KR20220119366A - 신경보호를 위한 방법 및 조성물 - Google Patents

신경보호를 위한 방법 및 조성물 Download PDF

Info

Publication number
KR20220119366A
KR20220119366A KR1020227017020A KR20227017020A KR20220119366A KR 20220119366 A KR20220119366 A KR 20220119366A KR 1020227017020 A KR1020227017020 A KR 1020227017020A KR 20227017020 A KR20227017020 A KR 20227017020A KR 20220119366 A KR20220119366 A KR 20220119366A
Authority
KR
South Korea
Prior art keywords
disorder
disease
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.)
Withdrawn
Application number
KR1020227017020A
Other languages
English (en)
Korean (ko)
Inventor
리자 레벤탈
토마스 엥버
라울 에두아르도 크라우스
라제쉬 데브라즈
로버트 오웬 휴스
토드 보사낙
수드히르 아그라왈
마르코 에이. 파시니
Original Assignee
디스암 테라퓨틱스, 인크.
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 디스암 테라퓨틱스, 인크. filed Critical 디스암 테라퓨틱스, 인크.
Publication of KR20220119366A publication Critical patent/KR20220119366A/ko
Withdrawn legal-status Critical Current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)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Steroid Compounds (AREA)
KR1020227017020A 2019-11-26 2020-11-25 신경보호를 위한 방법 및 조성물 Withdrawn KR20220119366A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962940437P 2019-11-26 2019-11-26
US62/940,437 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
KR20220119366A true KR20220119366A (ko) 2022-08-29

Family

ID=74046136

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227017020A Withdrawn KR20220119366A (ko) 2019-11-26 2020-11-25 신경보호를 위한 방법 및 조성물

Country Status (11)

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

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7528212B2 (ja) * 2019-11-26 2024-08-05 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 神経保護のための方法および組成物
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
US20240102018A1 (en) * 2022-07-21 2024-03-28 Amylyx Pharmaceuticals, Inc. Oligonucleotide compositions and methods thereof
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
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

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
JP5697297B2 (ja) * 2004-05-14 2015-04-08 ロゼッタ ジノミクス リミテッド マイクロｎａｓおよびその使用
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
WO2012178022A2 (en) * 2011-06-24 2012-12-27 University Of Massachusetts Therapeutic applications targeting sarm1
CN107921148A (zh) 2015-05-08 2018-04-17 哈佛学院校长同事会 通用供体干细胞和相关方法
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
CN109843914B (zh) 2016-07-06 2024-03-15 沃泰克斯药物股份有限公司 用于治疗疼痛相关病症的材料和方法
JP7044789B2 (ja) 2016-09-24 2022-03-30 ワシントン・ユニバーシティ Ｓａｒｍ１ ｎａｄアーゼ活性の阻害剤およびその使用
WO2018154413A1 (en) * 2017-02-22 2018-08-30 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
EP3697401B1 (en) 2017-10-18 2025-12-03 Washington University Dominant negative sarm1 molecules as a therapeutic strategy for neurodegenerative diseases or disorders
CN112839648B (zh) 2018-06-07 2025-04-04 达萨玛治疗公司 Sarm1抑制剂
CN112839647B (zh) 2018-06-07 2025-09-23 达萨玛治疗公司 Sarm1抑制剂
CA3108669A1 (en) 2018-06-07 2019-12-12 Disarm Therapeutics, Inc. Inhibitors of sarm1
JP7528212B2 (ja) 2019-11-26 2024-08-05 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 神経保護のための方法および組成物

Also Published As

Publication number Publication date
CN115176002A (zh) 2022-10-11
US20230242909A1 (en) 2023-08-03
TW202134436A (zh) 2021-09-16
WO2021108602A1 (en) 2021-06-03
BR112022009772A2 (pt) 2022-08-16
CA3159374A1 (en) 2021-06-03
AU2020391206A1 (en) 2022-05-19
IL293123A (en) 2022-07-01
JP2023503994A (ja) 2023-02-01
JP2024112834A (ja) 2024-08-21
JP7796797B2 (ja) 2026-01-09
JP7528212B2 (ja) 2024-08-05
US12595481B2 (en) 2026-04-07
EP4065714A1 (en) 2022-10-05
MX2022006146A (es) 2022-10-18

Similar Documents

Publication Publication Date Title
KR20220119366A (ko) 신경보호를 위한 방법 및 조성물
KR101900770B1 (ko) 근육긴장성 이영양증-단백질 키나제(dmpk) 발현의 조절 방법
KR102640776B1 (ko) 디아실글리세롤 아실전이효소 2(dgat2) 조절제
US20210189342A1 (en) Compositions and methods for modulating monocyte and macrophage inflammatory phenotypes and immunotherapy uses thereof
KR20100081994A (ko) 섬유아세포 성장 인자 수용체 4 발현의 안티센스 조절
KR20160002977A (ko) 조성물 및 방법
KR20160147891A (ko) Pkk 발현을 조절하는 조성물 및 방법
CN107267517A (zh) 用于治疗、延缓和/或预防人类遗传性疾病如强直性肌营养不良症1型的新化合物
US9359607B2 (en) Methods of diagnosing and treating motor neuron diseases
KR20190025970A (ko) Htra1 발현을 조절하기 위한 안티센스 올리고뉴클레오티드
TW202221120A (zh) 用於治療代謝症候群之組成物及方法
KR20220035137A (ko) Ppm1a 억제제 및 그의 사용 방법
WO2024213890A1 (en) Oligonucleotide decoy
TW201238973A (en) MiRNAs in joint disease
CA3159374C (en) Methods and compositions for neuroprotection
TW201241179A (en) MiRNAs in joint disease
US20220002726A1 (en) Methods of safe administration of an irf5 antisense oligonucleotide
Class et al. Patent application title: ANTISENSE OLIGONUCLEOTIDES THAT TARGET A CRYPTIC SPLICE SITE IN USH1C AS A THERAPEUTIC FOR USHER SYNDROME Inventors: Michelle L. Hastings (Lake Bluff, IL, US) Assignees: Rosalind Franklin University of Medicine and Science
JP2025503098A (ja) ＭｕＳＫ標的化オリゴヌクレオチド
JP2026507503A (ja) アポリポタンパク質ｅ（ａｐｏｅ）発現を調節するためのオリゴヌクレオチド及びその使用方法
TW201238974A (en) MiRNAs in joint disease
KR20110114799A (ko) 천식과 관련된 ｓｌｃ６ａ１２ 유전자 다형성

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20220520

Patent event code: PA01051R01D

Comment text: International Patent Application

PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20220520

Comment text: Request for Examination of Application

PG1501 Laying open of application
PC1202 Submission of document of withdrawal before decision of registration

Comment text: [Withdrawal of Procedure relating to Patent, etc.] Withdrawal (Abandonment)

Patent event code: PC12021R01D

Patent event date: 20231205

WITB Written withdrawal of application