CN115176002A - 用于神经保护的方法和组合物 - Google Patents

用于神经保护的方法和组合物 Download PDF

Info

Publication number
CN115176002A
CN115176002A CN202080082124.2A CN202080082124A CN115176002A CN 115176002 A CN115176002 A CN 115176002A CN 202080082124 A CN202080082124 A CN 202080082124A CN 115176002 A CN115176002 A CN 115176002A
Authority
CN
China
Prior art keywords
disease
disorder
antisense oligonucleotide
injury
subject
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
CN202080082124.2A
Other languages
English (en)
Chinese (zh)
Inventor
L·莱文塔尔
T·恩伯
R·E·克劳斯
R·德夫拉
R·O·休斯
T·波萨纳奇
S·阿拉瓦尔
M·A·帕西尼
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.)
Disarm Therapeutics Inc
Original Assignee
Disarm Therapeutics Inc
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 Disarm Therapeutics Inc filed Critical Disarm Therapeutics Inc
Publication of CN115176002A publication Critical patent/CN115176002A/zh
Pending legal-status Critical Current

Links

Images

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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • 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
    • 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
    • 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
    • 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)
    • C12Y302/02—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2) hydrolysing N-glycosyl compounds (3.2.2)
    • C12Y302/02006—NAD(P)+ nucleosidase (3.2.2.6)
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/316—Phosphonothioates
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Steroid Compounds (AREA)
CN202080082124.2A 2019-11-26 2020-11-25 用于神经保护的方法和组合物 Pending CN115176002A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962940437P 2019-11-26 2019-11-26
US62/940437 2019-11-26
PCT/US2020/062289 WO2021108602A1 (en) 2019-11-26 2020-11-25 Methods and compositions for neuroprotection

Publications (1)

Publication Number Publication Date
CN115176002A true CN115176002A (zh) 2022-10-11

Family

ID=74046136

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202080082124.2A Pending CN115176002A (zh) 2019-11-26 2020-11-25 用于神经保护的方法和组合物

Country Status (11)

Country Link
US (1) US12595481B2 (https=)
EP (1) EP4065714A1 (https=)
JP (2) JP7528212B2 (https=)
KR (1) KR20220119366A (https=)
CN (1) CN115176002A (https=)
AU (1) AU2020391206A1 (https=)
BR (1) BR112022009772A2 (https=)
IL (1) IL293123A (https=)
MX (1) MX2022006146A (https=)
TW (1) TW202134436A (https=)
WO (1) WO2021108602A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025261431A1 (zh) * 2024-06-19 2025-12-26 上海拓界生物医药科技有限公司 靶向SARM1的RNAi剂及其医药用途

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4065714A1 (en) * 2019-11-26 2022-10-05 Disarm Therapeutics, Inc. Methods and compositions for neuroprotection
PE20242222A1 (es) * 2022-03-14 2024-11-19 Lilly Co Eli Agentes de interferencia de arn de sarm1
CN116059367B (zh) * 2022-07-15 2025-02-18 浙江乘时佳生物科技有限公司 一种治疗阿尔茨海默症的sarm1抑制剂
JP2025523905A (ja) * 2022-07-21 2025-07-25 アミリックス ファーマシューティカルズ，インコーポレイティッド オリゴヌクレオチド組成物及びその方法
TW202547525A (zh) 2024-02-02 2025-12-16 美商美國禮來大藥廠 SARM1 RNAi藥劑
GB202408068D0 (en) 2024-06-06 2024-07-24 Univ London Treatment of cancer of the central nervous system

