Vertex Pharmaceuticals, Inc.

Thomas Jefferson University

Rowan University

Yi Shi

Muna Shrestha

Ales Medek

Robert Michael Hughes

David Andrew Siesel

Paul Angell

Kevin Gagnon

Mark Thomas Miller

Jinglan Zhou

Sara Sabina Hadida Ruah

Jason McCartney

Michael Aaron Brodney

Corey Don Anderson

Thomas Cleveland

Prasuna Paraselli

Jeremy J Clemens

Kathy Stavropoulos

Jean-Damien Charrier

Steven John Durrant

John R Studley

Benjamin Joseph Littler

Stefanie Roeper

Michael Waldo

Francoise Yvonne Theodora Marie Pierard

Armando Urbina

Andreas Peter Termin

Fredrick Van Goor

John Patrick Maxwell

Qing Tang

Kevin Michael Cottrell

Dennis James Hurley

Upul Keerthi Bandarage

Peter Diederik Jan Grootenhuis

Jinwang Xu

Ali Keshavarz-Shokri

Pramod Virupax Joshi

Paul Krenitsky

Haripada Khatuya

Simon Giroux

Adam R Looker

Michael John Boyd

Johnny Uy

Ronald Lee Grey, Jr

Stephen Andrew Thomson

Lev Tyler Dewey Fanning

Fabrice Jean Denis Pierre

Lori Ann Ferris

Raymond Stanley Gross

Mac Arthur Johnson, Jr

Alexander Russell Abela

Peter D Jones

Minson Baek

Philippe Marcel Nuhant

Andrew Zalewski

Brad D Maxwell

Anton V Gulevich

Timothy Alcacio

Clara Kuang-Ju Hsia

Diane M Boucher

Ping Kang

Cavan McKeon Bligh

Setu Roday

Rupa Sawant

Tiansheng Wang

Steven M Ronkin

Jingrong Cao

Brian Ledford

Peter Grootenhuis

Jon H Come

Beili Zhang

Bryan A Frieman

Timothy Richard Coon

Fabrice Pierre

Michael P Clark

Suganthini S Nanthakumar

Peter Jamison Rose

Warren A Dorsch

Elaine B Krueger

Sara E Swift

Leslie A Dakin

Timothy J Senter

Carl Zwicker

Kirk Overhoff

Anne Fortier

Olivier Nicolas

Camil Sayegh

Mariam Zaky

David J Lauffer

Pan Li

Mark Willem Ledeboer

Valdas Jurkauskas

Praveen Mudunuri

Randal Byrn

Kwame Wiredu Nti-Addae

Sarah Robertson

Steven Jones

Anuj K Kuldipkumar

Shereen Ibrahim

Mohammed Asmal

Simon Adam O'Neil

Compounds of Formula (I): pharmaceutically acceptable salts thereof, deuterated derivatives of any of the foregoing, and metabolites of any of the foregoing are disclosed. Pharmaceutical compositions comprising the same, methods of treating cystic fibrosis using the same, and methods for making the same are also disclosed. Also disclosed are solid state forms of Compound 1 and salts and solvates thereof.

Modulator of the Cystic Fibrosis Transmembrane Conductance Regulator, pharmaceutical compositions, methods of treatment, and process for making the modulator

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

Crystalline forms of Compound I: pharmaceutically acceptable salts thereof, and solvates and hydrates thereof are disclosed. Pharmaceutical compositions comprising the same, methods of treating cystic fibrosis using the same, and methods for making the same are also disclosed.

Crystalline forms of CFTR modulators

The disclosure provides at least one entity chosen from compounds of formula (I), solid state forms of the same, compositions comprising the same, and methods of using the same, including use in treating focal segmental glomerulosclerosis (FSGS) and/or non-diabetic kidney disease (NDKD).

Inhibitors of APOL1 and methods of using same

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

The disclosure provides a compound of Formula (I), including deuterated derivatives, pharmaceutically acceptable salts, and solvates thereof, solid state forms of those compounds, compositions comprising those compounds and solid forms, and methods of using the same, including use in treating focal segmental glomerulosclerosis (FSGS) and/or non-diabetic kidney disease (NDKD). 

Modulator of the cystic fibrosis transmembrane conductance regulator, pharmaceutical compositions, methods of treatment, and process for making the modulator

The present invention relates to processes and intermediates for preparing compounds useful as inhibitors of ATR kinase, such as aminopyrazine-isoxazole derivatives and related molecules. The present invention also relates to compounds useful as inhibitors of ATR protein kinase. The invention relates to pharmaceutically acceptable compositions comprising the compounds of this invention; methods of treating of various diseases, disorders, and conditions using the compounds of this invention; processes for preparing the compounds of this invention; intermediates for the preparation of the compounds of this invention; and solid forms of the compounds of this invention. 

Processes for preparing ATR inhibitors

The present invention relates to processes and intermediates for preparing compounds useful as inhibitors of ATR kinase, such as aminopyrazine-isoxazole derivatives and related molecules. The present invention also relates to compounds useful as inhibitors of ATR protein kinase. The invention relates to pharmaceutically acceptable compositions comprising the compounds of this invention; methods of treating of various diseases, disorders, and conditions using the compounds of this invention; processes for preparing the compounds of this invention; intermediates for the preparation of the compounds of this invention; and solid forms of the compounds of this invention. The compounds of this invention have formula I or II: wherein the variables are as defined herein.

Processes for making compounds useful as inhibitors of ATR kinase

Polymorphic forms of Compound (1) or a pharmaceutically acceptable salt thereof, wherein Compound (1) is represented by the following structural formula: are Form A of HCl salt of Compound (1).&#xbd;HO, Form F of HCl salt of Compound (1).3HO, Form D of HCl salt of Compound (1), Form A of Compound (1), and Form A of tosylate salt of Compound (1). Such polymorphic forms are employed for treating influenza, inhibiting the replication of influenza viruses, or reducing the amount of influenza viruses in a biological sample or in a subject.

Inhibitors of influenza viruses replication

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Natick, MA, United States of America

Yi Shi