# US 2019/0169244 A1 · Compositions And Methods For Enhancing Visual Function

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- Number: US 2019/0169244 A1
- Document ID: US20190169244A1
- Status: Published application
- Kind: A1
- Application number: 16/279,576
- Filed: 2019-02-19
- Published: 2019-06-06
- Earliest claimed: 2017-11-13
- Assignees: The Regents of the University of California
- Applicants: The Regents of the University of California
- Inventors: Ehud Y. ISACOFF
- Web page: https://patentgrep.com/patent/US20190169244A1

## CPC

- C07K 14/47: from mammals
- A61K 9/0019: {Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner (non-active ingredients are additionally classified in A61K47/00)}
- A61K 9/0048: {Eye, e.g. artificial tears}
- A61K 9/5138: {obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyvinyl pyrrolidone, poly(meth)acrylates}
- A61K 9/5153: {Polyesters, e.g. poly(lactide-co-glycolide)}
- A61K 48/0058: {Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct}
- A61K 48/0075: {characterised by an aspect of the delivery route, e.g. oral, subcutaneous}
- A61K 48/0083: {characterised by an aspect of the administration regime}
- A61P 27/04: Artificial tears; Irrigation solutions
- C07K 14/723: {G protein coupled receptor, e.g. TSHR-thyrotropin-receptor, LH/hCG receptor, FSH receptor}
- C12N 15/85: for animal cells
- A61K 38/00: Medicinal preparations containing peptides (peptides containing beta-lactam rings A61K31/00; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, A61K31/00; ergot alkaloids of the cyclic peptide type A61K31/48; containing macromolecular compounds having statistically distributed amino acid units A61K31/74; medicinal preparations containing antigens or antibodies A61K39/00; medicinal preparations characterised by the non-active ingredients, e.g. peptides as drug carriers, A61K47/00)
- C12N 2750/14143: viral genome or elements thereof as genetic vector
- C07K: PEPTIDES (peptides in foodstuffs A23; obtaining protein compositions for foodstuffs, working-up proteins for foodstuffs A23J; preparations for medicinal purposes A61K; peptides containing beta-lactam rings C07D; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, C07D; ergot alkaloids of the cyclic peptide type C07D519/02; macromolecular compounds having statistically distributed amino acid units in their molecules, i.e. when the preparation does not provide for a specific; but for a random sequence of the amino acid units, homopolyamides and block copolyamides derived from amino acids C08G69/00; macromolecular products derived from proteins C08H1/00; preparation of glue or gelatine C09H; single cell proteins, enzymes C12N; genetic engineering processes for obtaining peptides C12N15/00; compositions for measuring or testing processes involving enzymes C12Q; investigation or analysis of biological material G01N33/00)
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)

## Abstract

The present disclosure provides a method of restoring or enhancing visual function in an individual, the method comprising administering to the individual a nucleic acid comprising a nucleotide sequence encoding one or more of a medium wavelength cone opsin (MW-opsin), a long wavelength cone opsin (LW-opsin), and a short wavelength cone opsin (SW-opsin). One or more of the MW-opsin, LW-opsin, and SW-opsin is expressed in a retinal cell in the individual, thereby restoring or enhancing visual function.

## Claims

### Claim at position 1

-54. (canceled)

### Claim 55

A method of restoring or enhancing visual function in an individual, the method comprising administering to the individual at least one nucleic acid comprising a nucleotide sequence encoding a medium wavelength opsin (MW-opsin) and/or a long wavelength opsin (LW-opsin) and/or a short wavelength opsin (SW-opsin), wherein said administering provides for expression of the MW-opsin and/or the LW-opsin and/or the SW-opsin in a retinal cell in the individual and restoration or enhancement of visual function, wherein the retinal cell is a retinal ganglion cell, an amacrine cell, a horizontal cell, or a bipolar cell.

### Claim 56 (depends on 55)

The method of claim 55, wherein the MW-opsin comprises an amino acid sequence having at least 85% amino acid sequence identity to the amino acid sequence set forth in SEQ ID NO:1; wherein the LW-opsin comprises an amino acid sequence having at least 85% amino acid sequence identity to the amino acid sequence set forth in SEQ ID NO:4, and wherein the SW-opsin comprises an amino acid sequence having at least 85% amino acid sequence identity to the amino acid sequence set forth in SEQ ID NO:5.

### Claim 57 (depends on 55)

The method of claim 55, wherein the SW-opsin comprises an amino acid sequence having at least 87% amino acid sequence identity to the human SW-opsin amino acid sequence set forth in SEQ ID NO:5 or having at least 87% amino acid sequence identity to the mouse SW-opsin amino acid sequence set forth in SEQ ID NO:6.

