Modernatx, Inc.

Merck + Co., Inc.

Merck Sharp + Dohme Corp.

Sirna Therapeutics, Inc.

Other

Isis Pharmaceuticals, Inc.

Gabor Butora

Lihu Yang

Matthew G Stanton

Edward John Miracco

Alexander Pasternak

Jennifer Nelson

Changyou Zhou

Stephen D Goble

Sander G Mills

Stephen G Hoge

William Joseph Issa

Andrew W Fraley

Amy E Rabideau

Christopher Richard Moyes

Gregori J Morriello

Ian W Davies

Shankaran Kothandaraman

Deodialsingh Guiadeen

Richard Jiao

Amy Rhoden Smith

Thomas Steele

Malcolm MacCoss

Ning Qi

Denise M Kenski

William Michael Flanagan

Balkrishen Bhat

William H Parsons

Frank W Bennett

Ann E Weber

Stephane L Bogen

David Brian Olsen

Malcolm Maccoss

Meredith Packer

Cheng Tang

Wenlang Fu

Dipendra Gyawali

Serenus Hua

Gregory John Mercer

Lingyan Wang

Vinay M Girijavallabhan

Atanu Roy

Antonin De Fougerolles

Anne Bettina Eldrup

Quang T Truong

Angela Dawn Kerekes

Yuhua Huang

Kothandaraman Shankaran

Quang Truong

Brian R Fritz

Kenneth Alan Koeplinger

Daniel R McMasters

Christopher R Conlee

Amy Rhoden-smith

William Issa

Anne E Weber

Anne B Eldrup

The invention relates to improved RNA compositions for use in therapeutic applications. The RNA compositions are particularly suited for use in human therapeutic application (e.g., in RNA therapeutics). The RNA compositions are made by improved processes, in particular, improved in vitro-transcription (IVT) processes. The invention also relates to methods for producing and purifying RNA (e.g, therapeutic RNAs), as well as methods for using the RNA compositions and therapeutic applications thereof.

High purity RNA compositions and methods for preparation thereof

The invention relates to improved RNA compositions for use in therapeutic applications. The RNA compositions are particularly suited for use in human therapeutic application (e.g., in RNA therapeutics). The RNA compositions are made by inproved processes, in particular, improved in vitro-transcription (IVT) processes. The invention also relates to methods for producing and purifying RNA (e.g, therapeutic RNAs), as well as methods for using the RNA compositions and therapeutic applications thereof.

The invention features polynucleotides encoding a polypeptide including a 3'-stabilizing region and having increased stability compared to wild-type polynucleotides.

Polynucleotides containing a stabilizing tail region

The disclosure provides polynucleotides encoding a polypeptide including a morpholino linker. In some embodiments, the polynucleotides of the invention have increased stability compared to wild-type polynucleotides.

Polynucleotides containing a morpholino linker

The present disclosure relates to cap analogs, which can result in high levels of capping efficiency and transcription and improved translation efficiencies. The present disclosure also relates to methods useful for preparing cap analogs and using mRNA species containing such analogs, as well as kits containing the novel cap analogs.

Trinucleotide mRNA cap analogs

Provided herein are compositions and methods of reducing adduct formation.

Lipid nanoparticle compositions and methods of formulating the same

The present disclosure relates to cap analogs, which can result in high levels of capping efficiency and transcription and improved translation efficiencies. The present disclosure also relates to methods useful for preparing cap analogs and using mRNA species containing such analogs, as well as kits containing the cap analogs.

Phosphate replacement MRNA cap analogs

Phosphate replacement mRNA cap analogs

The present disclosure provides alternative nucleosides, nucleotides, and nucleic acids, and methods of using them.

Alternative nucleic acid molecules and uses thereof

The present invention relates to disulfide masked prodrug compounds of formula I, compositions and methods that are amenable to bioactivation by a reducing agent such as glutathione:W&#x2014;P(V)LG&#x2003;&#x2003;(Formula I),wherein P(V)LG is a pentavalent phosphorus leaving group; and W is: wherein R, R, R, R, R, R, Rand Rare as defined in the specification. Such disulfide based compounds, compositions, and methods can be useful, for example, in providing novel prodrugs for use as therapeutics. Once administered, the prodrug is metabolized in vivo into an active metabolite in a process termed bioactivation.

Disulfide masked prodrug compositions and methods

Growing community of inventors

Martinsville, NJ, United States of America

Gabor Butora