MA47847A - Thérapie génique pour traiter la mucopolysaccharidose de type i - Google Patents
Thérapie génique pour traiter la mucopolysaccharidose de type iInfo
- Publication number
- MA47847A MA47847A MA047847A MA47847A MA47847A MA 47847 A MA47847 A MA 47847A MA 047847 A MA047847 A MA 047847A MA 47847 A MA47847 A MA 47847A MA 47847 A MA47847 A MA 47847A
- Authority
- MA
- Morocco
- Prior art keywords
- gene therapy
- mucopolysaccharidosis type
- treat mucopolysaccharidosis
- treat
- type
- Prior art date
Links
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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
-
- 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
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- 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
- 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)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01076—L-Iduronidase (3.2.1.76)
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—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)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Psychiatry (AREA)
- Physical Education & Sports Medicine (AREA)
- Obesity (AREA)
- Pulmonology (AREA)
- Hospice & Palliative Care (AREA)
- Diabetes (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Ophthalmology & Optometry (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662430795P | 2016-12-06 | 2016-12-06 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MA47847A true MA47847A (fr) | 2020-01-29 |
Family
ID=62491308
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MA047847A MA47847A (fr) | 2016-12-06 | 2017-12-06 | Thérapie génique pour traiter la mucopolysaccharidose de type i |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20220339296A1 (fr) |
| EP (1) | EP3551750A4 (fr) |
| JP (1) | JP2019536484A (fr) |
| KR (1) | KR20190089988A (fr) |
| CN (1) | CN110214182A (fr) |
| AU (1) | AU2017370662A1 (fr) |
| BR (1) | BR112019011635A2 (fr) |
| CA (1) | CA3046079A1 (fr) |
| IL (1) | IL267057A (fr) |
| MA (1) | MA47847A (fr) |
| MX (1) | MX2019006552A (fr) |
| RU (1) | RU2019120721A (fr) |
| WO (1) | WO2018106807A1 (fr) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN109641063B (zh) | 2016-04-20 | 2022-11-15 | 能源环境和技术研究中心O.A., M.P. | 用于增强pklr的基因表达的组合物和方法 |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| CN111163810B (zh) | 2017-10-16 | 2024-09-10 | 能源环境和技术研究中心O.A.,M.P. | 用于递送pklr以治疗丙酮酸激酶缺乏症的慢病毒载体 |
| SG11202008400SA (en) | 2018-04-11 | 2020-09-29 | Rocket Pharmaceuticals Ltd | Compositions and methods for stem cell transplant |
| JP7664829B2 (ja) * | 2018-07-30 | 2025-04-18 | セントロ デ インベスティガシオンス エネルジェチカス メディオアンビエンタゥス イェ テクノロジカス オー.エイ. エム.ピー. | 造血細胞の遺伝子改変のための方法 |
| CN115109790A (zh) * | 2021-03-23 | 2022-09-27 | 北京据德医药科技有限公司 | 一种重组a-L-艾杜糖醛酸普酶及其制备方法 |
| JP2024520797A (ja) * | 2021-06-08 | 2024-05-24 | タッチライト・アイピー・リミテッド | レンチウイルスベクター |
| WO2024201066A1 (fr) * | 2023-03-31 | 2024-10-03 | Dawn Therapeutics Limited | Nouveau vecteur |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2872170B1 (fr) * | 2004-06-25 | 2006-11-10 | Centre Nat Rech Scient Cnrse | Lentivirus non interactif et non replicatif, preparation et utilisations |
| US20110294114A1 (en) * | 2009-12-04 | 2011-12-01 | Cincinnati Children's Hospital Medical Center | Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells |
| TR201819015T4 (tr) * | 2011-06-10 | 2019-01-21 | Bluebird Bio Inc | Adrenolökodi̇strofi̇ ve adrenomi̇yelonöropati̇ i̇çi̇n gen terapi̇ vektörleri̇ |
| EP2855684A1 (fr) * | 2012-05-25 | 2015-04-08 | Commissariat à l'Énergie Atomique et aux Énergies Alternatives | Vecteur pour le silençage sélectif d'un gène dans des astrocytes |
| US20160122727A1 (en) * | 2013-06-13 | 2016-05-05 | Shire Human Genetic Therapies, Inc. | Messenger rna based viral production |
| US11326183B2 (en) * | 2016-02-12 | 2022-05-10 | Bluebird Bio, Inc. | VCN enhancer compositions and methods of using the same |
-
2017
- 2017-12-06 AU AU2017370662A patent/AU2017370662A1/en not_active Abandoned
- 2017-12-06 CN CN201780083351.5A patent/CN110214182A/zh active Pending
- 2017-12-06 MA MA047847A patent/MA47847A/fr unknown
- 2017-12-06 EP EP17877511.0A patent/EP3551750A4/fr not_active Withdrawn
- 2017-12-06 KR KR1020197019344A patent/KR20190089988A/ko not_active Ceased
- 2017-12-06 JP JP2019551256A patent/JP2019536484A/ja active Pending
- 2017-12-06 BR BR112019011635-4A patent/BR112019011635A2/pt not_active IP Right Cessation
- 2017-12-06 US US16/466,130 patent/US20220339296A1/en not_active Abandoned
- 2017-12-06 MX MX2019006552A patent/MX2019006552A/es unknown
- 2017-12-06 CA CA3046079A patent/CA3046079A1/fr not_active Abandoned
- 2017-12-06 WO PCT/US2017/064913 patent/WO2018106807A1/fr not_active Ceased
- 2017-12-06 RU RU2019120721A patent/RU2019120721A/ru not_active Application Discontinuation
-
2019
- 2019-06-03 IL IL267057A patent/IL267057A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CA3046079A1 (fr) | 2018-06-14 |
| RU2019120721A (ru) | 2021-01-11 |
| EP3551750A4 (fr) | 2020-07-22 |
| AU2017370662A1 (en) | 2019-06-27 |
| IL267057A (en) | 2019-08-29 |
| MX2019006552A (es) | 2019-10-15 |
| BR112019011635A2 (pt) | 2019-11-12 |
| EP3551750A1 (fr) | 2019-10-16 |
| US20220339296A1 (en) | 2022-10-27 |
| CN110214182A (zh) | 2019-09-06 |
| WO2018106807A1 (fr) | 2018-06-14 |
| JP2019536484A (ja) | 2019-12-19 |
| KR20190089988A (ko) | 2019-07-31 |
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