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040096830A1 (en) 2002-11-16 2004-05-20 Isis Pharmaceuticals Inc. Modulation of protein kinase D2 expression
US7250496B2 (en) * 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
US20050244851A1 (en) * 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
CA2566519C (en) * 2004-05-14 2020-04-21 Rosetta Genomics Ltd. Micrornas and uses thereof
US7901882B2 (en) * 2006-03-31 2011-03-08 Affymetrix, Inc. Analysis of methylation using nucleic acid arrays
JP2013003911A (ja) * 2011-06-17 2013-01-07 Sony Corp 電子機器、電子機器の制御方法およびプログラム
US20120328629A1 (en) 2011-06-24 2012-12-27 University Of Miami Therapeutic Applications Targeting SARM1
EP3294342A4 (en) 2015-05-08 2018-11-07 President and Fellows of Harvard College Universal donor stem cells and related methods
JP7198489B2 (ja) * 2016-03-23 2023-01-04 国立大学法人 岡山大学 リン酸化ｓａｒｍ１、抗体、ｓａｒｍ１リン酸化阻害剤、神経変性疾患の予防又は治療薬、スクリーニング方法、ｓａｒｍ１改変体及び使用
AU2017292173B2 (en) 2016-07-06 2022-01-13 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
WO2018057989A1 (en) 2016-09-24 2018-03-29 Washington University INHIBITORS OF SARM1 NADase ACTIVITY AND USES THEREOF
EP3585897A1 (en) * 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of dystrophic epidermolysis bullosa (deb) and other collagen type vii alpha 1 chain (col7a1) gene related conditions or disorders
US12576130B2 (en) * 2017-10-18 2026-03-17 Washington University Dominant negative SARM1 molecules comprising a substitution at position 189, 190, 193, 194, 570 and/or 685 of SARM1
US12448374B2 (en) * 2018-06-07 2025-10-21 Disarm Therapeutics, Inc. Inhibitors of SARM1
JP7481329B2 (ja) 2018-06-07 2024-05-10 ディスアーム セラピューティクス， インコーポレイテッド Ｓａｒｍ１阻害剤
ES3028093T3 (en) 2018-06-07 2025-06-18 Disarm Therapeutics Inc Inhibitors of sarm1
EP4065714A1 (en) * 2019-11-26 2022-10-05 Disarm Therapeutics, Inc. Methods and compositions for neuroprotection

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025261431A1 (zh) * 2024-06-19 2025-12-26 上海拓界生物医药科技有限公司 靶向SARM1的RNAi剂及其医药用途

Also Published As

Publication number Publication date
KR20220119366A (ko) 2022-08-29
JP2024112834A (ja) 2024-08-21
MX2022006146A (es) 2022-10-18
US20230242909A1 (en) 2023-08-03
US12595481B2 (en) 2026-04-07
JP7528212B2 (ja) 2024-08-05
TW202134436A (zh) 2021-09-16
JP7796797B2 (ja) 2026-01-09
JP2023503994A (ja) 2023-02-01
WO2021108602A1 (en) 2021-06-03
IL293123A (en) 2022-07-01
AU2020391206A1 (en) 2022-05-19
CA3159374A1 (en) 2021-06-03
BR112022009772A2 (pt) 2022-08-16
EP4065714A1 (en) 2022-10-05

Similar Documents

Publication Publication Date Title
JP7796797B2 (ja) 神経保護のための方法および組成物
JP7354342B2 (ja) タウ発現を調節するための組成物
US10159670B2 (en) Methods of diagnosing and treating motor neuron diseases and other cellular stress-related diseases
CN106983768B (zh) 用于在对象中调节smn2剪接的组合物和方法
EP3394259B1 (en) Compositions and methods for decreasing tau expression
JP2022519019A (ja) オリゴヌクレオチド組成物及びその方法
US20230340477A1 (en) Uses for prevention or treatment of brain diseases using microrna
US20180264027A1 (en) Method for treating a degenerative neurological disorders comprising administering asm inhibitor
TW201641691A (zh) Ｔａｕ反義寡聚物及其用途
JP2024504266A (ja) リジン分解関連障害の治療
JP7463621B2 (ja) ミトコンドリアアミドキシム還元成分１（ｍａｒｃ１）発現を阻害するための組成物および方法
US11242529B2 (en) Method for treating a neurological disorder comprising administering ASM inhibitors
CA3159374C (en) Methods and compositions for neuroprotection
KR102508432B1 (ko) eIF4E 저해제를 포함하는 eIF4E 활성증가와 관련된 상태의 진단 또는 치료용 조성물
KR102897439B1 (ko) 타겟 유전자 발현을 조절할 수 있는 핵산 분자 및 이의 용도
TW201241179A (en) MiRNAs in joint disease
HK1254822B (en) Compositions and methods for decreasing tau expression

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