Methods of treating phenylketonuria with mrna encoding phenylalanine hydroxylase (pah)
AssigneeCureVac AG
InventorsMariola Fotin-Mleczek, Patrick Baumhof
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AssigneeCureVac AG
InventorsMariola Fotin-Mleczek, Patrick Baumhof
Estimating the term…
The present invention relates to a complexed RNA, comprising at least one RNA complexed with one or more oligopeptides, wherein the oligopeptide has a length of 8 to 15 amino acids and has the empirical formula (Arg)l;(;Lys)m;(His)n;(Orn)o;(Xaa)x. The invention further relates to a method for transfecting a cell or an organism, thereby applying the inventive complexed RNA. Additionally, pharmaceutical compositions and kits comprising the inventive complexed RNA, as well as the use of the inventive complexed RNA for transfecting a cell, tissue or an organism and/or for modulating, preferably inducing or enhancing, an immune response are disclosed herein.
A method of treating a subject having phenylketonuria comprising administering an effective amount of a pharmaceutical composition comprising a purified mRNA encoding phenylalanine hydroxylase (PAH) to the subject, wherein the mRNA comprises a 5′ cap structure;
A method of inducing or enhancing an innate immune response in a subject in need thereof, comprising administering to the subject a complexed ribonucleic acid (RNA) comprising at least one RNA molecule complexed with one or more oligopeptides, wherein the at least one RNA molecule is not covalently bound to the one or more oligopeptides, wherein the nitrogen/phosphate ratio (N/P-ratio) of the RNA to the one or more oligopeptides is in the range of 0.75-25, and wherein the oligopeptide is 8 to 15 amino acids in length and comprises the following formula:
A method of treating a subject having an ornithine transcarbamylase deficiency comprising administering an effective amount of a pharmaceutical composition comprising mRNA encoding human omrnithine transcarbamylase (OTC) to the subject, wherein the mRNA comprises a 5′ cap structure;
Dependencies are inferred from the claim text.