set by the action of an agent which inhibits at least one regulatory protein of the cell-cycle in a manner which counter-balances the effect of the impairment.

Immunosuppressant target proteins

The present invention relates to the discovery of novel proteins of mammalian origin which are immediate downstream targets for FKBP/rapamycin complexes and provides, e.g., isolated polypeptides, nucleic acids encoding such, antibodies, screening methods, and diagnostic and therepeutic methods.

MODULATORS OF ALPHA-1 ANTITRYPSIN

Novel compounds, compositions, and methods of using and preparing the same, which may be useful for treating alpha-1 antitrypsin deficiency (AATD). 28-. (canceled)10. The compound of claim 1 , a tautomer thereof claim 1 , a pharmaceutically acceptable salt of the compound or the tautomer claim 1 , or a deuterated derivative of the compound claim 1 , the tautomer claim 1 , or the salt claim 1 , wherein at least one of Z claim 1 , Z claim 1 , and Zis nitrogen.11. The compound of claim 10 , a tautomer thereof claim 10 , a pharmaceutically acceptable salt of the compound or the tautomer claim 10 , or a deuterated derivative of the compound claim 10 , the tautomer claim 10 , or the salt claim 10 , wherein two of Z claim 10 , Z claim 10 , and Zare nitrogen and the other is chosen from carbon and nitrogen.15. (canceled)17. (canceled)19. (canceled)21. A composition comprising at least one compound chosen from the compounds according to claim 1 , pharmaceutically acceptable salts of any of the foregoing claim 1 , and deuterated derivatives of any of the foregoing.2324-. (canceled)2631-. (canceled)32. Compound 32 in the form of a monohydrochloride salt claim 1 , hemihydrochloride hemihydrate claim 1 , an amorphous form claim 1 , or ethanol solvate.3336-. (canceled)37. A composition comprising the Amorphous Form of Compound 32 claim 32 , the Compound 32 monohydrochloride salt claim 32 , the Compound 32 hemihydrochloride hemihydrate claim 32 , the and/or Compound 32 ethanol solvate according to .38. A method of treating alpha-1 antitrypsin deficiency comprising administering to a patient in need thereof a therapeutically effective amount of at least one compound according to .39. The method of claim 38 , wherein said therapeutically effective amount of the at least one compound is administered in combination with AAT augmentation therapy and/or AAT replacement therapy.4042-. (canceled)4548-. (canceled)50. (canceled)52. (canceled) This application claims the benefit of U.S. ...

ALPHA-1 ANTITRYPSIN MODULATORS

Alpha-1 antitrypsin modulators

Nuevos compuestos, composiciones y métodos para usar y preparar los mismos, los cuales pueden ser útiles para tratar la deficiencia de alfa-1 antitripsina (AATD). New compounds, compositions, and methods for using and preparing the same, which may be useful in treating alpha-1 antitrypsin deficiency (AATD).

Indole derivatives as alpha-1-antitrypsin modulators for treating alpha-1-antitrypsin deficiency (aatd)

Indole derivatives as alpha-1-antitrypsin modulators for treating alpha-1-antitrypsin deficiency (AATD).

Modulators of alpha-1 antitrypsin

Pyrano[4,3-b]indole derivatives as alpha-1-antitrypsin modulators for treating alpha-1-antitrypsin deficiency (AATD)

Indole derivatives as alpha-1 -antitrypsin modulators for treating alpha-1 -antitrypsin deficiency (aatd)

Indole derivatives as alpha-l-antitrypsin modulators for treating alpha-l-antitrypsin deficiency (AATD).

Reagents and methods for modulating dkk-mediated interactions

The present invention provides reagents, compounds, compositions, and methods relating to novel interactions of the extracellular domain of LRP5, HBM (a variant of LRP5), and/or LRP6 with Dkk, including Dkk-1. The various nucleic acids, polypeptides, antibodies, assay methods, diagnostic methods, and methods of treatment of the present invention are related to and impact on Dkk, LRP5, LRP6, HBM, and Wnt signaling. Dkk, LRP5, LRP6, HBM, and Wnt are implicated in bone and lipid cellular signaling. Thus, the present invention provides reagents and methods for modulating lipid levels and/or bone mass and is useful in the treatment and diagnosis of abnormal lipid levels and bone mass disorders, such as osteoporosis.

