BR112022017970A2 - Terapia gênica para degeneração da retina associada ao nmnat1 - Google Patents
Terapia gênica para degeneração da retina associada ao nmnat1Info
- Publication number
- BR112022017970A2 BR112022017970A2 BR112022017970A BR112022017970A BR112022017970A2 BR 112022017970 A2 BR112022017970 A2 BR 112022017970A2 BR 112022017970 A BR112022017970 A BR 112022017970A BR 112022017970 A BR112022017970 A BR 112022017970A BR 112022017970 A2 BR112022017970 A2 BR 112022017970A2
- Authority
- BR
- Brazil
- Prior art keywords
- nmnat1
- gene therapy
- retina degeneration
- degeneration
- associated retina
- Prior art date
Links
- 201000007737 Retinal degeneration Diseases 0.000 title abstract 3
- 238000001415 gene therapy Methods 0.000 title abstract 3
- 101000996052 Homo sapiens Nicotinamide/nicotinic acid mononucleotide adenylyltransferase 1 Proteins 0.000 title 1
- 102100034451 Nicotinamide/nicotinic acid mononucleotide adenylyltransferase 1 Human genes 0.000 title 1
- 101710143608 Nicotinamide/nicotinic acid mononucleotide adenylyltransferase 1 Proteins 0.000 abstract 3
- 238000000034 method Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 230000035772 mutation Effects 0.000 abstract 1
- 230000004258 retinal degeneration Effects 0.000 abstract 1
Classifications
-
- 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
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/45—Transferases (2)
-
- 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
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0048—Eye, e.g. artificial tears
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
- C12Y207/07—Nucleotidyltransferases (2.7.7)
- C12Y207/07001—Nicotinamide-nucleotide adenylyltransferase (2.7.7.1)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Genetics & Genomics (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Ophthalmology & Optometry (AREA)
- Biomedical Technology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Immunology (AREA)
- Dermatology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202062988260P | 2020-03-11 | 2020-03-11 | |
| PCT/US2021/021936 WO2021183779A1 (en) | 2020-03-11 | 2021-03-11 | Gene therapy for nmnat1-associated retinal degeneration |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112022017970A2 true BR112022017970A2 (pt) | 2022-10-25 |
Family
ID=77672442
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112022017970A BR112022017970A2 (pt) | 2020-03-11 | 2021-03-11 | Terapia gênica para degeneração da retina associada ao nmnat1 |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US12257320B2 (https=) |
| EP (1) | EP4117644A4 (https=) |
| JP (1) | JP2023517929A (https=) |
| KR (1) | KR20220152550A (https=) |
| CN (1) | CN115379834A (https=) |
| AU (1) | AU2021236233A1 (https=) |
| BR (1) | BR112022017970A2 (https=) |
| CA (1) | CA3174781A1 (https=) |
| IL (1) | IL296262A (https=) |
| MX (1) | MX2022011177A (https=) |
| WO (1) | WO2021183779A1 (https=) |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2008501343A (ja) * | 2004-06-04 | 2008-01-24 | ワシントン・ユニバーシティ | 神経障害を治療するための方法および組成物 |
| EP3030665B9 (en) | 2013-08-05 | 2023-10-04 | Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum | Recombinant aav-crumbs homologue composition and methods for treating lca-8 and progressive rp |
| KR102537394B1 (ko) * | 2014-03-17 | 2023-05-30 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법 |
| IL248102B (en) | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| PT3191139T (pt) | 2014-07-24 | 2020-11-05 | Massachusetts Eye & Ear Infirmary | Terapia do gene rpgr para retinite pigmentosa |
| PL3265568T3 (pl) * | 2015-03-06 | 2020-11-30 | Massachusetts Eye & Ear Infirmary | Terapie augmentacji genów w przypadku wrodzonego zwyrodnienia siatkówki spowodowanego mutacjami genu prpf31 |
| EP3364973A1 (en) * | 2015-10-23 | 2018-08-29 | The Jackson Laboratory | Nicotinamide for use in the treatment and prevention of ocular neurodegenerative disorder (e.g. glaucoma) |
| US11260081B2 (en) | 2016-06-29 | 2022-03-01 | Ramot At Tel-Aviv University Ltd. | Engineered parasites for delivering protein to the central nervous system (CNS) |
| CN116509885A (zh) | 2016-12-21 | 2023-08-01 | 新南创新私人有限公司 | 增加血管密度的方法 |
| KR20200036912A (ko) * | 2017-07-31 | 2020-04-07 | 리플렉션 바이오테크놀러지스 리미티드 | 안과 질환을 위한 세포 모델 및 치료요법 |
-
2021
- 2021-03-11 US US17/199,115 patent/US12257320B2/en active Active
- 2021-03-11 KR KR1020227034618A patent/KR20220152550A/ko active Pending
- 2021-03-11 WO PCT/US2021/021936 patent/WO2021183779A1/en not_active Ceased
- 2021-03-11 AU AU2021236233A patent/AU2021236233A1/en active Pending
- 2021-03-11 CA CA3174781A patent/CA3174781A1/en active Pending
- 2021-03-11 BR BR112022017970A patent/BR112022017970A2/pt not_active Application Discontinuation
- 2021-03-11 CN CN202180027825.0A patent/CN115379834A/zh active Pending
- 2021-03-11 EP EP21767865.5A patent/EP4117644A4/en active Pending
- 2021-03-11 JP JP2022554440A patent/JP2023517929A/ja active Pending
- 2021-03-11 MX MX2022011177A patent/MX2022011177A/es unknown
- 2021-03-11 IL IL296262A patent/IL296262A/en unknown
-
2025
- 2025-02-21 US US19/060,033 patent/US20250269068A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2023517929A (ja) | 2023-04-27 |
| IL296262A (en) | 2022-11-01 |
| WO2021183779A1 (en) | 2021-09-16 |
| KR20220152550A (ko) | 2022-11-16 |
| MX2022011177A (es) | 2022-12-13 |
| CA3174781A1 (en) | 2021-09-16 |
| EP4117644A1 (en) | 2023-01-18 |
| US20250269068A1 (en) | 2025-08-28 |
| US20210299277A1 (en) | 2021-09-30 |
| AU2021236233A1 (en) | 2022-09-22 |
| US12257320B2 (en) | 2025-03-25 |
| CN115379834A (zh) | 2022-11-22 |
| EP4117644A4 (en) | 2024-08-14 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| B11A | Dismissal acc. art.33 of ipl - examination not requested within 36 months of filing | ||
| B11Y | Definitive dismissal - extension of time limit for request of examination expired [chapter 11.1.1 patent gazette] |