Global Blood Therapeutics, Inc.

Sgx Pharmaceuticals, Inc.

Takeda Pharmaceutical Company Limited

Syntex (u.s.a.) Inc.

Cytokinetics, Inc.

Zhe Li

Qing Xu

Brian Walter Metcalf

Calvin Wesley Yee

Ming Yu

Stephen L Gwaltney, Ii

Jason R Harris

Mark E Wilson

Ruo W Steensma

William D Arnold

Andreas Gosberg

Ian A McDonald

Lina Quattrocchio Setti

Pierre Bounaud

Hing L Sham

Manuel Zancanella

Stephan D Parent

Travis Lee Houston

Chul Yu

Stephen L Gwaltney

Brian Walter Eastman

Paul A Sprengeler

Shahul Nilar

George Yiannikouros

Julieana Cocuz

Jeffrey Alan Stafford

Stephen W Kaldor

Michael Patrick Dillon

Chihyuan Chuang

Uma Sinha

Chixu Chen

Stephanie Hopkins

Stefan N Gradl

Peter Michael Rademacher

Carsten Alt

Nathan Guz

Walter Keung

Yiyang Shao

Markus Frieser

Liang Liao

Zhiyuan Zhang

Benjamin Jones

Betty Lam

Andre A Kiryanov

John S Tyhonas

Lily Kwok

Craig A Behnke

David Llewellyn Coffen

Anthony P D W Ford

Timothy James Williams

Kobina N Dufu

Matthew A Lardy

James Partridge

Xin Geng

Steven Richards

Stephen L Ii Gwaltney

Ii Stephen L Gwaltney

Stephen L Gwaltney Ii

The subject matter described herein is directed to ferroportin inhibitor compounds of Formula I or I' and pharmaceutical salts thereof, methods of preparing the compounds, pharmaceutical compositions comprising the compounds, and methods of administering the compounds for prophylaxis and/or treatment of diseases caused by a lack of hepcidin or iron metabolism disorders, particularly iron overload states, such as thalassemia, sickle cell disease and hemochromatosis, and also kidney injuries.

Cycloalkyl pyrimidines as ferroportin inhibitors

The present disclosure provides compounds of Formula (I) that are histone methyltransferases G9a and/or GLP inhibitors and are therefore useful for the treatment of diseases treatable by inhibition of G9a and/or GLP such as cancers and hemoglobinopathies (e.g., beta-thalassemia and sickle cell disease). Also provided are pharmaceutical compositions containing such compounds and processes for preparing such compounds. where R, alk, R, Z, Z, X, R, R, B, a, and b are as described herein.

Histone methyltransferase inhibitors

The present disclosure provides certain tricyclic compounds that are histone methyltransferases G9a and/or GLP inhibitors and are therefore useful for the treatment of diseases treatable by inhibition of G9a and/or GLP such as cancers and hemoglobinopathies (e.g., beta-thalassemia and sickle cell disease). Also provided are pharmaceutical compositions containing such compounds and processes for preparing such compounds.

Tricyclic compounds as histone methyltransferase inhibitors

The present disclosure provides tricyclic compounds of formula (I) that are histone methyltransferases G9a and/or GLP inhibitors and are therefore useful for the treatment of diseases treatable by inhibition of G9a and/or GLP such as cancers and hemoglobinopathies (e.g., beta-thalassemia and sickle cell disease). Also provided are pharmaceutical compositions containing such compounds and processes for preparing such compounds. 

The subject matter described herein is directed to pyruvate kinase activating compounds of Formula I: and pharmaceutical salts thereof, methods of preparing the compounds, pharmaceutical compositions comprising the compounds and methods of administering the compounds for the treatment of diseases associated with PKR and/or PKM2, such as pyruvate kinase deficiency, sickle cell disease, and beta-thalassemia.

Substituted 2,6-dihydropyrrolo[3,4-c]pyrazoles as pyruvate kinase activators

The present disclosure relates generally to compounds and pharmaceutical compositions suitable as modulators of hemoglobin, and methods for their use in treating disorders mediated by hemoglobin.

Modulators of hemoglobin

Provide herein are compounds and pharmaceutical compositions suitable as modulators of hemoglobin, methods and intermediates for their preparation, and methods for their use in treating disorders mediated by hemoglobin and disorders that would benefit from tissue and/or cellular oxygenation.

Compounds and uses thereof for the modulation of hemoglobin

Disclosed are crystalline free base ansolvate forms of 2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde (or Compound 1), such as the free base Form I, Form II and Material N. Also disclosed are crystalline free base solvates of 2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde (or Compound 1).

Crystalline polymorphs of the free base of 2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde

The subject matter described herein is directed to Ferroportin inhibitor compounds of Formula I and pharmaceutical salts thereof, methods of preparing the compounds, pharmaceutical compositions comprising the compounds and methods of administering the compounds for prophylaxis and/or treatment of diseases caused by a lack of hepcidin or iron metabolism disorders, particularly iron overload states, such as thalassemia, sickle cell disease and hemochromatosis. The compounds of Formula I and pharmaceutical salts thereof have the following structure: wherein R, R, R, X, X, X, X, Y, Y, and Yare as described herein.

Ferroportin inhibitors and methods of use

The present disclosure provides certain compounds that are histone methyltransferases G9a and/or GLP inhibitors and are therefore useful for the treatment of diseases treatable by inhibition of G9a and/or GLP such as cancers and hemoglobinpathies such as beta-thalassemia and sickle cell disease. Also provided are pharmaceutical compositions containing such compounds as well as processes and intermediates for preparing such compounds.

The present disclosure provides certain angular tricyclic compounds that are histone methyltransferases G9a and/or GLP inhibitors and are therefore useful for the treatment of diseases treatable by inhibition of G9a and/or GLP such as cancers and hemoglobinopathies (e.g., beta-thalassemia and sickle cell disease). Also provided are pharmaceutical compositions containing such compounds and processes for preparing such compounds.

Azaindole compounds as histone methyltransferase inhibitors

Growing community of inventors

San Diego, CA, United States of America

Zhe Li