mRNA Encoding Engineered CFTR
AssigneeTranslate Bio, Inc.
InventorsRichard Wooster, Frank DeRosa, Lianne Boeglin, Priyaanka Nanduri, Anusha Dias, Khang Anh Tran
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Abstract
The present invention provides, among other things, improved methods and pharmaceutical compositions for treating cystic fibrosis based on codon optimized mRNA encoding an engineered or mutant Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein.
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A pharmaceutical composition for treating cystic fibrosis, comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), wherein the CFTR comprises one or more mutations that produce an activated CFTR protein that is more stable than a wild type CFTR protein, wherein the mutation is an amino acid substitution.
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A nucleic acid encoding a modified Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, wherein the modified CFTR protein comprises one or more amino acid substitutions to glutamic acid.
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A modified CFTR protein comprising one or more amino acid substitutions to glutamic acid or at serine residues or at threonine residues,
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- wherein the modified CFTR protein further comprises one or more mutations in the CFTR regulatory domain, a membrane-spanning domain (MSD), and/or a nucleotide-binding domain.
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Dependencies are inferred from the claim text.