PH12022550282A1 - Viral particles for use in treating synucleinopathies such as parkinson`s diseases by gene therapy - Google Patents
Viral particles for use in treating synucleinopathies such as parkinson`s diseases by gene therapyInfo
- Publication number
- PH12022550282A1 PH12022550282A1 PH1/2022/550282A PH12022550282A PH12022550282A1 PH 12022550282 A1 PH12022550282 A1 PH 12022550282A1 PH 12022550282 A PH12022550282 A PH 12022550282A PH 12022550282 A1 PH12022550282 A1 PH 12022550282A1
- Authority
- PH
- Philippines
- Prior art keywords
- gene therapy
- diseases
- viral particles
- parkinson
- treating
- Prior art date
Links
- 239000002245 particle Substances 0.000 title abstract 4
- 230000003612 virological effect Effects 0.000 title abstract 4
- 208000032859 Synucleinopathies Diseases 0.000 title abstract 3
- 238000001415 gene therapy Methods 0.000 title abstract 3
- 201000010099 disease Diseases 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title 1
- 102000004547 Glucosylceramidase Human genes 0.000 abstract 1
- 108010017544 Glucosylceramidase Proteins 0.000 abstract 1
- 208000018737 Parkinson disease Diseases 0.000 abstract 1
- 108700019146 Transgenes Proteins 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
Classifications
-
- 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)
- C12Y302/01062—Glycosylceramidase (3.2.1.62)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- 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)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K49/00—Preparations for testing in vivo
- A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
-
- 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)
- C12N9/2405—Glucanases
-
- 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
-
- 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
-
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- 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
- C12N2750/14145—Special targeting system for viral vectors
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)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Psychology (AREA)
- Toxicology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Rheumatology (AREA)
- Endocrinology (AREA)
- Urology & Nephrology (AREA)
- Diabetes (AREA)
- Immunology (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP19382706 | 2019-08-12 | ||
| PCT/EP2020/072087 WO2021028299A1 (en) | 2019-08-12 | 2020-08-06 | Viral particles for use in treating synucleinopathies such as parkinson's diseases by gene therapy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PH12022550282A1 true PH12022550282A1 (en) | 2022-11-21 |
Family
ID=67659024
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PH1/2022/550282A PH12022550282A1 (en) | 2019-08-12 | 2020-08-06 | Viral particles for use in treating synucleinopathies such as parkinson`s diseases by gene therapy |
Country Status (18)
| Country | Link |
|---|---|
| US (1) | US20220298528A1 (https=) |
| EP (1) | EP4013437A1 (https=) |
| JP (1) | JP2023500011A (https=) |
| KR (1) | KR20220099944A (https=) |
| CN (1) | CN114786694A (https=) |
| AR (1) | AR119609A1 (https=) |
| AU (1) | AU2020328827A1 (https=) |
| BR (1) | BR112022002615A2 (https=) |
| CA (1) | CA3149844A1 (https=) |
| CL (1) | CL2022000292A1 (https=) |
| CO (1) | CO2022001192A2 (https=) |
| EC (1) | ECSP22008949A (https=) |
| IL (1) | IL290357A (https=) |
| MX (1) | MX2022001676A (https=) |
| PE (1) | PE20220601A1 (https=) |
| PH (1) | PH12022550282A1 (https=) |
| TW (1) | TW202120687A (https=) |
| WO (1) | WO2021028299A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112021015312A2 (pt) | 2019-02-04 | 2021-11-09 | Freeline Therapeutics Ltd | Polinucleotídeos, partícula viral e composição |
| EP4192961A2 (en) * | 2020-08-06 | 2023-06-14 | Fundacion para la Investigacion Medica Aplicada | Viral particles for use in treating tauopathies such as alzheimer's diseases by gene therapy |
| WO2023202637A1 (en) * | 2022-04-19 | 2023-10-26 | Shanghai Vitalgen Biopharma Co., Ltd. | Recombinant aav vectors for treating neurodegenerative disorders |
| WO2024074142A1 (en) * | 2022-10-08 | 2024-04-11 | Lingyi Biotech Co., Ltd. | Polynucleotides for the treatment of disease associated with gcase deficiency |