Cell-cycle regulatory proteins from human pathogens, and uses related thereto

The present invention relates to the discovery of novel cell cycle regulatory proteins from the human pathogen Candida.

Reagents and methods for modulating dkk-mediated interactions

The present invention provides reagents, compounds, compositions, and methods relating to novel interactions of the extracellular domain of LRP5, HBM (a variant of LRP5), and/or LRP6 with Dkk, including Dkk-1. The various nucleic acids, polypeptides, antibodies, assay methods, diagnostic methods, and methods of treatment of the present invention are related to and impact on Dkk, LRP5, LRP6, HBM, and Wnt signaling. Dkk, LRP5, LRP6, HBM, and Wnt are implicated in bone and lipid cellular signaling. Thus, the present invention provides reagents and methods for modulating lipid levels and/or bone mass and is useful in the treatment and diagnosis of abnormal lipid levels and bone mass disorders, such as osteoporosis.

Modulators of alpha-1 antitrypsin

Novel compounds, compositions, and methods of using and preparing the same, which may be useful for treating alpha-1 antitrypsin deficiency (AATD).

Reagents and methods for modulating dkk-mediated interactions

The present invention provides reagents, compounds, compositions, and methods relating to novel interactions of the extracellular domain of LRP5, HBM (a variant of LRP5), and/or LRP6 with Dkk, including Dkk-1. The various nucleic acids, polypeptides, antibodies, assay methods, diagnostic methods, and methods of treatment of the present invention are related to and impact on Dkk, LRP5, LRP6, HBM, and Wnt signaling. Dkk, LRP5, LRP6, HBM, and Wnt are implicated in bone and lipid cellular signaling. Thus, the present invention provides reagents and methods for modulating lipid levels and/or bone mass and is useful in the treatment and diagnosis of abnormal lipid levels and bone mass disorders, such as osteoporosis.

Modulators of alpha-1 antitrypsin

Novel compounds, compositions, and methods of using and preparing the same, which may be useful for treating alpha-1 antitrypsin deficiency (AATD).

Derivados de indol como moduladores de alfa-1-antitripsina para tratar la deficiencia de alfa-1-antitripsina (aatd).

Derivados de indol como moduladores de alfa-1- antitripsina para tratar la deficiencia de alfa-1-antitripsina (AATD).

Modulators of alpha-1 antitrypsin

The disclosure provides compounds useful for treating alpha-1 antitrypsin deficiency (AATD), according to formula (I):tautomers thereof, pharmaceutically acceptable salts of the compounds, pharmaceutically acceptable salts of the tautomers, deuterated derivatives of the compounds, deuterated derivatives of the tautomers, and deuterated derivatives of the salts, solid forms of those compounds and processes for making those compounds.

Test und reagentien zur identifizierung von antiproliferierenden stoffen

Modulators of alpha-1 antitrypsin

Novel compounds, compositions, and methods of using and preparing the same, which maybe useful for treating alpha-1 antitrypsin deficiency (AATD).

1 h-pyrazolo[4,3-g]isoquinoline and 1 h-pyrazolo[4,3-g]quinoline derivatives as alpha-1 -antitrypsin modulators for treating alpha-1 -antitrypsin deficiency (aatd)

1H-pyrazolo[4,3-g]isoquinoline and 1H-pyrazolo[4,3-g]quinoline derivatives as alpha-1-antitrypsin modulators for treating alpha-1- antitrypsin deficiency (AATD).

Assays and reagents for identifying anti-fungal agents, and uses related thereto

The present invention relates to rapid, reliable and effective assays for screening and identifying pharamceutically effective compounds that specifically inhibit the biological activity of fungal GTPase proteins, particularly GTPases involved in cell wall integrity, hyphael formation, and/or other cellular functions critical to pathogenesis. Another aspect of the present invention relates to novel Candida genes and gene products.

Condensed tricyclic pyrroles as Alpha-1 Antitrypsin modulators.

