KR20230087436A - 당뇨병을 치료하기 위한 변형된 인슐린 및 글루코키나제 핵산 - Google Patents

당뇨병을 치료하기 위한 변형된 인슐린 및 글루코키나제 핵산 Download PDF

Info

Publication number
KR20230087436A
KR20230087436A KR1020237003717A KR20237003717A KR20230087436A KR 20230087436 A KR20230087436 A KR 20230087436A KR 1020237003717 A KR1020237003717 A KR 1020237003717A KR 20237003717 A KR20237003717 A KR 20237003717A KR 20230087436 A KR20230087436 A KR 20230087436A
Authority
KR
South Korea
Prior art keywords
seq
polynucleotide
nucleic acid
sequence
vector
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
KR1020237003717A
Other languages
English (en)
Korean (ko)
Inventor
나치 굽타
웨이란 션
미켈 가르시아 마르티네즈
베로니카 히메네즈 센자노
마리아 파티마 보쉬 투베르트
Original Assignee
크리야 테라퓨틱스, 인크.
우니버시타트 아우토노마 데 바르셀로나
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 크리야 테라퓨틱스, 인크., 우니버시타트 아우토노마 데 바르셀로나 filed Critical 크리야 테라퓨틱스, 인크.
Publication of KR20230087436A publication Critical patent/KR20230087436A/ko
Pending legal-status Critical Current

Links

Images

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
    • C07K14/575—Hormones
    • C07K14/62—Insulins
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/54—Interleukins [IL]
    • C07K14/5412—IL-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
    • 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
    • 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
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1205—Phosphotransferases with an alcohol group as acceptor (2.7.1), e.g. protein kinases
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/01—Phosphotransferases with an alcohol group as acceptor (2.7.1)
    • C12Y207/01002—Glucokinase (2.7.1.2)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/50—Fusion polypeptide containing protease site
    • 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
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Endocrinology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Emergency Medicine (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
KR1020237003717A 2020-07-03 2021-07-02 당뇨병을 치료하기 위한 변형된 인슐린 및 글루코키나제 핵산 Pending KR20230087436A (ko)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202063047965P 2020-07-03 2020-07-03
US63/047,965 2020-07-03
US202063054162P 2020-07-20 2020-07-20
US63/054,162 2020-07-20
US202063067264P 2020-08-18 2020-08-18
US63/067,264 2020-08-18
US202163141918P 2021-01-26 2021-01-26
US63/141,918 2021-01-26
US202163188778P 2021-05-14 2021-05-14
US63/188,778 2021-05-14
PCT/US2021/040366 WO2022006551A2 (en) 2020-07-03 2021-07-02 Modified insulin and glucokinase nucleic acids for treating diabetes

Publications (1)

Publication Number Publication Date
KR20230087436A true KR20230087436A (ko) 2023-06-16

Family

ID=79317842

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020237003717A Pending KR20230087436A (ko) 2020-07-03 2021-07-02 당뇨병을 치료하기 위한 변형된 인슐린 및 글루코키나제 핵산

Country Status (10)

Country Link
US (1) US20240279300A1 (https=)
EP (1) EP4176060A4 (https=)
JP (2) JP7837963B2 (https=)
KR (1) KR20230087436A (https=)
CN (1) CN116234904A (https=)
AU (1) AU2021300450A1 (https=)
CA (1) CA3174156A1 (https=)
IL (1) IL299545A (https=)
MX (1) MX2023000099A (https=)
WO (1) WO2022006551A2 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20250288693A1 (en) * 2022-04-29 2025-09-18 Kriya Therapeutics, Inc. Insulin and glucokinase gene therapy compositions and its use for treating diabetes
AU2024370040A1 (en) * 2023-11-01 2026-04-23 Kriya Therapeutics, Inc. Aav mediated insulin and glucokinase gene therapy for treating diabetes

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1836047B (zh) * 2003-06-17 2010-12-22 赛姆生物系统遗传公司 在植物中生产胰岛素的方法
WO2012007458A1 (en) 2010-07-12 2012-01-19 Universidad Autónoma De Barcelona Gene therapy composition for use in diabetes treatment
EP3242945B1 (en) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes

Also Published As

Publication number Publication date
CA3174156A1 (en) 2022-01-06
IL299545A (en) 2023-02-01
AU2021300450A1 (en) 2023-03-02
EP4176060A4 (en) 2024-08-07
CN116234904A (zh) 2023-06-06
WO2022006551A2 (en) 2022-01-06
MX2023000099A (es) 2023-04-20
JP2026034616A (ja) 2026-02-27
EP4176060A2 (en) 2023-05-10
WO2022006551A3 (en) 2022-02-03
US20240279300A1 (en) 2024-08-22
JP2023542241A (ja) 2023-10-05
JP7837963B2 (ja) 2026-03-31

Similar Documents

Publication Publication Date Title
KR102808368B1 (ko) 선택적 유전자 조절을 위한 조성물 및 방법
EP1287125B1 (en) Dna sequences encoding dystrophin minigenes and methods of use thereof
EP3242945B1 (en) Single-vector gene construct comprising insulin and glucokinase genes
AU2017322377B2 (en) Acid-alpha glucosidase variants and uses thereof
KR20200107949A (ko) 조작된 dna 결합 단백질
AU2019317754B2 (en) Mini-GDE for the treatment of glycogen storage disease III
KR20210144696A (ko) 라민병증 치료용 조성물 및 치료 방법
JP2026034616A (ja) 糖尿病を処置するための修飾型インスリンおよびグルコキナーゼ核酸
CN116685329B (zh) 核酸构建体及其用于治疗脊髓性肌肉萎缩症的用途
CN112639108A (zh) 治疗非综合征性感觉神经性听力损失的方法
KR20230003477A (ko) 비-바이러스성 dna 벡터 및 인자 ix 치료제 발현을 위한 이의 용도
AU2026201371A1 (en) Methods of treating CLRN1-associated hearing loss and/or vision loss
KR20230003478A (ko) 비-바이러스성 dna 벡터 및 고셰 치료제 발현을 위한 이의 용도
KR20230002788A (ko) 신피질 레이어 5 글루타메이트성 뉴런에서 유전자 발현을 선택적으로 조절하기 위한 인공 발현 작제물
US20250288693A1 (en) Insulin and glucokinase gene therapy compositions and its use for treating diabetes
WO2022221462A1 (en) Vector constructs for delivery of nucleic acids encoding therapeutic vlcad or mcad and methods of using the same
RU2780329C2 (ru) Варианты кислой альфа-глюкозидазы и их использование
WO2025096954A1 (en) Aav mediated insulin and glucokinase gene therapy for treating diabetes
CN117836420A (zh) 重组tert编码病毒基因组和运载体

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20230201

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20240702

Comment text: Request for Examination of Application