Translate Bio, Inc.

Ethris Gmbh

Shire Human Genetic Therapies, Inc.

Rana Therapeutics, Inc.

Lianne Smith

Michael W Heartlein

Frank DeRosa

Braydon Charles Guild

Shrirang Karve

Carsten Rudolph

Christian Plank

Ann Barbier

Jou-Ku Chung

Thomas McCauley

Kim Askew

Frank Derosa

Michael Heartlein

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

The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.

Composition and methods for treatment of ornithine transcarbamylase deficiency

The present invention provides, among other things, methods of treating ornithine transcarbamylase (OTC) deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase (OTC) protein at an effective dose and an administration interval such that at least one symptom or feature of the OTC deficiency is reduced in intensity, severity, or frequency or has delayed onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.

Messenger RNA therapy for the treatment of ornithine transcarbamylase deficiency

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

The present invention provides, among other things, methods of treating Argininosuccinate Synthetase Deficiency (ASD), including administering to a subject in need of treatment a composition comprising an mRNA encoding argininosuccinate synthetase (ASS1) at an effective dose and an administration interval such that at least one symptom or feature of ASD is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.

MRNA therapy for argininosuccinate synthetase deficiency

The present invention is particularly useful for treating cystic fibrosis. The disclosure concerns method and compositions for treating cystic fibrosis. The method comprises inducing CFTR expression in epithelial cells in a lung of a mammal by contacting the epithelial cells in the lung of the mammal with a composition, where the composition is a pharmaceutical composition comprising in vitro transcribed mRNA.

Pharmaceutical compositions comprising an mRNA-loaded nanoparticle, wherein the mRNA is an in vitro transcribed mRNA and has a coding sequence at least 80% identical to SEQ ID NO: 3, and wherein the mRNA encodes a human cystic fibrosis transmembrane conductance regulator (CFTR) protein comprising the amino acid sequence of SEQ ID NO:1 are provided. The present invention is particularly useful for treating cystic fibrosis.

CFTR mRNA compositions and related methods and uses

Materials, formulations, production methods, and methods for delivery of CFTR mRNA for induction of CFTR expression, including in the mammalian lung are provided. The present invention is particularly useful for treating cystic fibrosis.

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Lexington, MA, United States of America

Lianne Smith