Genzyme Corporation

Genetics Institute, Inc.

Taigen Biotechnology

Taigen Biotechnology Company, Ltd.

Massachusetts Institute of Technology

Harvard College

Yibin Xiang

Cassandra Celatka

Craig S Siegel

Cecilia M Bastos

David J Harris

Edward R Lee

Richard A Silva

Frederic Vinick

Lisa M Cuff

Junkai Liao

Mikaela Levine

Angeles Dios

John Kane

Thomas Henry Jozefiak

Jasbir S Seehra

Jean E Bemis

John C McKew

Bradford Hirth

Frank Eldridge Lovering

Zhong Zhao

Neelu Kaila

Ching-Cheng Wang

Seng H Cheng

Andrew Charles Good

John Marshall

Wen-Chang Chen

Ronald K Scheule

Lihren Chen

Markus Metz

Kak-Shan Shia

Chu-Chung Lin

Chi-Feng Yen

Shu-Jen Chen

Chih-Wei Chang

Elyse Bourque

Thomas A Natoli

Renato Tony Skerlj

Chun-Wei Kuo

Elina Makino

Gholam Hossein Hakimelahi

Oxana Beskrovnaya

Pei-Chin Cheng

Jiing-Jyh Jan

Jen-Fuh Liu

Shao-Ying Liao

Jen-Dar Wu

Mario A Cabrera-Salazar

Katherine Jancsics

Herve Husson

Feng-Yih Su

Syaulan Yang

Jia-Liang Zhu

John L Knopf

Ralph Mazitschek

Renato Skerlj

John P Leonard

Ming-Chu Hsu

Chien-Huang Wu

Jon Clardy

Ying-Huey Huang

Dyann F Wirth

Ming-Chen Chou

Christopher Yee

Andrew T Papoulis

Roger Wiegand

Alexandra Joseph

Paul Mason

Chen-Tso Tseng

Hua-Chien Chen

Joseph Cortese

Mary Lynn Baniecki

Jill Gregory

Sameer Urgaonkar

John Leonard

Kara Carter

Yiding Hu

Thomas H Jozafiak

Mohamud Mohamud

Sanjay Danthi

Yinyin Huang

Elyse M J Bourque

Jon C Clardy

Cassandra A Celatka

Katherine Jancisics

Ronald Scheule

Mario Cabrera-Salazar

Andrew Papoulis

Elyse MJ Bourque

The invention relates to inhibitors of glucosylceramide synthase (GCS) of the Formula I: represented by the following structural formula or a pharmaceutically acceptable salt thereof, wherein the variables are as defined herein, useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of cancer.

Glucosylceramide synthase inhibitors

The invention relates to inhibitors of glucosylceramide synthase (GCS), such as Compound of Formula I, shown below, as defined herein, useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of cancer. 

The invention features compounds of Formula (I), Formula (I)-A and Formula (I)-B as disclosed herein, as well as methods of synthesis, therapy, diagnostics, and assays. 

NAMPT inhibitors and methods

A compound for use in treating polycystic kidney disease is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating polycystic kidney disease in a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. Methods of treating in polycystic kidney disease in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof.

2-acylaminopropoanol-type glucosylceramide synthase inhibitors

A compound is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. The subject has type 2 diabetes; renal hypertrophy or hyperplasia associated with diabetic nephropathy; Tay-Sachs; Gaucher's; or Fabry's disease. Methods of decreasing plasma TNF-&#x3b1;, lowering blood glucose levels, decreasing glycated hemoglobin levels, inhibiting glucosylceramide synthase, and lowering glycosphingolipid concentrations in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof.

The present invention relates to methods of treating a subject with malaria comprising administering a 2-aminoindole compound represented by Formula: (I)&#x2014;The values and preferred values of the variables in Structural Formula I are defined herein. 

2-aminoindole compounds and methods for the treatment of malaria

Novel mechanism-based inhibitors of S-adenosyl-L-methionine decarboxylase are provided. These compounds of formula (1) inhibit the life cycle of trypanosomes, and are useful to treat subjects infected with African trypanosomes. The invention includes pharmaceutical compositions and methods of using the compounds of formula (1).

Inhibitors of S-adenosyl-L-methionine decarboxylase

A compound for use in treating polycystic kidney disease is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating polycystic kidney disease in a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. Methods of treating in polycystic kidney disease in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. 

A compound is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. The subject has type 2 diabetes; renal hypertrophy or hyperplasia associated with diabetic nephropathy; Tay-Sachs; Gaucher's; or Fabry's disease. Methods of decreasing plasma TNF-&#x3b1;, lowering blood glucose levels, decreasing glycated hemoglobin levels, inhibiting glucosylceramide synthase, and lowering glycosphingolipid concentrations in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. 

This invention relates to methods of preparing the compounds of formula (V): Each variable in this formula is defined in the specification.

Synthesis of polyamine compounds

This invention relates to treating an infection with a virus using protease inhibitors. Examples of the protease inhibitors include compounds of formula (II). Each variable is defined in the specification. 

Peptide-like compounds that inhibit coronaviral 3CL and flaviviridae viral proteases

This invention relates to a method for treating inflammatory diseases or immune diseases, developmental or degenerative diseases, or tissue injuries. The method includes administering to a subject in need thereof an effective amount of one or more compounds of the following formula. Each variable in this formula is defined in the specification. 

Polyamine compounds for treating chemokine receptor mediated diseases

This invention relates to treating inflammatory and immune diseases with certain aminoquinoline compounds that bind to CXCR3 receptors. The aminoquinoline compounds are covered by the formula (I) shown below. Each variable is defined in the specification. 

Aminoquinoline compounds

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Acton, MA, United States of America

Yibin Xiang