Translate Bio, Inc.

Shire Human Genetic Therapies, Inc.

Ethris Gmbh

Novartis Ag

Immunogen, Inc.

Apoptosis Technology, Inc.

Rana Therapeutics, Inc.

Braydon Charles Guild

Michael W Heartlein

Frank DeRosa

Lianne Smith

Carsten Rudolph

Christian Plank

Jerry Chi Zhang

Anusha Dias

Michael Roguska

Igor Splawski

Beate Diefenbach-Streiber

Adina Eberth

Yong-In Kim

Sophie M Lehar

Frank Derosa

Michael F Concino

Anthony R Rees

Stephen M Searle

Jan T Pedersen

Michael A Roguska

Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.

Ionizable cationic lipids

Disclosed herein are pharmaceutical compositions that comprise 'blends' of lipid nanoparticles and related methods of using such blended compositions to deliver polynucleotides to one or more target cells, tissues or organs. The blended compositions are generally characterized as being able to efficiently deliver polynucleotides to target cells and by their ability to enhance the expression of such polynucleotides and the production of functional proteins by target cells.

Synergistic enhancement of the delivery of nucleic acids via blended formulations

Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.

Lipid nanoparticle compositions and methods for mRNA delivery

Cleavable lipids

Lipid nanoparticle compositions and methods for MRNA delivery

The present disclosure relates to materials, formulations, production methods, and methods for delivery of CFTR mRNA, including but not limited to chemically modified mRNA for induction of CFTR expression, including in the mammalian lung. The present invention is particularly useful for treating cystic fibrosis, but is also useful in the treatment of diseases related to CFTR gene.

CFTR MRNA compositions and related methods and uses

Disclosed herein are novel lipids and liposomal compositions prepared using such compounds and related methods of neutralizing or otherwise modifying such liposomal compositions. The lipids described herein are useful for example, as liposomal vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and the subsequent transfection of such target cells. In certain embodiments, one or more of the compounds that comprise the liposomal delivery vehicle may be neutralized or further modified such that the properties of the liposomal delivery vehicle are modified.

Lipid-derived neutral nanoparticles

Growing community of inventors

Concord, MA, United States of America

Braydon Charles Guild