Arcturus Therapeutics, Inc.

Metabolic Solutions Development Company, LLC

Other

Pfizer Corporation

Promentis Pharmaceuticals, Inc.

Pharmacia & Upjohn Company

The Upjohn Co.

Pharmacia Corporation

Agouron Pharmaceuticals, Inc.

Arcturus, Inc.

Capstan Therapeutics, Inc.

Cirius Therapeutics, Inc.

Octeta Therapeutics, LLC

Steven Paul Tanis

Padmanabh Chivukula

Joseph E Payne

Priya Prakash Karmali

Scott D Larsen

Paul D May

James Nieman

Michael Neary

Daniel Lawton

Timothy Parker

James Robert Zeller

Gerald D Artman, Iii

Robert C Gadwood

Garry Robert Smith

Valerie Ann Vaillancourt

John Alan Tucker

Bradley Teegarden

Mark E Schnute

Robert Alan Chrusciel

Thomas Jay Lobl

Heinrich Josef Schostarez

Martin Durham Meglasson

Edward V Thomas

Jed F Fisher

Gerald D Artman

James R Zeller

Junhu Zhang

Gilbert M Rishton

Michael Bruno Plewe

Karen Rene Romines

Ted William Johnson

Gerard R Colca

Klaus Ruprecht Dress

Alexander Polinsky

Qiyue Hu

Masafumi Yamagishi

John C Sih

Tiziano Bandiera

Joseph Walter Strohbach

Malcolm Wilson Moon

Chiu-Hong Lin

Gilbert A Youngdale

Kiyoshi Tachikawa

James Robert Blinn

David John Anderson

Sajiv Krishnan Nair

Steven Ronald Turner

Yanjie Bao

Hai Wang

Jacqueline Lansen

Chunfeng Yin

William R Perrault

Donald Bainbridge Carter

Anle Yang

Allen Edwin Buhl

Michele M Cudahy

James A Nieman

Kumar Rajappan

Rolf F Kletzien

Bruce Evans

Paul Matthew Herrinton

Euibong J Kim

Thomas J Raub

Gregory J Fici

Geri A Sawada

Luisa Savini

Cesare Pellerano

Huichun Zhu

Priya Karmali

Padmanabh Chivikula

Gerald D Iii Artman

Disclosed are compositions of lipid nanoparticles (LNP) comprising an ionizable cationic lipid, a phospholipid, a sterol, and a PEG-lipid (non-functionalized and optionally functionalized). The functionalized PEG-lipid can be conjugated with a binding moiety to create a targeted LNP (tLNP). The disclosed tLNP preferentially deliver a nucleic acid molecule or other negatively charged payload to cells expressing a cell surface antigen recognized by the binding moiety of the tLNP, and are better tolerated, as compared to LNPs and tLNPs comprising ionizable cationic lipids found in marketed pharmaceuticals comprising LNPs.

Lipid nanoparticle formulations and compositions

ASGP-R binding molecular conjugates are provided. The conjugates are useful to deliver therapeutically effective amounts of biologically active molecules to target cells and tissues of a subject. Compositions are also provided comprising the molecular conjugates.

Asialoglycoprotein Receptor mediated delivery of therapeutically active conjugates

What is described is a compound of formula I consisting of a compound in which Ris a branched chain alkyl consisting of 10 to 31 carbons; Ris a linear alkyl, alkenyl, or alkynyl consisting of 2 to 20 carbons; Land Lare the same or different, each a linear alkylene or alkenylene consisting of 2 to 20 carbons; Xis S or O; Ris a linear or branched alkylene consisting of 1 to 6 carbons; and Rand Rare the same or different, each a hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons; or a pharmaceutically acceptable salt thereof.

Ionizable cationic lipid for RNA delivery

What is described is a trinucleotide cap analog comprising mG(5')p-NpNfor increased efficiency of in vitro transcription of m7G(5&#x2032;)p-RNA, wherein mG is N-methylguanosine or analog, (5&#x2032;)pis a 5&#x2032;,5&#x2032;-triphosphate bridge, and Nor Nor both ribonucleotide analogs linked to each other by a phosphate, p, and wherein the trinucleotide cap analog increases the efficiency of in vitro transcription.

Trinucleotide mRNA cap analogs

Disclosed herein is a compound of Formula I, wherein Ris a branched chain alkyl consisting of 10 to 31 carbons; Ris a linear alkyl, alkenyl, or alkynyl consisting of 2 to 20 carbons, or a branched chain alkyl consisting of 10 to 31 carbons; Land Lare the same or different, each a linear alkane of 1 to 20 carbons or a linear alkene of 2 to 20 carbons; Xis S or O; Ris a linear or branched alkylene consisting of 1 to 6 carbons; and Rand Rare the same or different, each a hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons; or a pharmaceutically acceptable salt or solvate thereof. 

Novel substituted N-acetyl-L-cysteine (NAC) derivatives and related compounds and methods of using these compounds for the treatment of diseases and/or conditions, including but not limited to diseases and/or conditions of, or involving, the Central Nervous System (CNS), including schizophrenia adrenoleukodystrophy, mitochondrial diseases (e.g. Leigh syndrome, Alpers' disease, and MELAS), Huntington's disease, trichotillomania, HIV-associated neurocognitive disorder, hypoxic-ischemic encephalopathy, drug craving, and drug addiction.

Substituted N-acetyl-L-cysteine derivatives and related compounds

What is described is a compound of formula I consisting of a compound in which

Trinucleotide MRNA cap analogs

What is described is a compound of formula I consisting of a compound in which Ris a branched chain alkyl consisting of 10 to 31 carbons; Ris a linear alkyl, alkenyl, or alkynyl consisting of 2 to 20 carbons; Land Lare the same or different, each a linear alkylene of 1 to 20 carbons or a linear alkenylene of 2 to 20 carbons; Xis S or O; Ris a linear or branched alkylene consisting of 1 to 6 carbons; and Rand Rare the same or different, each a hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons; or a pharmaceutically acceptable salt thereof.

What is described is a compound of formula I 

Aromatic ionizable cationic lipid

Described herein is a compound having the structure of formula I, II, or III, wherein R comprises a double stranded RNA molecule, and L, L, and Lindependently for each occurrence comprise a ligand selected from the group consisting of a carbohydrate, a cholesteryl, or a peptide; a pharmaceutically accepted salt or pharmaceutical composition thereof; and a method of making the compound.

MultiLigand agent for drug delivery

What is described is a compound of formula I wherein

Growing community of inventors

Carlsbad, CA, United States of America

Steven Paul Tanis