KR102618947B1 - 뇌 질환을 치료하기 위한 방법 및 조성물 - Google Patents
뇌 질환을 치료하기 위한 방법 및 조성물 Download PDFInfo
- Publication number
- KR102618947B1 KR102618947B1 KR1020177020915A KR20177020915A KR102618947B1 KR 102618947 B1 KR102618947 B1 KR 102618947B1 KR 1020177020915 A KR1020177020915 A KR 1020177020915A KR 20177020915 A KR20177020915 A KR 20177020915A KR 102618947 B1 KR102618947 B1 KR 102618947B1
- Authority
- KR
- South Korea
- Prior art keywords
- aav
- mice
- rhes
- subject
- mtorc1
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Active
Links
Images
Classifications
-
- 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
-
- 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
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
-
- 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
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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)
- C12Y306/05—Hydrolases acting on acid anhydrides (3.6) acting on GTP; involved in cellular and subcellular movement (3.6.5)
- C12Y306/05002—Small monomeric GTPase (3.6.5.2)
-
- 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)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Genetics & Genomics (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Immunology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Hospice & Palliative Care (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Psychiatry (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462098085P | 2014-12-30 | 2014-12-30 | |
| US62/098,085 | 2014-12-30 | ||
| PCT/US2015/068034 WO2016109649A1 (en) | 2014-12-30 | 2015-12-30 | Methods and compositions for treating brain diseases |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| KR20170098931A KR20170098931A (ko) | 2017-08-30 |
| KR102618947B1 true KR102618947B1 (ko) | 2023-12-27 |
Family
ID=56285031
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020177020915A Active KR102618947B1 (ko) | 2014-12-30 | 2015-12-30 | 뇌 질환을 치료하기 위한 방법 및 조성물 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US11612641B2 (https=) |
| EP (1) | EP3240577B1 (https=) |
| JP (1) | JP6782701B2 (https=) |
| KR (1) | KR102618947B1 (https=) |
| CN (1) | CN107405414A (https=) |
| AU (1) | AU2015374043B2 (https=) |
| BR (1) | BR112017013674A2 (https=) |
| CA (1) | CA2971687C (https=) |
| ES (1) | ES2962439T3 (https=) |
| SG (1) | SG11201704829QA (https=) |
| WO (1) | WO2016109649A1 (https=) |
| ZA (1) | ZA201704327B (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN119876138A (zh) | 2014-11-14 | 2025-04-25 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| CA3193811A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| WO2016109649A1 (en) | 2014-12-30 | 2016-07-07 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| CA3024449A1 (en) | 2016-05-18 | 2017-11-23 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| CA3024448C (en) | 2016-05-18 | 2025-09-09 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| CL2016003282A1 (es) * | 2016-12-21 | 2017-08-18 | Univ Chile | Virus aav/igf2, método de tratamiento genético y su uso en enfermedades relacionadas con mal plegamiento de proteínas tal como la enfermedad de huntington |
| WO2018132791A1 (en) * | 2017-01-16 | 2018-07-19 | The Regents Of The University Of California | Treatment of neurodegenerative conditions by disruption of rhes |
| JP2020518259A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. | ハンチントン病治療組成物および方法 |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20050255086A1 (en) * | 2002-08-05 | 2005-11-17 | Davidson Beverly L | Nucleic acid silencing of Huntington's Disease gene |
| US20050009112A1 (en) | 2003-03-07 | 2005-01-13 | Fred Hutchinson Cancer Research Center, Office Of Technology Transfer | Methods for identifying Rheb effectors as lead compounds for drug development for diabetes and diseases associated with abnormal cell growth |
| WO2006116716A2 (en) | 2005-04-27 | 2006-11-02 | University Of Florida | Materials and methods for enhanced degradation of mutant proteins associated with human disease |
| KR101286870B1 (ko) * | 2009-11-30 | 2013-07-16 | (주)아모레퍼시픽 | 황기 추출물을 포함하는 대사 촉진 조성물 |
| CN104039790B (zh) | 2011-10-28 | 2016-04-13 | 诺华股份有限公司 | 嘌呤衍生物及它们在治疗疾病中的应用 |
| EP2773382A4 (en) | 2011-11-01 | 2016-03-23 | Childrens Medical Center | SIMULTANEOUS ACTIVATION OF MTOR AND STAT3 PATHS TO PROMOTE NEURONAL SURVIVAL AND REGENERATION |
| CA2854926A1 (en) * | 2011-11-08 | 2013-05-16 | Intellikine, Llc | Treatment regimens using multiple pharmaceutical agents |
| WO2013116691A1 (en) | 2012-02-02 | 2013-08-08 | The Washington University | Methods for improving muscle strength |