| WO2025063407A1 (ko) * | 2023-09-22 | 2025-03-27 | 주식회사 스탠드업테라퓨티스 | 신규한 파킨슨 병 치료제 |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3292206B8 (en) * | 2015-05-07 | 2022-02-09 | Takeda Pharmaceutical Company Limited | Glucocerebrosidase gene therapy for parkinson's disease |
| EP3692075A4 (en) * | 2017-10-03 | 2022-02-09 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISEASES |
| WO2019068854A1 (en) * | 2017-10-06 | 2019-04-11 | Ospedale San Raffaele S.R.L. | GENE THERAPY OF NEURODEGENERATIVE DISEASES USING VAA VECTORS |
-
2020
- 2020-08-06 WO PCT/EP2020/072087 patent/WO2021028299A1/en not_active Ceased
- 2020-08-06 TW TW109126665A patent/TW202120687A/zh unknown
- 2020-08-06 US US17/634,112 patent/US20220298528A1/en not_active Abandoned
- 2020-08-06 BR BR112022002615A patent/BR112022002615A2/pt not_active IP Right Cessation
- 2020-08-06 AR ARP200102236A patent/AR119609A1/es not_active Application Discontinuation
- 2020-08-06 AU AU2020328827A patent/AU2020328827A1/en not_active Abandoned
- 2020-08-06 CN CN202080064577.2A patent/CN114786694A/zh active Pending
- 2020-08-06 JP JP2022507566A patent/JP2023500011A/ja not_active Withdrawn
- 2020-08-06 CA CA3149844A patent/CA3149844A1/en active Pending
- 2020-08-06 EP EP20750277.4A patent/EP4013437A1/en not_active Withdrawn
- 2020-08-06 KR KR1020227003990A patent/KR20220099944A/ko active Pending
- 2020-08-06 PE PE2022000219A patent/PE20220601A1/es unknown
- 2020-08-06 MX MX2022001676A patent/MX2022001676A/es unknown
- 2020-08-06 PH PH1/2022/550282A patent/PH12022550282A1/en unknown
-
2022
- 2022-02-04 CL CL2022000292A patent/CL2022000292A1/es unknown
- 2022-02-04 CO CONC2022/0001192A patent/CO2022001192A2/es unknown
- 2022-02-04 EC ECSENADI20228949A patent/ECSP22008949A/es unknown
- 2022-02-06 IL IL290357A patent/IL290357A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CN114786694A (zh) | 2022-07-22 |
| PE20220601A1 (es) | 2022-04-25 |
| AR119609A1 (es) | 2021-12-29 |
| ECSP22008949A (es) | 2022-03-31 |
| EP4013437A1 (en) | 2022-06-22 |
| TW202120687A (zh) | 2021-06-01 |
| BR112022002615A2 (pt) | 2022-05-03 |
| JP2023500011A (ja) | 2023-01-04 |
| AU2020328827A1 (en) | 2022-03-03 |
| CL2022000292A1 (es) | 2022-10-21 |
| CO2022001192A2 (es) | 2022-07-19 |
| WO2021028299A1 (en) | 2021-02-18 |
| MX2022001676A (es) | 2022-05-03 |
| CA3149844A1 (en) | 2021-02-18 |
| US20220298528A1 (en) | 2022-09-22 |
| IL290357A (en) | 2022-04-01 |
| KR20220099944A (ko) | 2022-07-14 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| PH12022550282A1 (en) | Viral particles for use in treating synucleinopathies such as parkinson`s diseases by gene therapy | |
| MX2020010694A (es) | Particulas de raav que codifican mir-708 y usos del mismo. | |
| WO2017191274A3 (en) | Rna encoding a therapeutic protein | |
| MX2022012490A (es) | Vectores de aav para la terapia genica de la retina y el snc. | |
| MX2017005834A (es) | Polinucleotidos aad para el tratamiento de la enfermedad de parkinson. | |
| PH12017501436A1 (en) | Enhanced delivery of viral particles to the striatum and cortex | |
| MX2022003859A (es) | Compuestos para usarse en el tratamiento o prevencion de retinitis pigmentosa. | |
| MY161415A (en) | A homeopathic formulation | |
| MX2018004755A (es) | Entrega de polinucleotidos dirigidos al sistema nervioso central. | |
| IN2014CN00688A (https=) | ||
| GB201103062D0 (en) | Method | |
| WO2021084277A3 (en) | Transcription regulatory elements | |
| MX2010008942A (es) | Paramyxovirus eficaz como antitumoral. | |
| MX2020013865A (es) | Tratamiento genico combinatorio. | |
| MX2023000968A (es) | Particulas virales para usarse en el tratamiento contra tauopatias tales como enfermedad de alzheimer mediante terapia genica. | |
| WO2013012771A3 (en) | Clinical applications of a recombinant human endostatin adenovirus (e10a) injection | |
| NZ704280A (en) | Baclofen and acamprosate based therapy of macular degeneration disorders | |
| HUE046546T2 (hu) | Kis interferáló RNS (siRNS) CLCN7 (ADO2 CLCN7-függõ) génmutáció által okozott 2-es típusú (ADO2) autoszómás domináns oszteopetrózis kezelésére | |
| TN2016000460A1 (en) | Aav vectors for retinal and cns gene therapy. | |
| HK1238679A1 (en) | Methods and compositions for nuclease-mediated genome engineering and correction in hematopoietic stem cells |