Alnylam Pharmaceuticals, Inc.

Arbutus Biopharma Corporation

Tekmira Pharmaceuticals Corporation

Renagade Therapeutics Management Inc.

Takeda Pharmaceutical Company Limited

E-therapeutics Plc

The University of British Columbia

Muthusamy Jayaraman

Kallanthottathil G Rajeev

Muthiah Manoharan

Martin A Maier

Shigeo Matsuda

Akin Akinc

Jayaprakash K Nair

David Butler

Pachamuthu Kandasamy

Stuart Milstein

Michael E Jung

Vasant R Jadhav

Klaus Charisse

Kevin Fitzgerald

Pieter Rutter Cullis

Michael J Hope

Laxman Eltepu

Jianxin Chen

Steven Ansell

Jayaprakash K Narayanannair

Thomas A Baillie

Alexander V Kel'in

Stephen Scully

Vladimir Presnyak

Mark K Schlegel

Alfica Sehgal

Donald Foster

Narayanannair K Jayaprakash

Alim Ladha

Rubina G Parmar

Adam Castoreno

Brian Goodman

Alexander V Kelin

William Querbes

Steven Michial Ansell

Rajesh Kumar Kainthan

Thomas D Madden

Anna Borodovsky

Marco A Ciufolini

Joseph R Dorkin

Guo He

Rajendra K Pandey

Kallanthottahil G Rajeev

Maja Janas

Christopher S Theile

Christopher Brown

Joseph Robert Dorkin

Xiaojun Qin

William Cantley

Kirk Brown

Devin Scott Quinlan

Inna Shcherbakova

Mario Rodriguez Mestre

Giedrius Gasiunas

Satoru Matsumoto

Ahmad Ali Mortazavi

Viviana Mannella

Elisabeth Narayanan

Ganapathy Subramanian Sankaran

Julie Borgel

Ines De Santiago

Duncan Brown

James Longden

Amy Mccarthy

Adrian Mogg

Alan Victor Whitmore

Mamta Kapoor

Laxmab Eltepu

Graham Craggs

Takeshi Yamada

Chang Geng Peng

Muthiah Mancharan

Alexandre Debacker

Soma De

Frances Wong

Nair K Jayaprakash

Ismail Mahmoud Hafez

The present invention provides lipids that are advantageously used in lipid particles for the in vivo delivery of therapeutic agents to cells. In particular, the invention provides lipids having the following structure wherein:

Lipids and compositions for the delivery of therapeutics

The present invention relates to inhibitors, and compositions containing inhibitors, and uses of the same in the treatment or prevention of diabetes.

Inhibitors of expression and/or function

The invention relates to a method of gene silencing, comprising administering to a cell or a subject in need thereof a therapeutically effective amount of the lipophilic moieties-conjugated double-stranded iRNAs at one or more internal positions on at least one strand, optionally via a linker or carrier.

Extrahepatic delivery

The present invention relates to a cationic lipid having one or more biodegradable groups located in a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.

Biodegradable lipids for the delivery of active agents

The present disclosure provides methods and compositions comprising Cas TypeV programmable nucleases and lipid nanoparticles capable of delivering the Cas TypeV programmable nucleases and genome editing systems comprising same.

Gene editing components, systems, and methods of use

The present disclosure details various lipids, compositions, and/or methods of optimized systems and delivery vehicles for the delivery of nucleic acid sequences, polypeptides or peptides for use in vaccinating against infectious agents.

Constrained lipids and methods of use thereof

The present invention relates to ligand conjugates of oligonucleotides (e.g., iRNA agents) and methods for their preparation. The ligands are derived primarily from monosaccharides These conjugates are useful for the in vivo delivery of oligonucleotides.

Oligonucleotide-ligand conjugates and process for their preparation

Disclosed are engineered retrons and methods of use such as to modify the genome of a host (e.g., mammalian) cell by delivering the engineered retron or the encoded ncRNA in vitro or in vivo to the host (e.g., mammalian) cell.

Engineered retrons and methods of use

Disclosed herein are lipid compositions comprising a cationic lipid of formula (I), a neutral lipid, a sterol and a PEG or PEG-modified lipid, wherein formula (I) is (F). Also disclosed are methods of producing the cationic lipid of formula (I).

Lipid compositions

One aspect of the present invention relates to a double stranded iRNA agent comprising an antisense strand which is complementary to a target gene; a sense strand which is complementary to said antisense strand; and one or more lipophilic moieties conjugated to one or more internal positions on at least one strand, optionally via a linker or carrier. Another aspect of the invention relates to a method of gene silencing, comprising administering to a cell or a subject in need thereof a therapeutically effective amount of the lipophilic moieties-conjugated double-stranded iRNAs.

Growing community of inventors

Cambridge, MA, United States of America

Muthusamy Jayaraman