| KR101626526B1 (ko) | 2014-10-30 | 2016-06-02 | 경북대학교 산학협력단 | hRheb를 유효성분으로 포함하는 허혈성 뇌질환의 예방 또는 치료용 조성물 |
| WO2016109649A1 (en) | 2014-12-30 | 2016-07-07 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
-
2015
- 2015-12-30 WO PCT/US2015/068034 patent/WO2016109649A1/en not_active Ceased
- 2015-12-30 US US15/540,746 patent/US11612641B2/en active Active
- 2015-12-30 CA CA2971687A patent/CA2971687C/en active Active
- 2015-12-30 BR BR112017013674A patent/BR112017013674A2/pt not_active Application Discontinuation
- 2015-12-30 CN CN201580077153.9A patent/CN107405414A/zh active Pending
- 2015-12-30 JP JP2017534822A patent/JP6782701B2/ja active Active
- 2015-12-30 AU AU2015374043A patent/AU2015374043B2/en active Active
- 2015-12-30 ES ES15876243T patent/ES2962439T3/es active Active
- 2015-12-30 SG SG11201704829QA patent/SG11201704829QA/en unknown
- 2015-12-30 EP EP15876243.5A patent/EP3240577B1/en active Active
- 2015-12-30 KR KR1020177020915A patent/KR102618947B1/ko active Active
-
2017
- 2017-06-26 ZA ZA2017/04327A patent/ZA201704327B/en unknown
Non-Patent Citations (3)
| Title |
|---|
| 2013. 43rd Annual Meeting of the Society-For-Neuroscience.(Poster #306.05)(2013.11.11.)* |
| Journal of Biological Chemistry. 2006. Vol.281, No.29, pp.19793-19797.* |
| Molecular Therapy. 2012. Vol.20, No.2, pp.275-286.* |
Also Published As
| Publication number | Publication date |
|---|---|
| US11612641B2 (en) | 2023-03-28 |
| EP3240577A1 (en) | 2017-11-08 |
| EP3240577A4 (en) | 2018-07-25 |
| CN107405414A (zh) | 2017-11-28 |
| HK1245127A1 (en) | 2018-08-24 |
| CA2971687A1 (en) | 2016-07-07 |
| US20180000907A1 (en) | 2018-01-04 |
| WO2016109649A1 (en) | 2016-07-07 |
| NZ733014A (en) | 2024-05-31 |
| ZA201704327B (en) | 2025-02-26 |
| JP6782701B2 (ja) | 2020-11-11 |
| CA2971687C (en) | 2024-05-28 |
| EP3240577B1 (en) | 2023-07-05 |
| JP2018502112A (ja) | 2018-01-25 |
| KR20170098931A (ko) | 2017-08-30 |
| ES2962439T3 (es) | 2024-03-19 |
| SG11201704829QA (en) | 2017-07-28 |
| BR112017013674A2 (pt) | 2018-02-06 |
| AU2015374043B2 (en) | 2021-05-13 |
| AU2015374043A1 (en) | 2017-07-13 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| KR102618947B1 (ko) | 뇌 질환을 치료하기 위한 방법 및 조성물 | |
| US20240226203A9 (en) | Compositions and methods of treating huntington's disease | |
| US20230295663A1 (en) | Compositions and methods of treating amyotrophic lateral sclerosis (als) | |
| US20220333131A1 (en) | Modulatory polynucleotides | |
| AU2018352236B2 (en) | Treatment of amyotrophic lateral sclerosis (ALS) | |
| US11155817B2 (en) | Therapeutic for treatment of diseases including the central nervous system | |
| JP2006521825A (ja) | rAAV導入を高めるための化合物および方法 | |
| US20240343768A1 (en) | Gene therapy for tuberous sclerosis | |
| CN112955169A (zh) | 用于肌萎缩性侧索硬化疗法中的胆固醇24-水解酶的表达载体 | |
| WO2020176732A1 (en) | TREATMENT OF PULMONARY FIBROSIS WITH SERCA2a GENE THERAPY | |
| EP3962514A1 (en) | Methods and compositions involving tert activating therapies | |
| KR20240150772A (ko) | 폴리q 질환 치료를 위한 치료 인자 | |
| HK1245127B (en) | Therapeutic agent that activates mtorc1 function for use in treating huntington's disease | |
| WO2025015328A2 (en) | Increasing nfe2l1 activity or expression as therapy for eye disorders | |
| JP2022531177A (ja) | 神経変性障害を治療するための方法 | |
| HK40052708B (zh) | 用於肌萎缩性侧索硬化疗法中的胆固醇24-羟化酶的表达载体 | |
| HK40052708A (zh) | 用於肌萎缩性侧索硬化疗法中的胆固醇24-羟化酶的表达载体 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PA0105 | International application |
Patent event date: 20170726 Patent event code: PA01051R01D Comment text: International Patent Application |
|
| PG1501 | Laying open of application | ||
| A201 | Request for examination | ||
| PA0201 | Request for examination |
Patent event code: PA02012R01D Patent event date: 20201224 Comment text: Request for Examination of Application |
|
| E902 | Notification of reason for refusal | ||
| PE0902 | Notice of grounds for rejection |
Comment text: Notification of reason for refusal Patent event date: 20230313 Patent event code: PE09021S01D |
|
| E701 | Decision to grant or registration of patent right | ||
| PE0701 | Decision of registration |
Patent event code: PE07011S01D Comment text: Decision to Grant Registration Patent event date: 20230925 |
|
| GRNT | Written decision to grant | ||
| PR0701 | Registration of establishment |
Comment text: Registration of Establishment Patent event date: 20231222 Patent event code: PR07011E01D |
|
| PR1002 | Payment of registration fee |
Payment date: 20231222 End annual number: 3 Start annual number: 1 |
|
| PG1601 | Publication of registration |