KR20240037996A - 안티센스 올리고뉴클레오티드 (aso) 유전자 억제 및 치료 - Google Patents

안티센스 올리고뉴클레오티드 (aso) 유전자 억제 및 치료 Download PDF

Info

Publication number
KR20240037996A
KR20240037996A KR1020247004273A KR20247004273A KR20240037996A KR 20240037996 A KR20240037996 A KR 20240037996A KR 1020247004273 A KR1020247004273 A KR 1020247004273A KR 20247004273 A KR20247004273 A KR 20247004273A KR 20240037996 A KR20240037996 A KR 20240037996A
Authority
KR
South Korea
Prior art keywords
aso
seq
jak2
ighmbp2
nucleotide sequence
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
KR1020247004273A
Other languages
English (en)
Korean (ko)
Inventor
바르틀로메이 프히호젠
산드라 스미제크
Original Assignee
반다 파마슈티칼즈, 인코퍼레이티드.
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 반다 파마슈티칼즈, 인코퍼레이티드. filed Critical 반다 파마슈티칼즈, 인코퍼레이티드.
Publication of KR20240037996A publication Critical patent/KR20240037996A/ko
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular 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
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • 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
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Neurosurgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020247004273A 2021-07-15 2022-07-13 안티센스 올리고뉴클레오티드 (aso) 유전자 억제 및 치료 Pending KR20240037996A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163222336P 2021-07-15 2021-07-15
US63/222,336 2021-07-15
US202163224362P 2021-07-21 2021-07-21
US63/224,362 2021-07-21
PCT/US2022/073668 WO2023288240A1 (en) 2021-07-15 2022-07-13 Antisense oligonucleotide (aso) gene inhibition and treatment

Publications (1)

Publication Number Publication Date
KR20240037996A true KR20240037996A (ko) 2024-03-22

Family

ID=82850241

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247004273A Pending KR20240037996A (ko) 2021-07-15 2022-07-13 안티센스 올리고뉴클레오티드 (aso) 유전자 억제 및 치료

Country Status (8)

Country Link
EP (1) EP4370133A1 (https=)
JP (1) JP2024525837A (https=)
KR (1) KR20240037996A (https=)
AU (1) AU2022312494A1 (https=)
CA (1) CA3224471A1 (https=)
IL (1) IL309813A (https=)
MX (1) MX2024000636A (https=)
WO (1) WO2023288240A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121039279A (zh) * 2023-05-05 2025-11-28 万达制药公司 反义寡核苷酸(aso)基因抑制和治疗

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014161046A1 (en) * 2013-04-04 2014-10-09 The Walter And Eliza Hall Institute Of Medical Research Methods of treating diseases characterized by excessive wnt signalling
WO2017214463A1 (en) * 2016-06-09 2017-12-14 The Regents Of The University Of California Compositions and methods for treating cancer and biomarkers to detect cancer stem cell reprogramming and progression

Also Published As

Publication number Publication date
AU2022312494A9 (en) 2024-01-25
MX2024000636A (es) 2024-02-06
WO2023288240A8 (en) 2023-12-14
AU2022312494A1 (en) 2024-01-18
JP2024525837A (ja) 2024-07-12
EP4370133A1 (en) 2024-05-22
CA3224471A1 (en) 2023-01-19
WO2023288240A1 (en) 2023-01-19
IL309813A (en) 2024-02-01
WO2023288240A9 (en) 2023-02-23

Similar Documents

Publication Publication Date Title
US10113171B2 (en) Methods for improving cognitive function via modulation of quinone reductase 2
EP4116418A1 (en) Compound and method for an allele-specific editing of the elane gene
KR20240037996A (ko) 안티센스 올리고뉴클레오티드 (aso) 유전자 억제 및 치료
US12036281B2 (en) Method of administering Lapatinib to a canine to treat bladder cancer harboring a BRAF mutation
US20240376478A1 (en) Antisense Oligonucleotide (ASO) Gene Inhibition and Treatment
US11951178B2 (en) Methods for treating subjects suffering from acute myeloid leukemia with FLT3 ligand-targeted miR-150 nanoparticles
CN117897161A (zh) 反义寡核苷酸(aso)基因抑制和治疗
JP2024529930A (ja) 低減されたオフターゲット効果を有する修飾低分子干渉ｒｎａ分子
US20220378879A1 (en) Application of peg interferon and protooncogene product targeting inhibitor in synergistic treatment of renal carcinoma
CN117043339A (zh) 用于治疗认知障碍的微小rna 195组合物和方法
JP7812535B2 (ja) 新型コロナウイルス感染症後遺症治療薬のスクリーニング方法及び新型コロナウイルス感染症後遺症モデル動物の製造方法
CN121039279A (zh) 反义寡核苷酸(aso)基因抑制和治疗
CN1501976A (zh) 用于联合治疗的crf2配体
CN112654706B (zh) 靶向淋巴细胞活化基因3(lag-3)的核酸适体及其用途
JP2002529083A (ja) アンチセンスオリゴマー
CN1948482B (zh) 抑制人RabJ基因表达的反义寡核苷酸序列及其应用
US20220184029A1 (en) Compositions and methods for treating neuroblastoma
He et al. Targeting Fgr-STAT3 Mediated Autophagy Inhibition in the Spinal Cord Alleviates Neuropathic Pain in Rats: L. He et al.
Lund et al. Selective Inhibition of Soluble Tumor Necrosis Factor Alters the Neuroinflammatory Response following Moderate Spinal Cord Injury in Mice. Biology 2023, 12, 845
KR20250009923A (ko) Gap43 활성 또는 발현을 조절하기 위한 조성물
WO2025096692A2 (en) Combination treatment of chronic myelomonocytic leukemia in patients with ras pathway mutations
KR101693186B1 (ko) ＰＡＮ-ＥｒｂＢ 억제제의 암 치료 효과 예측에 관한 정보 제공 방법
CN121588105A (zh) 吲哚胺2,3-二氧合酶1抑制剂在制备治疗癌症恶病质的药物中的应用

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20240205

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20250612

Comment text: Request for Examination of Application