RU2018104098A - Системный синтез и регуляция l-дофа - Google Patents

Системный синтез и регуляция l-дофа Download PDF

Info

Publication number
RU2018104098A
RU2018104098A RU2018104098A RU2018104098A RU2018104098A RU 2018104098 A RU2018104098 A RU 2018104098A RU 2018104098 A RU2018104098 A RU 2018104098A RU 2018104098 A RU2018104098 A RU 2018104098A RU 2018104098 A RU2018104098 A RU 2018104098A
Authority
RU
Russia
Prior art keywords
seq
expression system
group
host cell
promoter
Prior art date
Application number
RU2018104098A
Other languages
English (en)
Russian (ru)
Other versions
RU2018104098A3 (cg-RX-API-DMAC10.html
Inventor
Майкл МАКДОНАЛЬД
Original Assignee
Майодопа Лимитед
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Майодопа Лимитед filed Critical Майодопа Лимитед
Publication of RU2018104098A publication Critical patent/RU2018104098A/ru
Publication of RU2018104098A3 publication Critical patent/RU2018104098A3/ru

Links

Classifications

    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • 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)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • 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
    • C12N9/88—Lyases (4.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
    • C12Y114/16002—Tyrosine 3-monooxygenase (1.14.16.2)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04016—GTP cyclohydrolase I (3.5.4.16)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y402/00—Carbon-oxygen lyases (4.2)
    • C12Y402/03—Carbon-oxygen lyases (4.2) acting on phosphates (4.2.3)
    • C12Y402/03012—6-Pyruvoyltetrahydropterin synthase (4.2.3.12)
    • 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
    • C12N2750/14171—Demonstrated in vivo effect
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/13—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with NADH or NADPH as one donor, and incorporation of one atom of oxygen (1.14.13)
    • C12Y114/13162—2,5-Diketocamphane 1,2-monooxygenase (1.14.13.162), i.e. camphor 1,2-monooxygenase

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Dermatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Peptides Or Proteins (AREA)
RU2018104098A 2015-08-03 2016-08-01 Системный синтез и регуляция l-дофа RU2018104098A (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562200151P 2015-08-03 2015-08-03
US62/200,151 2015-08-03
PCT/EP2016/068315 WO2017021359A1 (en) 2015-08-03 2016-08-01 Systemic synthesis and regulation of l-dopa

Publications (2)

Publication Number Publication Date
RU2018104098A true RU2018104098A (ru) 2019-09-06
RU2018104098A3 RU2018104098A3 (cg-RX-API-DMAC10.html) 2020-08-10

Family

ID=56682101

Family Applications (1)

Application Number Title Priority Date Filing Date
RU2018104098A RU2018104098A (ru) 2015-08-03 2016-08-01 Системный синтез и регуляция l-дофа

Country Status (8)

Country Link
US (1) US20190032079A1 (cg-RX-API-DMAC10.html)
EP (1) EP3331570A1 (cg-RX-API-DMAC10.html)
JP (1) JP2018522595A (cg-RX-API-DMAC10.html)
KR (1) KR20180034467A (cg-RX-API-DMAC10.html)
CN (1) CN108136048A (cg-RX-API-DMAC10.html)
CA (1) CA2992511A1 (cg-RX-API-DMAC10.html)
RU (1) RU2018104098A (cg-RX-API-DMAC10.html)
WO (1) WO2017021359A1 (cg-RX-API-DMAC10.html)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2839698C1 (ru) * 2019-10-25 2025-05-12 Одентес Терапьютикс, Инк. Композиции и способы лечения нарушений накопления гликогена

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
US20210093735A1 (en) * 2018-04-26 2021-04-01 The University Of North Carolina At Chapel Hill Methods and compositions for treatment of hemophilia
US12313635B2 (en) * 2018-07-27 2025-05-27 The Regents Of The University Of California Biomarker for thoracic aortic aneurysm
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
KR20210151785A (ko) * 2019-03-13 2021-12-14 제너레이션 바이오 컴퍼니 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도
KR20210148273A (ko) * 2019-04-08 2021-12-07 제네똥 근육 발현을 위한 하이브리드 프로모터
GB2587319A (en) * 2019-08-12 2021-03-31 Healing Genes Llc Genetic construct
EP4081643A1 (en) * 2019-12-24 2022-11-02 Asklepios Biopharmaceutical, Inc. Regulatory nucleic acid sequences
EP4107259A1 (en) * 2020-02-18 2022-12-28 Vrije Universiteit Brussel Novel combination of nucleic acid regulatory elements and methods and uses thereof
CN115515646A (zh) 2020-03-04 2022-12-23 波赛达治疗公司 用于治疗代谢性肝病的组合物和方法
WO2021222653A1 (en) * 2020-04-29 2021-11-04 Saliogen Therapeutics, Inc. Compositions and methods for treatment of familial hypercholesterolemia and elevated low-density lipoprotein cholesterol
CA3187002A1 (en) * 2020-07-15 2022-01-20 Danmarks Tekniske Universitet Therapeutic microbes
GB202107473D0 (en) * 2021-05-26 2021-07-07 Univ Denmark Tech Dtu Therapeutic microbes
IL301255A (en) * 2020-10-09 2023-05-01 UCB Biopharma SRL Nucleic acid structures, viral vectors and viral particles
GB2601752A (en) * 2020-12-08 2022-06-15 Maavrx Ltd Expression vector
WO2023012313A1 (en) * 2021-08-04 2023-02-09 Genethon Hybrid promoters for gene expression in muscles and in the cns
WO2023049874A1 (en) * 2021-09-24 2023-03-30 Duke University Compositions for and methods of treating and/or preventing glutaric aciduria type-i
CN117106825B (zh) * 2023-08-28 2024-11-01 康霖生物科技（杭州）有限公司 用于治疗帕金森病的基因治疗载体及其用途

