KR20240043752A - 막횡단 단백질 1(kremen1) 억제제를 함유하는 크링글을 사용하여 감소된 골밀도를 치료하는 방법 - Google Patents

막횡단 단백질 1(kremen1) 억제제를 함유하는 크링글을 사용하여 감소된 골밀도를 치료하는 방법 Download PDF

Info

Publication number
KR20240043752A
KR20240043752A KR1020247003956A KR20247003956A KR20240043752A KR 20240043752 A KR20240043752 A KR 20240043752A KR 1020247003956 A KR1020247003956 A KR 1020247003956A KR 20247003956 A KR20247003956 A KR 20247003956A KR 20240043752 A KR20240043752 A KR 20240043752A
Authority
KR
South Korea
Prior art keywords
kremen1
nucleic acid
subject
variant
acid molecule
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
KR1020247003956A
Other languages
English (en)
Korean (ko)
Inventor
조나스 보빈
올루카요데 소시나
루카 안드레아 로타
아리스 바라스
Original Assignee
리제너론 파마슈티칼스 인코포레이티드
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 리제너론 파마슈티칼스 인코포레이티드 filed Critical 리제너론 파마슈티칼스 인코포레이티드
Publication of KR20240043752A publication Critical patent/KR20240043752A/ko
Pending legal-status Critical Current

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1138Non-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 receptors or cell surface proteins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6813Hybridisation assays
    • C12Q1/6827Hybridisation assays for detection of mutation or polymorphism
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/156Polymorphic or mutational markers

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pathology (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
KR1020247003956A 2021-07-02 2022-06-30 막횡단 단백질 1(kremen1) 억제제를 함유하는 크링글을 사용하여 감소된 골밀도를 치료하는 방법 Pending KR20240043752A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163218212P 2021-07-02 2021-07-02
US63/218,212 2021-07-02
PCT/US2022/035783 WO2023278741A1 (en) 2021-07-02 2022-06-30 Methods of treating decreased bone mineral density with kringle containing transmembrane protein 1 (kremen1) inhibitors

Publications (1)

Publication Number Publication Date
KR20240043752A true KR20240043752A (ko) 2024-04-03

Family

ID=82748403

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247003956A Pending KR20240043752A (ko) 2021-07-02 2022-06-30 막횡단 단백질 1(kremen1) 억제제를 함유하는 크링글을 사용하여 감소된 골밀도를 치료하는 방법

Country Status (9)

Country Link
US (1) US20230021584A1 (https=)
EP (1) EP4363585A1 (https=)
JP (1) JP2024524375A (https=)
KR (1) KR20240043752A (https=)
CN (1) CN117836412A (https=)
AU (1) AU2022300984A1 (https=)
CA (1) CA3222830A1 (https=)
IL (1) IL309385A (https=)
WO (1) WO2023278741A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118298911B (zh) * 2024-04-12 2024-11-22 中山大学孙逸仙纪念医院 一种多部位慢性疼痛潜在治疗靶点确定系统

Also Published As

Publication number Publication date
JP2024524375A (ja) 2024-07-05
US20230021584A1 (en) 2023-01-26
EP4363585A1 (en) 2024-05-08
AU2022300984A1 (en) 2024-01-04
CN117836412A (zh) 2024-04-05
WO2023278741A1 (en) 2023-01-05
IL309385A (en) 2024-02-01
CA3222830A1 (en) 2023-01-05

Similar Documents

Publication Publication Date Title
AU2020250262B2 (en) Compositions for modulating tau expression
KR102149483B1 (ko) 예측변수 인자들을 이용하여 동정된 환자 부분 모집단에서 암 치료를 위한 마시티닙의 용도
KR102723469B1 (ko) 타우 발현을 조절하기 위한 올리고뉴클레오티드
KR102585898B1 (ko) B형 간염 감염을 치료하기 위한 PAPD5 및 PAPD7 mRNA의 감소용 핵산 분자
AU2017276286A1 (en) Modulation of huntingtin expression
KR20220056252A (ko) B형 간염 감염을 치료하기 위해 PAPD5 또는 PAPD7 mRNA를 감소시키기 위한 핵산 분자
KR20150023904A (ko) 전립선암의 진단 및 치료에서의 마커의 용도
US20030235847A1 (en) Association of polymorphisms in the SOST gene region with bone mineral density
US20030099958A1 (en) Diagnosis and treatment of vascular disease
KR20230057410A (ko) Pcsk9 및 ldlr 조절인자를 사용한 패혈증 치료
KR20240043752A (ko) 막횡단 단백질 1(kremen1) 억제제를 함유하는 크링글을 사용하여 감소된 골밀도를 치료하는 방법
CN115362255A (zh) 用无七之子2(sos2)抑制剂治疗眼科疾患
CN117795076A (zh) 用丝裂原活化蛋白激酶激酶激酶15(map3k15)抑制剂治疗代谢病症的方法
KR20240159455A (ko) 신경성 유전자좌 노치 상동체 단백질 3(notch3) 제제를 이용한 뇌혈관 질환의 치료
KR20190081158A (ko) Itpkc 및 slc11a1 유전자 다형성을 이용한 가와사키병 발병 예측 방법
CN116356008A (zh) 先天性心脏病相关setd2基因新发变异位点及其应用
CN113825839A (zh) 用固醇调节元件结合蛋白切割激活蛋白(scap)抑制剂治疗脂质水平升高
AU2017211976B2 (en) Means and methods for staging, typing and treating a cancerous disease
KR20240043753A (ko) Wnt 패밀리 구성원 5B(WNT5B) 저해제를 사용한 감소된 골밀도의 치료
TW202600823A (zh) Mapt反義寡核苷酸
CN115485394A (zh) 溶质载剂家族26成员5(slc26a5)变体及其用途
RU2823361C1 (ru) Композиции для модулирования экспрессии тау-белка

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

A201 Request for examination
E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000