CN116457466A - 用于抑制α-突触核蛋白聚集的组合物和方法 - Google Patents

用于抑制α-突触核蛋白聚集的组合物和方法 Download PDF

Info

Publication number
CN116457466A
CN116457466A CN202180070652.0A CN202180070652A CN116457466A CN 116457466 A CN116457466 A CN 116457466A CN 202180070652 A CN202180070652 A CN 202180070652A CN 116457466 A CN116457466 A CN 116457466A
Authority
CN
China
Prior art keywords
vector
aav
promoter
composition
seq
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.)
Pending
Application number
CN202180070652.0A
Other languages
English (en)
Chinese (zh)
Inventor
拉吉·梅赫拉
蒂莫西·惠特克
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Silos Therapeutics Ltd
Original Assignee
Silos Therapeutics Ltd
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 Silos Therapeutics Ltd filed Critical Silos Therapeutics Ltd
Publication of CN116457466A publication Critical patent/CN116457466A/zh
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/10—Animals modified by protein administration, for non-therapeutic purpose
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0356—Animal model for processes and diseases of the central nervous system, e.g. stress, learning, schizophrenia, pain, epilepsy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/60—Vector systems having a special element relevant for transcription from viruses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
CN202180070652.0A 2020-09-23 2021-09-23 用于抑制α-突触核蛋白聚集的组合物和方法 Pending CN116457466A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063082208P 2020-09-23 2020-09-23
US63/082,208 2020-09-23
PCT/US2021/051758 WO2022066911A1 (en) 2020-09-23 2021-09-23 Compositions and methods for inhibiting alpha-synuclein aggregation

Publications (1)

Publication Number Publication Date
CN116457466A true CN116457466A (zh) 2023-07-18

Family

ID=80845817

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180070652.0A Pending CN116457466A (zh) 2020-09-23 2021-09-23 用于抑制α-突触核蛋白聚集的组合物和方法

Country Status (7)

Country Link
US (1) US20240025953A1 (https=)
EP (1) EP4216978A4 (https=)
JP (1) JP2023542950A (https=)
CN (1) CN116457466A (https=)
AU (1) AU2021349261A1 (https=)
CA (1) CA3192936A1 (https=)
WO (1) WO2022066911A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116590342B (zh) * 2023-05-11 2026-04-03 普递瑞(上海)医药有限公司 一种aav载体及其应用
WO2024229777A1 (zh) * 2023-05-11 2024-11-14 深圳先进技术研究院 一种aav载体及其应用

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104902923A (zh) * 2012-11-05 2015-09-09 建新公司 用于治疗蛋白质病的组合物和方法
CN108699565A (zh) * 2015-12-11 2018-10-23 加州理工学院 用于定向腺相关病毒(aav)的靶向肽
WO2019068854A1 (en) * 2017-10-06 2019-04-11 Ospedale San Raffaele S.R.L. GENE THERAPY OF NEURODEGENERATIVE DISEASES USING VAA VECTORS
CN109641028A (zh) * 2016-06-29 2019-04-16 加利福尼亚大学董事会 α-突触核蛋白聚集的基于结构的肽抑制剂

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1680145A4 (en) * 2003-10-14 2007-05-23 Neurologix Res Inc METHODS AND COMPOSITIONS FOR THE TREATMENT OF NEUROLOGICAL DISEASES
EP2154969B1 (en) * 2007-05-16 2015-11-18 The Brigham and Women's Hospital, Inc. Treatment of synucleinopathies
US20170184612A1 (en) * 2014-04-09 2017-06-29 The Trustees Of Columbia University In The City Of New York Use of leukocytes and novel biomarkers in the diagnosis, confirmation, and treatment of a neurological disorder
WO2019036673A1 (en) * 2017-08-18 2019-02-21 The Medical College Of Wisconsin, Inc. THERAPY BASED ON SRCP1 FOR DISEASES ASSOCIATED WITH PROTEIN AGGREGATION
CA3096293A1 (en) * 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration
CA3118692A1 (en) * 2018-11-06 2020-05-14 Alsatech, Inc. Cell-based gene therapy for neurodegenerative diseases

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104902923A (zh) * 2012-11-05 2015-09-09 建新公司 用于治疗蛋白质病的组合物和方法
CN108699565A (zh) * 2015-12-11 2018-10-23 加州理工学院 用于定向腺相关病毒(aav)的靶向肽
CN109641028A (zh) * 2016-06-29 2019-04-16 加利福尼亚大学董事会 α-突触核蛋白聚集的基于结构的肽抑制剂
WO2019068854A1 (en) * 2017-10-06 2019-04-11 Ospedale San Raffaele S.R.L. GENE THERAPY OF NEURODEGENERATIVE DISEASES USING VAA VECTORS

Also Published As

Publication number Publication date
CA3192936A1 (en) 2022-03-31
EP4216978A4 (en) 2025-06-11
EP4216978A1 (en) 2023-08-02
AU2021349261A9 (en) 2024-07-04
JP2023542950A (ja) 2023-10-12
US20240025953A1 (en) 2024-01-25
WO2022066911A1 (en) 2022-03-31
AU2021349261A1 (en) 2023-06-01

Similar Documents

Publication Publication Date Title
US20240131093A1 (en) Compositions and methods of treating huntington's disease
TWI897844B (zh) 調節多核苷酸
AU2017267665B2 (en) Modulatory polynucleotides
JP2023052805A (ja) Ａａｖを送達するための組成物および方法
JP2022084940A (ja) β－サルコグリカン及びマイクロＲＮＡ－２９のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療
US20250122531A1 (en) Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
EP3826719A1 (en) Systems and methods for producing gene therapy formulations
WO2020150556A1 (en) Methods and systems for producing aav particles
JP2021500029A (ja) Ｎｔ−３遺伝子治療のための方法及び材料
JP2021528983A (ja) 肢帯型筋ジストロフィー２ａを治療するための組換えアデノ随伴ウイルス産物及び方法
KR20160010617A (ko) 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
JP2017536116A (ja) 中枢神経系を標的化したａａｖベクター
JP2016514152A (ja) アデノ随伴ウイルスベクターおよびその使用の方法
KR20220107243A (ko) Apoe 유전자 요법
CN110325219A (zh) 编码甲基-CpG结合蛋白2的重组腺相关病毒的鞘内递送
JP2023520374A (ja) 神経学的障害に対する活動依存性遺伝子療法
CN116457466A (zh) 用于抑制α-突触核蛋白聚集的组合物和方法
WO2021231575A1 (en) Immunosuppressive agents and viral delivery re-dosing methods for gene therapy
WO2009071679A1 (en) Novel aav vector and uses thereof
HK40104471A (zh) 用於目的基因的表达盒及其应用
WO2024097602A2 (en) Gene therapy for lemd2 cardiomyopathy
JP2022531177A (ja) 神経変性障害を治療するための方法
KR20250097939A (ko) 투여 경로
JP2024515623A (ja) 髄腔内送達によってピット・ホプキンス症候群を治療するためのメチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルス
EP2673002A2 (en) Methods of treating alzheimer's disease with apo a-1 milano

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination