PE20240137A1 - Composiciones y metodos para mejorar la funcion visual - Google Patents
Composiciones y metodos para mejorar la funcion visualInfo
- Publication number
- PE20240137A1 PE20240137A1 PE2023003082A PE2023003082A PE20240137A1 PE 20240137 A1 PE20240137 A1 PE 20240137A1 PE 2023003082 A PE2023003082 A PE 2023003082A PE 2023003082 A PE2023003082 A PE 2023003082A PE 20240137 A1 PE20240137 A1 PE 20240137A1
- Authority
- PE
- Peru
- Prior art keywords
- visual function
- methods
- compositions
- opsin
- individual
- Prior art date
Links
- 230000004382 visual function Effects 0.000 title abstract 3
- 239000000203 mixture Substances 0.000 title abstract 2
- 108010054609 middle-wavelength opsin Proteins 0.000 abstract 2
- 108050001704 Opsin Proteins 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 102000040430 polynucleotide Human genes 0.000 abstract 1
- 108091033319 polynucleotide Proteins 0.000 abstract 1
- 239000002157 polynucleotide Substances 0.000 abstract 1
- 230000002207 retinal effect Effects 0.000 abstract 1
- 230000003612 virological effect Effects 0.000 abstract 1
Classifications
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- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
-
- 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
-
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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- 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
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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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
-
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
-
- 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
- C12N2750/14145—Special targeting system for viral vectors
-
- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Cell Biology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Ophthalmology & Optometry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Toxicology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Dental Preparations (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
La presente revelacion presenta composiciones y metodos para restaurar o mejorar la funcion visual en un individuo mediante la administracion a dicho individuo de una composicion farmaceutica que comprende un vector viral adeno-asociado recombinante (rAAV) que tiene una secuencia de polinucleotidos que codifica una opsina de cono de longitud de onda media (MW-opsina). La MW-opsina se expresa en una celula de la retina en el individuo, restaurando o mejorando asi la funcion visual
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202163191525P | 2021-05-21 | 2021-05-21 | |
US202163292746P | 2021-12-22 | 2021-12-22 | |
PCT/IB2022/054650 WO2022243913A1 (en) | 2021-05-21 | 2022-05-19 | Compositions and methods for enhancing visual function |
Publications (1)
Publication Number | Publication Date |
---|---|
PE20240137A1 true PE20240137A1 (es) | 2024-01-30 |
Family
ID=81928130
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PE2023003082A PE20240137A1 (es) | 2021-05-21 | 2022-05-19 | Composiciones y metodos para mejorar la funcion visual |
Country Status (15)
Country | Link |
---|---|
US (1) | US20230049217A1 (es) |
EP (1) | EP4341412A1 (es) |
JP (1) | JP2024522072A (es) |
KR (1) | KR20240010489A (es) |
AU (1) | AU2022278028A1 (es) |
CA (1) | CA3218689A1 (es) |
CO (1) | CO2023017779A2 (es) |
CR (1) | CR20230599A (es) |
DO (1) | DOP2023000255A (es) |
EC (1) | ECSP23095204A (es) |
IL (1) | IL308027A (es) |
PE (1) | PE20240137A1 (es) |
TW (1) | TW202313981A (es) |
UY (1) | UY39772A (es) |
WO (1) | WO2022243913A1 (es) |
Family Cites Families (9)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
IL105914A0 (en) | 1992-06-04 | 1993-10-20 | Univ California | Methods and compositions for in vivo gene therapy |
USH2191H1 (en) * | 2000-10-24 | 2007-06-05 | Snp Consortium | Identification and mapping of single nucleotide polymorphisms in the human genome |
WO2006043354A1 (ja) * | 2004-10-20 | 2006-04-27 | National Institute Of Radiological Sciences | 組込型の低線量放射線誘導性ベクター |
EP3119798B1 (en) * | 2014-03-17 | 2020-08-05 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
MA41346A (fr) * | 2015-01-12 | 2017-11-21 | Juno Therapeutics Inc | Eléments régulateurs post-transcriptionnels d'hépatite modifiée |
EP3210632B1 (en) * | 2016-02-23 | 2022-09-28 | EyeServ GmbH | Gene therapy for the treatment of a retinal degeneration disease |
KR20230039779A (ko) | 2016-07-29 | 2023-03-21 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법 |
EP3720460A1 (en) * | 2017-12-05 | 2020-10-14 | University of Washington | Compositions and methods for enhancing functional expression of therapeutic genes in photoreceptors |
KR20220108096A (ko) * | 2019-11-29 | 2022-08-02 | 파로스 바이오, 인코포레이티드 | 신경퇴행성 질환에 대한 유전자 요법 |
-
2022
- 2022-05-18 UY UY0001039772A patent/UY39772A/es unknown
- 2022-05-18 US US17/747,881 patent/US20230049217A1/en active Pending
- 2022-05-19 AU AU2022278028A patent/AU2022278028A1/en active Pending
- 2022-05-19 WO PCT/IB2022/054650 patent/WO2022243913A1/en active Application Filing
- 2022-05-19 IL IL308027A patent/IL308027A/en unknown
- 2022-05-19 PE PE2023003082A patent/PE20240137A1/es unknown
- 2022-05-19 CR CR20230599A patent/CR20230599A/es unknown
- 2022-05-19 TW TW111118711A patent/TW202313981A/zh unknown
- 2022-05-19 JP JP2023571759A patent/JP2024522072A/ja active Pending
- 2022-05-19 CA CA3218689A patent/CA3218689A1/en active Pending
- 2022-05-19 EP EP22727448.7A patent/EP4341412A1/en active Pending
- 2022-05-19 KR KR1020237043720A patent/KR20240010489A/ko unknown
-
2023
- 2023-11-16 DO DO2023000255A patent/DOP2023000255A/es unknown
- 2023-12-19 EC ECSENADI202395204A patent/ECSP23095204A/es unknown
- 2023-12-19 CO CONC2023/0017779A patent/CO2023017779A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
EP4341412A1 (en) | 2024-03-27 |
CA3218689A1 (en) | 2022-11-24 |
IL308027A (en) | 2023-12-01 |
CR20230599A (es) | 2024-01-25 |
WO2022243913A1 (en) | 2022-11-24 |
CO2023017779A2 (es) | 2024-01-15 |
JP2024522072A (ja) | 2024-06-11 |
ECSP23095204A (es) | 2024-01-31 |
US20230049217A1 (en) | 2023-02-16 |
AU2022278028A1 (en) | 2023-11-09 |
AU2022278028A9 (en) | 2023-11-16 |
DOP2023000255A (es) | 2023-12-29 |
TW202313981A (zh) | 2023-04-01 |
UY39772A (es) | 2023-01-31 |
KR20240010489A (ko) | 2024-01-23 |
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