Vertex Pharmaceuticals, Inc.

Alios Biopharma, Inc.

Ales Medek

Michael Waldo

Yi Shi

Kevin Gagnon

Praveen Mudunuri

Lori Ann Ferris

Muna Shrestha

Michael Aaron Brodney

Carl Zwicker

Beili Zhang

Mariusz Krawiec

Yuegang Zhang

Jean-Damien Charrier

Joanne Louise Pinder

John R Studley

Damien Fraysse

John Patrick Maxwell

Dennis James Hurley

Qing Tang

Kevin Michael Cottrell

Upul Keerthi Bandarage

Jinwang Xu

Francoise Pierard

Simon Giroux

Adam R Looker

Michael John Boyd

David Matthew Shaw

Stephen Andrew Thomson

Stefanie Roeper

Gorka Etxebarria I Jardi

Ronald Lee Grey, Jr

Christopher John Davis

Lev Tyler Dewey Fanning

Maninder Panesar

Young Chun Jung

Eleni Dokou

William Aloysius Nugent

Mac Arthur Johnson, Jr

Peter D Jones

Dragutin Knezic

Anuj K Kuldipkumar

Philippe Marcel Nuhant

Diane M Boucher

Katie McCarty

Brad D Maxwell

Cavan McKeon Bligh

Setu Roday

Amy Beth Hall

Rebecca Jane Swett

Timothy Lewis Tapley

Sarathy Kesavan

Mark Thomas Miller

Jinglan Zhou

Sara Sabina Hadida Ruah

Jason McCartney

Tiansheng Wang

Corey Don Anderson

Steven M Ronkin

Jingrong Cao

Brian Ledford

Peter Grootenhuis

Jon H Come

Thomas Cleveland

Bryan A Frieman

Timothy Richard Coon

Prasuna Paraselli

Jeremy J Clemens

Fabrice Pierre

Michael P Clark

Suganthini S Nanthakumar

Kathy Stavropoulos

Peter Jamison Rose

Warren A Dorsch

Elaine B Krueger

Kwame Wiredu Nti-Addae

Brian Luisi

Sara E Swift

Timothy J Senter

Leslie A Dakin

Kirk Overhoff

Steven C Johnston

Mark Jeffrey Sullivan

Sneha Ghanshyam Arekar

Anne Fortier

Olivier Nicolas

Camil Sayegh

Mariam Zaky

Mark Willem Ledeboer

Valdas Jurkauskas

Patrick Raymond Connelly

Randal Byrn

Majed Fawaz

Steven Jones

Sarah Robertson

John P Caesar, Jr

Mohammed Asmal

Simon Adam O'Neil

John Gregg Van Alsten

Mark Strohmeier

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

The present invention features compositions comprising a plurality of therapeutic agents wherein the presence of one therapeutic agent enhances the properties of at least one other therapeutic agent. In one embodiment, the therapeutic agents are cystic fibrosis transmembrane conductance regulators (CFTR) such as a CFTR corrector or CFTR potentiator for the treatment of CFTR mediated diseases such as cystic fibrosis. Methods and kits thereof are also disclosed.

Pharmaceutical compositions for the treatment of cystic fibrosis transmembrane conductance regulator mediated diseases

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). 

The present invention 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-A or I-B: wherein the variables are as defined herein.

Compounds useful as inhibitors of ATR kinase

The present application relates to solid state forms, for example, crystalline forms of 2'-C-methyluridine-5&#x2032;-(O-phenyl-N-(S)-1-(isopropoxycarbonyl)ethyl)thiophosphoramidate, pharmaceutical compositions that can include one or more solid forms of 2&#x2032;-C-methyluridine-5&#x2032;-(O-phenyl-N-(S)-1-(isopropoxycarbonyl)ethyl)thiophosphoramidate, and methods of treating or ameliorating diseases and/or conditions with one or more solid forms of 2&#x2032;-C-methyluridine-5&#x2032;-(O-phenyl-N-(S)-1-(isopropoxycarbonyl)ethyl)thiophosphoramidate. Also disclosed herein are methods of treating diseases and/or conditions with one or more solid forms of 2&#x2032;-C-methyluridine-5&#x2032;-(O-phenyl-N-(S)-1-(isopropoxycarbonyl)ethyl)thiophosphoramidate in combination with one or more other agents.

Solid forms of a thiophosphoramidate nucleotide prodrug

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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Winchester, MA, United States of America

Ales Medek