Modernatx, Inc.

Athanasios Dousis

Kanchana Ravichandran

Amy E Rabideau

Elissa Hobert

Kerry Benenato

Stephen G Hoge

Vladimir Presnyak

Ellalahewage Sathyajith Kumarasinghe

Iain McFadyen

Zhinan Xia

Margaret Franklin

Kevin Smith

Michelle Lynn Hall

Barry Ticho

Nadege Briancon-Eris

Seymour De Picciotto

Patrick Finn

Ding An

Paolo G V Martini

Jingsong Cao

Jesse Chen

Raj Rajendran

Paolo GV Martini

The present disclosure provides, in some aspects, variant RNA polymerases, the use of which increases transcription efficiency while reducing the number of double-stranded RNA contaminates and run-on transcripts produced during an in vitro transcription reaction.

RNA polymerase variants

The present disclosure provides RNA polymerase variants for high efficiency transcription.

RNA polymerase variants for co-transcriptional capping

The invention relates to mRNA therapy for the treatment of fibrosis and/or cardiovascular disease. mRNAs for use in the invention, when administered in vivo, encode human relaxin, isoforms thereof, functional fragments thereof, and fusion proteins comprising relaxin. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of relaxin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient relaxin activity in subjects.

Polynucleotides encoding relaxin

This disclosure relates to mRNA therapy for the treatment of hyperphenylalaninemias such as phenylketonuria (PKU). mRNAs for use in the invention, when administered in vivo, encode human phenylalanine hydroxylase (PAH), functional fragments thereof (e.g., those comprising the catalytic domain or the catalytic domain and the tetramerization domains), and fusion proteins comprising PAH. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of PAH expression and/or activity in subjects. mRNA therapies of the invention further decrease abnormal accumulation of phenylalanine associated with deficient PAH activity in subjects.

Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria

The present disclosure provides, in some aspects, in vitro transcription systems (including, for example, nucleic acid constructs and polymerases), the use of which increases transcription efficiency while reducing the amount of truncated single-stranded ribonucleic acid transcript produced during an in vitro transcription reaction.

In vitro transcription methods and constructs

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Cambridge, MA, United States of America

Athanasios Dousis