The disclosure provides compounds useful for treating alpha-1 antitrypsin deficiencv (AATD), according to formula (I), tautomers thereof, pharmaceutically acceptable salts of the compounds, pharmaceutically acceptable salts of the tautomers, deuterated derivatives of the compounds, deuterated derivatives of the tautomers, and deuterated derivatives of the salts, solid forms of those compounds and processes for making those compounds.

Assays and reagents for identifying anti-fungal agents, and uses related thereto

The present invention relates to rapid, reliable and effective assays for screening and identifying pharmaceutically effective compounds that specifically inhibit the biological activity of fungal GTPase proteins, particularly GTPases involved in cell wall integrity, hyphael formation, and/or other cellular functions critical to pathogenesis. Another aspect of the present invention relates to novel Candida genes and gene products.

Pirroles triciclicos condensados como moduladores de alfa-1 antitripsina.

La descripción proporciona compuestos útiles para tratar la deficiencia de alfa-1 antitripsina (AATD), de acuerdo con la fórmula (I), tautómeros de estos, sales farmacéuticamente aceptables de los compuestos, sales farmacéuticamente aceptables de los tautómeros, derivados deuterados de los compuestos, derivados deuterados de los tautómeros y derivados deuterados de las sales, formas sólidas de esos compuestos y procesos para preparar esos compuestos. (ver Fórmula).

Moduladores de alfa-1-antitripsina.

Compuestos, composiciones y métodos novedosos para usar y preparar los mismos, que pueden ser útiles para tratar la deficiencia de alfa-1-antitripsina (AATD).

Derivados de pirrolo[2,3-f]indazol e 2,4,5,10-tetrazatriciclo[7.3.0.03,7]dodeca-1,3(7),5,8,11- penteno como moduladores da alfa-1-antitripsina para tratamento da deficiência de alfa-1-antitripsina (aatd)

DERIVADOS DE PIRROLO[2,3-F]INDAZOL E 2,4,5,10-TETRAZATRICICLO[7.3.0.03,7]DODECA-1,3(7),5,8,11- PENTENO COMO MODULADORES DA ALFA-1-ANTITRIPSINA PARA TRATAMENTO DA DEFICIÊNCIA DE ALFA-1-ANTITRIPSINA (AATD). Derivados de pirrolo[2,3-f]indazol e 2,4,5,10- tetrazatriciclo[7.3.0.03,7]dodeca-1,3(7),5,8,11-penteno como moduladores da alfa-1 antripsina para tratamento da deficiência de alfa-1 antitripsina (AATD).

Modulators of alpha-1 antitrypsin

Pyrrolo[2,3-f]indazole and 2,4,5,10-tetrazatricyclo[7.3.0.03,7]dodeca-l,3(7),5,8,ll-pentaene derivatives as alpha-l-antitrypsin modulators for treating alpha-l-antitrypsin deficiency (AATD)

Pyrrolo[2,3-f]indazole and 2,4,5,10-tetrazatricyclo[7.3.0.03,7]dodeca-1,3(7),5,8,11-pentaene derivatives as alpha-1-antitrypsin modulators for treating alpha-1-antitrypsin deficiency (aatd)

Pyrrolo[2,3-f]indazole and 2,4,5,10-tetrazatricyclo[7.3.0.03,7]dodeca-1,3(7),5,8,11-pentaene derivatives as alpha-l-antitrypsin modulators for treating alpha-l-antitrypsin deficiency (AATD)

Condensed tricyclic pyrroles as alpha-1 antitrypsin modulators

Pyrroles tricycliques condensés utilisés comme modulateurs de l'alpha-1 antitrypsine

Pirroles tricíclicos condensados como moduladores de alfa-1 antitripsina

La descripción proporciona compuestos útiles para tratar la deficiencia de alfa-1 antitripsina (AATD), de acuerdo con la fórmula (I), tautómeros de estos, sales farmacéuticamente aceptables de los compuestos, sales farmacéuticamente aceptables de los tautómeros, derivados deuterados de los compuestos, derivados deuterados de los tautómeros y derivados deuterados de las sales, formas sólidas de esos compuestos y procesos para preparar esos compuestos. (LA FORMULA SE OBSERVA EN LA MEMORIA TECNICA)

Pirroles triciclicos condensados como moduladores de alfa-1 antitripsina

La descripción proporciona compuestos útiles para tratar la deficiencia de alfa-1 antitripsina (AATD), de acuerdo con la fórmula (I), tautómeros de estos, sales farmacéuticamente aceptables de los compuestos, sales farmacéuticamente aceptables de los tautómeros, derivados deuterados de los compuestos, derivados deuterados de los tautómeros y derivados deuterados de las sales, formas sólidas de esos compuestos y procesos para preparar esos compuestos.

