Arcturus Therapeutics, Inc.

Arisa Cale

Padmanabh Chivukula

Kiyoshi Tachikawa

Pattraranee Limphong

Daiki Matsuda

Carlos Gustavo Perez-Garcia

Carlos G Perez-garcia

Angel I-Jou Leu

Jared Davis

A range of therapeutic mRNA molecules expressible to provide a target polypeptide or protein. The RNA molecules can contain one or more 5-methoxyuridines and 5-methylcytidines. Further provided are DNA templates, which can be transcribed to provide a target mRNA, and can have altered nucleotides, such as reduced deoxyadenosines. Also provided are processes for making the therapeutic mRNA molecules. The RNA molecules can be translated in vitro or in vivo to provide an active polypeptide or protein. The RNA molecules can be included in a composition used for preventing, treating, or ameliorating at least one symptom of a disease or condition in a subject in need thereof.

Translatable molecules and synthesis thereof

This invention provides expressible polynucleotides, which can express a target protein or polypeptide. Synthetic mRNA constructs for producing a protein or polypeptide can contain one or more 5' UTRs, where a 5&#x2032; UTR may be expressed by a gene of a plant. In some embodiments, a 5&#x2032; UTR may be expressed by a gene of a member ofgenus. The synthetic mRNA constructs can be used as pharmaceutical agents for expressing a target protein or polypeptide in vivo.

Synthesis and structure of high potency RNA therapeutics

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San Diego, CA, United States of America

Arisa Cale