Modernatx, Inc.

Takeda Pharmaceutical Company Limited

Paolo G V Martini

Patrick Finn

Vladimir Presnyak

Athanasios Dousis

Albert Barnes Seymour

Kanchana Ravichandran

Madhusudan Natarajan

Ding An

Patrick Anthony John Haslett

Andrea Lea Frassetto

Zhijian Zhuo

Raj Rajendran

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

This disclosure relates mRNA therapy for the treatment of ornithine transcarbamylase deficiency (OTCD). mRNAs for use in the invention, when administered in vivo, encode human ornithine transcarbamylase (OTC), isoforms thereof, functional fragments thereof, and fusion proteins comprising OTC. 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 OTC expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic ammonia associated with deficient OTC activity in subjects.

Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders

The present invention provides, among other things, bi-specific molecules including, but not limited to, antibodies, fynomers, aptamers, fusion proteins, and protein binding domains that bind both CCL2 and LOXL2 and uses thereof, in particular, for treatment of scleroderma and related fibrotic and/or inflammatory diseases, disorders and conditions. In some embodiments, the present invention further provides methods and compositions for treatment of scleroderma and related fibrotic and/or inflammatory diseases, disorders and conditions based on the combination of mono-specific anti-CCL2 and anti-LOXL2 molecules.

Anti-CCL2 and anti-LOXL2 combination therapy for treatment of scleroderma

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Boston, MA, United States of America

Paolo G V Martini