Messenger rna therapy for treatment of ocular diseases

The method of any one of the preceding claims, wherein administering the composition results in expression of the protein encoded by the mRNA in the retinal tissue.

The method of any one of the preceding claims, wherein administering the composition results in expression of the protein encoded by the mRNA in the choroid.

The method of any one of the preceding claims, wherein administering the composition results in expression of the protein encoded by the mRNA in the sclera.

The method of any one of the preceding claims, wherein the effective amount of mRNA administered to the subject ranges from 0.01 μg to 500 μg mRNA.

The method of any one of the preceding claims, wherein the effective amount of mRNA administered to the subject ranges from 0.025 μg to 100 μg mRNA.

The method of any one of the preceding claims, wherein the effective amount of mRNA administered to the subject ranges from 0.05 μg to 50 μg mRNA.

The method of any one of the preceding claims, wherein the effective amount of mRNA administered to the subject is about 0.0625 μg, or about 0.125 μg, or about 0.25 μg, or about 0.5 μg, or about 1 μg.

The method of any one of the preceding claims, wherein the subject is human.

The method of any one of the preceding claims, wherein subject is suffering from a disease or disorder affecting the anterior retinal layers.

The method of any one of the preceding claims, wherein the lipid nanoparticle comprises one or more cationic lipids, one or more non-cationic lipids and a PEG-modified lipid.

A method of treating an ocular disease or disorder in a subject in need thereof, comprising:

• administering to an eye of the subject a composition comprising an effective amount of mRNA encoding a protein,
• wherein the mRNA is encapsulated in a lipid nanoparticle, and
• wherein administering the composition results in expression of the protein or the peptide encoded by the mRNA in one or more cells located in the nerve fiber layer, the ganglionic cell layer (GCL), the inner plexiform layer (IPL), the inner nuclear layer (INL), the outer plexiform layer (OPL), the outer nuclear layer (ONL), the inner segment photoreceptors (IS), the outer segment photoreceptors (OS), the retinal pigmented epithelium layer (RPE) of the retinal tissue, the choroid, and/or the sclera of the eye.

The method of any one of the preceding claims, wherein the mRNA encodes a protein or a peptide selected from a group consisting of an ocular protein or a peptide, a vaccine, an antibody or a fragment thereof, a hormone, a structural protein or peptide, an extracellular matrix protein or peptide, a vascular protein or peptide, an anti-tumor protein or peptide, an angiogenic protein or peptide, an anti-angiogenic protein or peptide, an antioxidant protein or peptide, a receptor protein or peptide, a signaling protein or peptide, a transcription factor and an enzyme.

The method of any one of the preceding claims, wherein the mRNA encodes an ocular protein or a peptide selected from a group consisting of ADAM metallopeptidase domain 9, adhesins, ATP synthase, bestrophin 1, cadherins, chemokines, ciliary neurotrophic factor, collagens, complement factors, cytochromes, IGF, metalloproteinases, mitofusin, NADH dehydrogenase, OPA1, PDGF, peripherin 2, retinoschisin, SOD2, thrombospondin receptor, and vascular endothelial growth factor (VEGF).

The method of any one of the preceding claims, wherein the mRNA encodes an antibody or a fragment thereof, that binds to ADAM metallopeptidase domain 9, adhesins, ATP synthase, bestrophin 1, cadherins, chemokines, ciliary neurotrophic factor, collagens, complement factors, cytochromes, IGF, metalloproteinases, mitofusin, NADH dehydrogenase, OPA1, PDGF, peripherin 2, retinoschisin, SOD2, thrombospondin receptor, or vascular endothelial growth factor (VEGF).

The method of any one of the preceding claims, wherein the mRNA encodes an antibody or a fragment thereof that binds to VEGF.