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
DE3681787D1 (de) 1985-07-05 1991-11-07 Whitehead Biomedical Inst Expression von fremdem genetischem material in epithelzellen.
HK1008049A1 (en) 1987-09-11 1999-04-30 Whitehead Institute For Biomedical Research Transduced fibroblasts and use therefor
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5677158A (en) 1995-06-07 1997-10-14 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6451306B1 (en) 1998-04-15 2002-09-17 The Regents Of The University Of California Methods for therapy of neurodegenerative disease of the brain
CA2329259C (en) 1998-05-27 2003-08-05 Avigen, Inc. Convection-enhanced delivery of aav vectors
GB0024550D0 (cg-RX-API-DMAC10.html) * 2000-10-06 2000-11-22 Oxford Biomedica Ltd
KR100456062B1 (ko) * 2001-06-18 2004-11-08 박영식 재조합 대장균에 의한 테리딘 화합물의 제조방법
AU2010316982B2 (en) * 2009-11-09 2013-09-12 Genepod Therapeutics Ab Novel viral vector construct for neuron specific continuous DOPA synthesis in vivo
GB201118636D0 (en) * 2011-10-28 2011-12-07 Oxford Biomedica Ltd Nucleotide sequence

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2839698C1 (ru) * 2019-10-25 2025-05-12 Одентес Терапьютикс, Инк. Композиции и способы лечения нарушений накопления гликогена

Also Published As

Publication number Publication date
EP3331570A1 (en) 2018-06-13
JP2018522595A (ja) 2018-08-16
CN108136048A (zh) 2018-06-08
RU2018104098A3 (cg-RX-API-DMAC10.html) 2020-08-10
CA2992511A1 (en) 2017-02-09
KR20180034467A (ko) 2018-04-04
WO2017021359A1 (en) 2017-02-09
US20190032079A1 (en) 2019-01-31

Similar Documents

Publication Publication Date Title
US20250011783A1 (en) Gene therapy for neurodegenerative disorders
CN116096734A (zh) Aav衣壳的向性的重定向
JP6878433B2 (ja) ジストロフィンエクソン５３スキッピングのための効果的な遺伝子治療ツール
KR20220004681A (ko) 신규 aav 캡시드 및 이를 포함하는 조성물
WO2011154427A1 (en) MODIFIED snRNAs FOR USE IN THERAPY
JP2019513399A5 (cg-RX-API-DMAC10.html)
FI3628334T3 (fi) Geenihoito verkkokalvon pigmenttirappeuma
IL300410A (en) Novel AAV capsids and compositions containing them
KR20240164824A (ko) 조작된 소형 핵 rna(snrna)를 포함하는 조성물 및 방법
JP2020054367A (ja) 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用
JP2022541070A (ja) 調節可能な発現系
CA2992511A1 (en) Systemic synthesis and regulation of l-dopa
US20230233710A1 (en) Regulatory nucleic acid sequences
US20240209354A1 (en) MULTIPLEX CRISPR/Cas9-MEDIATED TARGET GENE ACTIVATION SYSTEM
CN120641136A (zh) 用于额颞叶痴呆的基因疗法
JP2025513887A (ja) 肝臓における遺伝子発現を脱標的化するための要素
EP4634206A2 (en) Recombinant adeno-associated virus gene therapy vectors with reduced liver tropism and enhanced transduction of cardiac cells for the therapy of heart diseases and diseases associated with heart dysfunction
JP2024524270A (ja) 調節性核酸配列
CN120752343A (zh) 调控核酸序列
HK40085441A (en) Redirection of tropism of aav capsids
WO2026011131A1 (en) Tric-derived scaffolds for circular rna biogenesis
JP2026507523A (ja) 遺伝子療法のためのｂｃｌ２関連アタノゲン３（ｂａｇ３）をコードする核酸

Legal Events

Date Code Title Description
FA92 Acknowledgement of application withdrawn (lack of supplementary materials submitted)

Effective date: 20201210