WO2023039175A3 - Predictors of achromatopsia treatment efficacy - Google Patents
Predictors of achromatopsia treatment efficacy Download PDFInfo
- Publication number
- WO2023039175A3 WO2023039175A3 PCT/US2022/043077 US2022043077W WO2023039175A3 WO 2023039175 A3 WO2023039175 A3 WO 2023039175A3 US 2022043077 W US2022043077 W US 2022043077W WO 2023039175 A3 WO2023039175 A3 WO 2023039175A3
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- achromatopsia
- predictors
- treatment efficacy
- subject
- treatment
- Prior art date
Links
- 208000006992 Color Vision Defects Diseases 0.000 title abstract 3
- 201000000761 achromatopsia Diseases 0.000 title abstract 3
- 201000007254 color blindness Diseases 0.000 title abstract 3
- 208000017442 Retinal disease Diseases 0.000 abstract 2
- 238000000034 method Methods 0.000 abstract 1
- 230000002207 retinal effect Effects 0.000 abstract 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61B—DIAGNOSIS; SURGERY; IDENTIFICATION
- A61B3/00—Apparatus for testing the eyes; Instruments for examining the eyes
- A61B3/10—Objective types, i.e. instruments for examining the eyes independent of the patients' perceptions or reactions
- A61B3/102—Objective types, i.e. instruments for examining the eyes independent of the patients' perceptions or reactions for optical coherence tomography [OCT]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Gastroenterology & Hepatology (AREA)
- Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Toxicology (AREA)
- Genetics & Genomics (AREA)
- Immunology (AREA)
- Molecular Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Cell Biology (AREA)
- Ophthalmology & Optometry (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
The present invention provides methods for treating or selecting a subject for treatment of a retinal disease or disorder, including, without limitation, achromatopsia (ACHM), based on an analysis of the subject's retinal structure and/or function.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202163242448P | 2021-09-09 | 2021-09-09 | |
US63/242,448 | 2021-09-09 |
Publications (2)
Publication Number | Publication Date |
---|---|
WO2023039175A2 WO2023039175A2 (en) | 2023-03-16 |
WO2023039175A3 true WO2023039175A3 (en) | 2023-04-27 |
Family
ID=85506810
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/US2022/043077 WO2023039175A2 (en) | 2021-09-09 | 2022-09-09 | Predictors of achromatopsia treatment efficacy |
Country Status (1)
Country | Link |
---|---|
WO (1) | WO2023039175A2 (en) |
Citations (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20140364486A1 (en) * | 2013-05-16 | 2014-12-11 | Guo-jie Ye | Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases |
US20200392536A1 (en) * | 2018-01-12 | 2020-12-17 | Ucl Business Ltd | Compositions and Methods for Treating Retinal Disorders |
-
2022
- 2022-09-09 WO PCT/US2022/043077 patent/WO2023039175A2/en unknown
Patent Citations (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20140364486A1 (en) * | 2013-05-16 | 2014-12-11 | Guo-jie Ye | Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases |
US20200392536A1 (en) * | 2018-01-12 | 2020-12-17 | Ucl Business Ltd | Compositions and Methods for Treating Retinal Disorders |
Non-Patent Citations (3)
Title |
---|
FAHIM ET AL.: "Diagnostic Fundus Autofluorescence Patterns in Achromatopsia", AMERICAN JOURNAL OF OPHTHALMOLOGY, vol. 156, no. 6, 2013, pages 1211 - 1219, XP028773804, DOI: 10.1016/j.ajo.2013.06.033 * |
GREENBERG JONATHAN P., SHERMAN JEROME, ZWEIFEL SANDRINE A., CHEN ROYCE W. S., DUNCKER TOBIAS, KOHL SUSANNE, BAUMANN BRITTA, WISSIN: "Spectral-Domain Optical Coherence Tomography Staging and Autofluorescence Imaging in Achromatopsia", JAMA OPHTHALMOLOGY, AMERICAN MEDICAL ASSOCIATION, US, vol. 132, no. 4, 1 April 2014 (2014-04-01), US , pages 437, XP093064735, ISSN: 2168-6165, DOI: 10.1001/jamaophthalmol.2013.7987 * |
LI DANJIE, KISHI SHOJI: "Loss of Photoreceptor Outer Segment in Acute Zonal Occult Outer Retinopathy", ARCHIVES OF OPHTHALMOLOGY, AMERICAN MEDICAL ASSOCIATION, UNITED STATES, vol. 125, no. 9, 1 September 2007 (2007-09-01), United States , pages 1194, XP093064738, ISSN: 0003-9950, DOI: 10.1001/archopht.125.9.1194 * |
Also Published As
Publication number | Publication date |
---|---|
WO2023039175A2 (en) | 2023-03-16 |
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