### Claim 58 (depends on 55)

The method of claim 55, wherein the at least one nucleic acid comprises a first nucleic acid and a second nucleic acid, and wherein: i) the first nucleic acid comprises a nucleotide sequence encoding an SW-opsin comprising an amino acid sequence having at least 87% amino acid sequence identity to the human SW-opsin amino acid sequence set forth in SEQ ID NO:5; and ii) the second nucleic acid comprises a nucleotide sequence encoding an SW-opsin comprising an amino acid sequence having at least 87% amino acid sequence identity to the mouse SW-opsin amino acid sequence set forth in SEQ ID NO:6.

### Claim 59 (depends on 55)

The method of claim 55, wherein the SW-opsin is a chimeric SW-opsin comprising mouse SW-opsin intracellular domains and human SW-opsin transmembrane domains.

### Claim 60 (depends on 59)

The method of claim 59, wherein the chimeric SW-opsin comprises an amino acid sequence having at least 90% amino acid sequence identity to the chimeric SW-opsin amino acid sequence set forth in SEQ ID NO:7.

### Claim 61 (depends on 55)

The method of claim 55, wherein said restoring or enhancing provides for patterned vision and image recognition by the individual.

### Claim 62 (depends on 61)

The method of claim 61, wherein the image recognition is of a static image or a pattern.

### Claim 63 (depends on 61)

The method of claim 61, wherein the image recognition is of a moving image or a pattern.

### Claim 64 (depends on 55)

The method of claim 55, wherein said restoring or enhancing provides for image recognition at a light intensity of from about 104 W/cm2 to about 1 W/cm2.

### Claim 65 (depends on 55)

The method of claim 55, wherein expression of the MW-opsin and/or the LW-opsin and/or the SW-opsin in the retinal cell provides for image recognition at a light intensity that is at least 10-fold lower than the light intensity required to provide for image recognition by an individual expressing a channelrhodopsin polypeptide in a retinal cell.

### Claim 66 (depends on 55)

The method of claim 55, wherein expression of the MW-opsin and/or the LW-opsin and/or the SW-opsin in the retinal cell provides for kinetics that are at least 2-fold faster than the kinetics conferred on a retinal cell by a rhodopsin polypeptide.

### Claim 67 (depends on 55)

The method of claim 55, wherein the at least one nucleic acid is a recombinant expression vector.

### Claim 68 (depends on 67)

The method of claim 67, wherein the recombinant expression vector is a recombinant viral vector.

### Claim 69 (depends on 68)

The method of claim 68, wherein the recombinant viral vector is an adeno-associated viral vector, a lentiviral vector, a herpes simplex virus vector, or a retroviral vector.

### Claim 70 (depends on 55)

The method of claim 55, wherein the nucleotide sequence is operably linked to a transcriptional control element that is functional in a retinal cell.

### Claim 71 (depends on 70)

The method of claim 70, wherein the transcriptional control element is a retinal cell-specific promoter.

### Claim 72 (depends on 71)

The method of claim 71, wherein the promoter is a synapsin promoter, a CAG promoter, a CMV promoter, a grm6 promoter, a Pleiades promoter, a ChAT promoter, a V-glut promoter, a GAD promoter, a PV promoter, a somatostatin (SST) promoter, a neuropeptide Y (NPY) promoter, a VIP promoter, a red cone opsin promoter, rhodopsin promoter, a rhodopsin kinase promoter, vitelliform macular dystrophy 2 (VMD2) gene promoter, or an interphotoreceptor retinoid-binding protein (IRBP) gene promoter.

### Claim 73 (depends on 55)

The method of claim 55, wherein said administering is via intraocular injection, intravitreal injection, or subretinal injection.

### Claim 74 (depends on 55)

The method of claim 55, wherein the individual has an ocular disease selected from retinitis pigmentosa, macular degeneration, retinoschisis, and Leber's Congenital Amaurosis, and diabetic retinopathy.

### Claim 75 (depends on 55)

The method of claim 55, wherein the individual has experienced retinal detachment or photoreceptor loss due to trauma or head injury.

### Claim 76 (depends on 55)

The method of claim 55, wherein the retinal cell is a retinal ganglion cell.

### Claim 77 (depends on 55)

The method of claim 55, wherein the at least one nucleic acid is a recombinant adenoassociated viral vector.

### Claim 78 (depends on 55)

The method of claim 55, wherein the individual has retinitis pigmentosa.