Genzyme Corporation

Imago Biosciences, Inc.

Massachusetts Institute of Technology

Harvard College

Daimed

Cassandra Celatka

Yibin Xiang

Cecilia M Bastos

Amy E Tapper

Michael John Clare

Craig S Siegel

David J Harris

Edward R Lee

Richard A Silva

Hugh Young Rienhoff, Jr

Frederic Vinick

Lisa M Cuff

Junkai Liao

John Kane

Mikaela Levine

Angeles Dios

John Michael McCall

Thomas Henry Jozefiak

Bradford Hirth

Renato Tony Skerlj

Seng H Cheng

Andrew Charles Good

John Marshall

Ronald K Scheule

Markus Metz

Zhong Zhao

Jon Clardy

Thomas A Natoli

Elyse Bourque

Dyann F Wirth

Elina Makino

Roger Wiegand

Ralph Mazitschek

Jian Lin

Arthur Glenn Romero

He Zhao

Renato Skerlj

Lei Jin

Kerry Leigh Spear

Amy Ripka

Thomas D Bannister

Manuel A Navia

Namrata Vora

Tsvetelina Ivanova Lazarova

John P Leonard

Jon C Clardy

Oxana Beskrovnaya

Mario A Cabrera-Salazar

Joseph Cortese

Katherine Jancsics

Herve Husson

Hongfeng Deng

Elisabeth C A Brot

Roger Xie

Michael Rynkiewicz

Zihong Guo

Nizal Chandrakumar

Jian-Xie Chen

Xuemei Chen

Mary Lynn Baniecki

Zhihua Li

Xiaoming Liao

Scott T Moe

John Leonard

Parag Ved

Betina Biolatto

Sameer Urgaonkar

Elyse M J Bourque

Toni Chancellor

Michael L Booker

Vishal P Patel

Thomas H Jozafiak

Pamela Nagafuji

James E Stickler

Patricia Soulliac

John M Mccall

Katherine Jancisics

Ronald Scheule

Mario Cabrera-Salazar

Andrew Good

Thomas Natoli

Jr Hugh Y Reinhoff

Yabin Xiang

Elyse MJ Bourque

Jr Rienhoff Hugh Young

Michael Clare

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 present disclosure relates to compounds and methods which may be useful as inhibitors of KDM1A for the treatment or prevention of diseases. Methods of inhibition of KDM1A, methods of increasing gamma globin gene expression, and methods to induce differentiation in cancer cells in a human or animal subject are also provided for treatment of disease such as acute myelogenous leukemia.

KDM1A inhibitors for the treatment of disease

Disclosed herein are new compounds and compositions and their application as pharmaceuticals for the treatment of diseases. Methods of inhibition of KDM1A, methods of increasing gamma globin gene expression, and methods to induce differentiation of cancer cells in a human or animal subject are also provided for the treatment of diseases such as acute myelogenous leukemia.

Provided in this disclosure are methods for the synthesis of substituted 2-arylcyclopropylamines and 2-heteroarylcyclopropylamines and related compounds. Also provided are methods for reduction of thioesters to aldehydes, and methods for reductive animation of cyclopropylamines.

Methods and processes for the preparation of KDM1A 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. 

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

Inhibitors of dihydroorotate dehydrogenase (DHODH) for theenzyme have been identified and characterized. The inhibitors have high specificity, submicromolar efficacy against cultured parasite strains, exhibit drug-like properties, and are not overtly cytotoxic.

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Hull, MA, United States of America

Cassandra Celatka