SG11202112078TA - Compositions and methods for the treatment of atpase-mediated diseases - Google Patents
Compositions and methods for the treatment of atpase-mediated diseasesInfo
- Publication number
- SG11202112078TA SG11202112078TA SG11202112078TA SG11202112078TA SG11202112078TA SG 11202112078T A SG11202112078T A SG 11202112078TA SG 11202112078T A SG11202112078T A SG 11202112078TA SG 11202112078T A SG11202112078T A SG 11202112078TA SG 11202112078T A SG11202112078T A SG 11202112078TA
- Authority
- SG
- Singapore
- Prior art keywords
- atpase
- compositions
- treatment
- methods
- mediated diseases
- Prior art date
Links
- 201000010099 disease Diseases 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title 1
- 230000001404 mediated effect Effects 0.000 title 1
- 239000000203 mixture Substances 0.000 title 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
-
- 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
- A61K38/46—Hydrolases (3)
- A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
-
- 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
- A61K38/46—Hydrolases (3)
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- 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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
- C12Y306/01—Hydrolases acting on acid anhydrides (3.6) in phosphorus-containing anhydrides (3.6.1)
- C12Y306/0103—Hydrolases acting on acid anhydrides (3.6) in phosphorus-containing anhydrides (3.6.1) m7G(5')pppN diphosphatase (3.6.1.30)
-
- 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
- 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
-
- 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
- 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
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
-
- 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)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Toxicology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962847416P | 2019-05-14 | 2019-05-14 | |
| PCT/US2020/032978 WO2020232297A1 (en) | 2019-05-14 | 2020-05-14 | Compositions and methods for the treatment of atpase-mediated diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SG11202112078TA true SG11202112078TA (en) | 2021-11-29 |
Family
ID=73289760
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11202112078TA SG11202112078TA (en) | 2019-05-14 | 2020-05-14 | Compositions and methods for the treatment of atpase-mediated diseases |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US11717560B2 (pt) |
| EP (1) | EP3969597A4 (pt) |
| JP (1) | JP2022531809A (pt) |
| KR (1) | KR20220032005A (pt) |
| CN (1) | CN114072514A (pt) |
| AU (1) | AU2020274200A1 (pt) |
| BR (1) | BR112021022951A2 (pt) |
| CA (1) | CA3140049A1 (pt) |
| IL (1) | IL287796A (pt) |
| MX (1) | MX2021013913A (pt) |
| SG (1) | SG11202112078TA (pt) |
| WO (1) | WO2020232297A1 (pt) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3774854A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
| TW202102525A (zh) | 2019-03-21 | 2021-01-16 | 美商史崔德生物公司 | 重組腺相關病毒載體 |
| MX2022004352A (es) | 2019-10-17 | 2022-07-19 | Stridebio Inc | Vectores virales adenoasociados para el tratamiento de la enfermedad de niemann-pick tipo c. |
| TW202323528A (zh) * | 2021-10-18 | 2023-06-16 | 美商邏輯生物療法公司 | 用於治療威爾森氏病之基因療法 |
| WO2023230466A1 (en) * | 2022-05-23 | 2023-11-30 | Lutsenka Svetlana | Compositions and methods for treating wilson's disease |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2138601A (en) * | 1999-12-22 | 2001-07-03 | Aventis Pasteur Limited | Chlamydia antigens and corresponding DNA fragments and uses thereof |
| US7473531B1 (en) * | 2003-08-08 | 2009-01-06 | Colora Corporation | Pancreatic cancer targets and uses thereof |
| US20100095387A1 (en) * | 2005-07-15 | 2010-04-15 | Martin Smith | Methods and reagents for screening new drugs and for treating ion pump associated disorders and diseases |
| US20110038852A1 (en) * | 2009-06-10 | 2011-02-17 | 3-V Biosciences, Inc. | Antivirals that target transporters, carriers, and ion channels |
| CN103732259A (zh) * | 2011-06-24 | 2014-04-16 | 森彻斯有限公司 | 细胞外靶向药物缀合物 |
| US20140350087A9 (en) * | 2012-03-22 | 2014-11-27 | Halozyme, Inc. | Oncovector Nucleic Acid Molecules and Methods of Use |
| AU2013243946A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of membrane proteins |
| CA2878401C (en) * | 2012-07-06 | 2022-07-19 | University Of Iowa Research Foundation | Modified adeno-associated virus vector compositions |
| ES2693465T3 (es) * | 2014-06-06 | 2018-12-11 | Uroimmun Medizinische Labordiagnostika Ag | Diagnóstico de una enfermedad neurológica |
| JP6865167B2 (ja) * | 2014-12-17 | 2021-04-28 | フンダシオン パラ ラ インベスティガシオン メディカ アプリカダ | ウィルソン病の処置に使用するための核酸構築物及び遺伝子治療ベクター |
| EP3907231A1 (en) * | 2015-07-19 | 2021-11-10 | Yeda Research and Development Co., Ltd. | Selective inhibitors of alpha2-containing isoforms of na,k-atpase and use thereof for reducing intraocular pressure |
| US11738093B2 (en) * | 2018-04-09 | 2023-08-29 | Hope For Annabel | AAV-mediated delivery of ATP1A3 genes to central nervous system |
| ES2970216T3 (es) * | 2019-01-04 | 2024-05-27 | Ultragenyx Pharmaceutical Inc | Constructos de terapia génica para tratar la enfermedad de Wilson |
-
2020
- 2020-05-14 EP EP20805008.8A patent/EP3969597A4/en active Pending
- 2020-05-14 AU AU2020274200A patent/AU2020274200A1/en not_active Abandoned
- 2020-05-14 BR BR112021022951A patent/BR112021022951A2/pt not_active Application Discontinuation
- 2020-05-14 SG SG11202112078TA patent/SG11202112078TA/en unknown
- 2020-05-14 JP JP2021567820A patent/JP2022531809A/ja active Pending
- 2020-05-14 CA CA3140049A patent/CA3140049A1/en active Pending
- 2020-05-14 CN CN202080047216.7A patent/CN114072514A/zh active Pending
- 2020-05-14 KR KR1020217040927A patent/KR20220032005A/ko unknown
- 2020-05-14 MX MX2021013913A patent/MX2021013913A/es unknown
- 2020-05-14 WO PCT/US2020/032978 patent/WO2020232297A1/en unknown
-
2021
- 2021-11-02 IL IL287796A patent/IL287796A/en unknown
- 2021-11-11 US US17/524,466 patent/US11717560B2/en active Active
-
2023
- 2023-06-23 US US18/340,028 patent/US20240075111A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| KR20220032005A (ko) | 2022-03-15 |
| CA3140049A1 (en) | 2020-11-19 |
| US20220088152A1 (en) | 2022-03-24 |
| US11717560B2 (en) | 2023-08-08 |
| EP3969597A4 (en) | 2023-01-25 |
| IL287796A (en) | 2022-01-01 |
| CN114072514A (zh) | 2022-02-18 |
| BR112021022951A2 (pt) | 2022-01-25 |
| AU2020274200A1 (en) | 2021-11-25 |
| US20240075111A1 (en) | 2024-03-07 |
| JP2022531809A (ja) | 2022-07-11 |
| EP3969597A1 (en) | 2022-03-23 |
| MX2021013913A (es) | 2022-03-17 |
| WO2020232297A1 (en) | 2020-11-19 |
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