MX2023000815A - Composiciones y metodos para el tratamiento de trastornos neurologicos relacionados con la deficiencia de glucosilceramidasa beta. - Google Patents
Composiciones y metodos para el tratamiento de trastornos neurologicos relacionados con la deficiencia de glucosilceramidasa beta.Info
- Publication number
- MX2023000815A MX2023000815A MX2023000815A MX2023000815A MX2023000815A MX 2023000815 A MX2023000815 A MX 2023000815A MX 2023000815 A MX2023000815 A MX 2023000815A MX 2023000815 A MX2023000815 A MX 2023000815A MX 2023000815 A MX2023000815 A MX 2023000815A
- Authority
- MX
- Mexico
- Prior art keywords
- compositions
- methods
- treatment
- neurological disorders
- disorders related
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 4
- 238000000034 method Methods 0.000 title abstract 3
- 230000007812 deficiency Effects 0.000 title abstract 2
- 101000997662 Homo sapiens Lysosomal acid glucosylceramidase Proteins 0.000 title 1
- 102100033342 Lysosomal acid glucosylceramidase Human genes 0.000 title 1
- 208000012902 Nervous system disease Diseases 0.000 title 1
- 208000025966 Neurological disease Diseases 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 abstract 3
- 102000004169 proteins and genes Human genes 0.000 abstract 3
- 101150028412 GBA gene Proteins 0.000 abstract 1
- 208000018737 Parkinson disease Diseases 0.000 abstract 1
- 230000003247 decreasing effect Effects 0.000 abstract 1
- 230000002708 enhancing effect Effects 0.000 abstract 1
- 238000000338 in vitro Methods 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
- 239000002245 particle Substances 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
- 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)
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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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/12—Viral antigens
- A61K39/235—Adenoviridae
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
-
- 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
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- 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)
- C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
-
- 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)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Virology (AREA)
- Psychiatry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Hospice & Palliative Care (AREA)
- Plant Pathology (AREA)
- Psychology (AREA)
- Mycology (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063057265P | 2020-07-27 | 2020-07-27 | |
| PCT/US2021/043216 WO2022026409A1 (en) | 2020-07-27 | 2021-07-26 | Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2023000815A true MX2023000815A (es) | 2023-04-11 |
Family
ID=77338944
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2023000815A MX2023000815A (es) | 2020-07-27 | 2021-07-26 | Composiciones y metodos para el tratamiento de trastornos neurologicos relacionados con la deficiencia de glucosilceramidasa beta. |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20230227802A1 (https=) |
| EP (1) | EP4189095A1 (https=) |
| JP (1) | JP2023536091A (https=) |
| KR (1) | KR20230093241A (https=) |
| CN (1) | CN117120619A (https=) |
| AU (1) | AU2021315876A1 (https=) |
| BR (1) | BR112023001456A2 (https=) |
| CA (1) | CA3190309A1 (https=) |
| IL (1) | IL299928A (https=) |
| MX (1) | MX2023000815A (https=) |
| TW (1) | TW202221125A (https=) |
| WO (1) | WO2022026409A1 (https=) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20250049955A1 (en) | 2021-11-17 | 2025-02-13 | Voyager Therapeutics, Inc. | Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
| WO2023215851A2 (en) * | 2022-05-06 | 2023-11-09 | Apic Bio, Inc. | Plasmid optimized for packaging of aav vectors |
| AU2023427408A1 (en) * | 2023-02-02 | 2025-09-04 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
| WO2024226761A2 (en) * | 2023-04-26 | 2024-10-31 | Voyager Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
| WO2024226790A1 (en) * | 2023-04-26 | 2024-10-31 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| WO2025039622A1 (zh) * | 2023-08-23 | 2025-02-27 | 上海金珂博生物技术有限公司 | 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途 |
| CN117264912A (zh) * | 2023-09-25 | 2023-12-22 | 中国科学院深圳先进技术研究院 | 一种重组腺相关病毒颗粒、重组腺相关病毒载体系统及其应用 |
| CN118064436B (zh) * | 2024-04-18 | 2024-07-26 | 上海凌医生物科技有限公司 | 一种增强基因表达的嵌合启动子 |
| CN121518479B (zh) * | 2026-01-16 | 2026-04-24 | 凌意(杭州)生物科技有限公司 | 基因编辑复合型突变小鼠模型的构建方法 |
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| KR102423442B1 (ko) | 2015-12-11 | 2022-07-20 | 캘리포니아 인스티튜트 오브 테크놀로지 | 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드 |
| BR112020006661A2 (pt) * | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| AU2018346104B2 (en) | 2017-10-03 | 2023-07-27 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| WO2019136484A1 (en) | 2018-01-08 | 2019-07-11 | Prevail Therapeutics, Inc. | Pet imaging ligands for in vivo detection of gba1 |
| JP2021517168A (ja) * | 2018-03-09 | 2021-07-15 | アブロバイオ，インコーポレーテッド | パーキンソン病を処置するための組成物及び方法 |
| BR112020021962A2 (pt) | 2018-04-30 | 2021-01-26 | Amicus Therapeutics, Inc. | construtos para terapia gênica e métodos de uso |
| US20210214749A1 (en) * | 2018-05-16 | 2021-07-15 | Voyager Therapeutics, Inc. | Directed evolution |
| WO2020132455A1 (en) | 2018-12-21 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
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| AU2021315876A1 (en) | 2023-02-23 |
| CN117120619A (zh) | 2023-11-24 |
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