Pirroles tricciclicos condensados como moduladores de antitripsina alfa-1

PIRROLES TRICCICLICOS CONDENSADOS COMO MODULADORES DE ANTITRIPSINA ALFA-1

Pirrois tricíclicos condensados como moduladores da alfa-1 antitripsina

pirrois tricíclicos condensados como moduladores da alfa-1 antitripsina. a divulgação fornece compostos úteis para tratar a deficiência de alfa-1 antitripsina (aatd), de acordo com a fórmula (i), seus tautômeros, sais farmaceuticamente aceitáveis dos compostos, sais farmaceuticamente aceitáveis dos tautômeros, derivados deuterados dos compostos, derivados deuterados dos tautômeros e derivados deuterados dos sais, formas sólidas desses compostos e processos para produzir esses compostos.

Condensed tricyclic pyrroles as alpha-1 antitrypsin Modulators

The disclosure provides compounds useful for treating alpha-1 antitrypsin deficiency (AATD), according to formula (I), tautomers thereof, pharmaceutically acceptable salts of the compounds, pharmaceutically acceptable salts of the tautomers, deuterated derivatives of the compounds, deuterated derivatives of the tautomers, and deuterated derivatives of the salts, solid forms of those compounds and processes for making those compounds.

Assays and reagents for identifying anti-fungal agents, and uses related thereto

The present invention relates to rapid, reliable and effective assays for screening and identifying pharmaceutically effective compounds that specifically inhibit the biological activity of fungal GTPase proteins, particularly GTPases involved in cell wall integrity, hyphael formation, and/or other cellular functions critical to pathogenesis. Another aspect of the present invention relates to novel Candida genes and gene products.

Immunosuppressant Target Proteins

The present invention relates to the discovery of novel proteins of mammalian origin which are immediate downstream targets for FKBP/rapamycin complexes.

Proteina diana inmunosupresoras.

LA PRESENTE INVENCION SE RELACIONA CON EL DESCUBRIMIENTO DE NUEVAS PROTEINAS DE ORIGEN MAMIFERO, QUE SON DIANAS SITUADAS INMEDIATAMENTE AGUAS ABAJO EN RELACION CON LOS COMPLEJOS FKBP/RAPAMICINA.

Immunsuppressive proteinziele

Immunosuppressant target proteins

The present invention relates to the discovery of novel proteins of mammalian origin which are immediate downstream targets for FKBP/rapamycin complexes.

Immunosuppressive proteinziele

Cyclin activation kinase (cak) from human pathogens, and uses related thereto

The present invention relates to the discovery of novel cell cycle regulatory proteins from the human pathogen Candida.

Assays and reagents for identifying anti-fungal agents, and uses related thereto

The present invention relates to rapid, reliable and effective assays for screening and identifying pharmaceutically effective compounds that specifically inhibit the biological activity of fungal GTPase proteins, particularly GTPases involved in cell wall integrity, hyphael formation, and/or other cellular functions critical to pathogenesis. Another aspect of the present invention relates to novel Candida genes and gene products.

Substituted 5-hydroxyindole compounds as modulators of alpha-1 antitrypsin

Novel compounds, compositions, and methods of using and preparing the same, which may be useful for treating alpha-1 antitrypsin deficiency (AATD).

Cyclin activation kinase (cak) from human pathogens, and uses related thereto

The present invention relates to the discovery of novel cell cycle regulatory proteins from the human pathogen Candida.

Modulators of alpha-1 antitrypsin

The disclosure provides compounds useful for treating alpha-1 antitrypsin deficiency (AATD), according to formula (I): tautomers thereof, pharmaceutically acceptable salts of the compounds, pharmaceutically acceptable salts of the tautomers, deuterated derivatives of the compounds, deuterated derivatives of the tautomers, and deuterated derivatives of the salts, solid forms of those compounds and processes for making those compounds.