KR20190089988A - I형 점액다당류증에 대한 유전자 요법 - Google Patents

I형 점액다당류증에 대한 유전자 요법 Download PDF

Info

Publication number
KR20190089988A
KR20190089988A KR1020197019344A KR20197019344A KR20190089988A KR 20190089988 A KR20190089988 A KR 20190089988A KR 1020197019344 A KR1020197019344 A KR 1020197019344A KR 20197019344 A KR20197019344 A KR 20197019344A KR 20190089988 A KR20190089988 A KR 20190089988A
Authority
KR
South Korea
Prior art keywords
promoter
cell
cells
mps
polynucleotide
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.)
Ceased
Application number
KR1020197019344A
Other languages
English (en)
Korean (ko)
Inventor
켄드릭 에이. 고스
제프리 비. 파슨스
Original Assignee
블루버드 바이오, 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 블루버드 바이오, 인코포레이티드. filed Critical 블루버드 바이오, 인코포레이티드.
Publication of KR20190089988A publication Critical patent/KR20190089988A/ko
Ceased 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
    • C12N15/63—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
    • 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
    • A61P11/00—Drugs for disorders of the respiratory system
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/16—Otologicals
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • 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)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Ophthalmology & Optometry (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Diabetes (AREA)
KR1020197019344A 2016-12-06 2017-12-06 I형 점액다당류증에 대한 유전자 요법 Ceased KR20190089988A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662430795P 2016-12-06 2016-12-06
US62/430,795 2016-12-06
PCT/US2017/064913 WO2018106807A1 (en) 2016-12-06 2017-12-06 Gene therapy for mucopolysaccharidosis, type i

Publications (1)

Publication Number Publication Date
KR20190089988A true KR20190089988A (ko) 2019-07-31

Family

ID=62491308

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020197019344A Ceased KR20190089988A (ko) 2016-12-06 2017-12-06 I형 점액다당류증에 대한 유전자 요법

Country Status (13)

Country Link
US (1) US20220339296A1 (cg-RX-API-DMAC10.html)
EP (1) EP3551750A4 (cg-RX-API-DMAC10.html)
JP (1) JP2019536484A (cg-RX-API-DMAC10.html)
KR (1) KR20190089988A (cg-RX-API-DMAC10.html)
CN (1) CN110214182A (cg-RX-API-DMAC10.html)
AU (1) AU2017370662A1 (cg-RX-API-DMAC10.html)
BR (1) BR112019011635A2 (cg-RX-API-DMAC10.html)
CA (1) CA3046079A1 (cg-RX-API-DMAC10.html)
IL (1) IL267057A (cg-RX-API-DMAC10.html)
MA (1) MA47847A (cg-RX-API-DMAC10.html)
MX (1) MX2019006552A (cg-RX-API-DMAC10.html)
RU (1) RU2019120721A (cg-RX-API-DMAC10.html)
WO (1) WO2018106807A1 (cg-RX-API-DMAC10.html)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102673203B1 (ko) 2016-04-20 2024-06-10 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. Pklr의 유전자 발현 증강을 위한 조성물 및 방법
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
AU2018352583B2 (en) 2017-10-16 2025-04-17 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency
US12539316B2 (en) 2018-04-11 2026-02-03 Spacecraft Seven, Llc Compositions and methods for stem cell transplant
IL280465B2 (en) * 2018-07-30 2025-03-01 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Methods for gene modification of hematopoietic cells
CN115109790A (zh) * 2021-03-23 2022-09-27 北京据德医药科技有限公司 一种重组a-L-艾杜糖醛酸普酶及其制备方法
MX2023014228A (es) * 2021-06-08 2024-01-25 Touchlight Ip Ltd Vector lentiviral.
WO2024201066A1 (en) * 2023-03-31 2024-10-03 Dawn Therapeutics Limited Novel vector

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2872170B1 (fr) * 2004-06-25 2006-11-10 Centre Nat Rech Scient Cnrse Lentivirus non interactif et non replicatif, preparation et utilisations
US20110294114A1 (en) * 2009-12-04 2011-12-01 Cincinnati Children's Hospital Medical Center Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells
DK2717922T3 (en) * 2011-06-10 2019-01-07 Bluebird Bio Inc GENERATIVE VECTORS FOR ADDRENOLEUCODYSTROPHY AND ADDRENOMYELONE EUROPE
EP2855684A1 (en) * 2012-05-25 2015-04-08 Commissariat à l'Énergie Atomique et aux Énergies Alternatives Vector for the selective silencing of a gene in astrocytes
WO2014201252A2 (en) * 2013-06-13 2014-12-18 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
CN108883136A (zh) * 2016-02-12 2018-11-23 蓝鸟生物公司 Vcn增强子组合物及其使用方法

Also Published As

Publication number Publication date
US20220339296A1 (en) 2022-10-27
CA3046079A1 (en) 2018-06-14
MA47847A (fr) 2020-01-29
BR112019011635A2 (pt) 2019-11-12
IL267057A (en) 2019-08-29
JP2019536484A (ja) 2019-12-19
EP3551750A4 (en) 2020-07-22
AU2017370662A1 (en) 2019-06-27
MX2019006552A (es) 2019-10-15
CN110214182A (zh) 2019-09-06
WO2018106807A1 (en) 2018-06-14
RU2019120721A (ru) 2021-01-11
EP3551750A1 (en) 2019-10-16

Similar Documents

Publication Publication Date Title
CN110214182A (zh) 用于i型黏多糖贮积症的基因治疗
AU2018346105B2 (en) Gene therapies for lysosomal disorders
AU2015263150B2 (en) Lentiviral vectors
CN107002095B (zh) 用于治疗溶酶体贮积症的腺伴随病毒载体
CN110835633B (zh) 利用优化的基因密码子扩展系统制备ptc稳定细胞系及应用
CN110564772A (zh) 改造宿主细胞基因组的方法及其用途
DK2828384T3 (en) PROCEDURE FOR SURVIVING SENSITIVITY TO CHEMICAL DNA MODIFICATIONS OF CONSTRUCTED SPEECH DNA BINDING DOMAINS
KR20090103922A (ko) 비분절 음성 가닥 ｒｎａ 바이러스 생성하는 세포 및 방법
CA3005474A1 (en) Compositions and methods for correction of heritable ocular disease
US20220323612A1 (en) Gene therapy of neuronal ceroid lipofuscinoses
US20200071721A1 (en) Gene therapy for mucopolysaccharidosis, type ii
CN111886342B (zh) 拉沙疫苗
CN115707777B (zh) 重组肠道病毒a71病毒样颗粒及其用途
CN115707778B (zh) 重组柯萨奇病毒a10病毒样颗粒及其用途
CN113652450B (zh) 慢病毒载体的制备方法及得到的慢病毒载体和应用
US20030031650A1 (en) Drug inducible system and use thereof
KR102543504B1 (ko) 세포 손상 관찰을 위한 형광 단백질 변이체 및 이를 이용한 약물 독성 평가 방법
HK40048832A (en) Gene therapies for lysosomal disorders
HK40046514A (en) Gene therapies for lysosomal disorders
WO2002094989A2 (en) Retroviral vectors and methods of using same

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

R17-X000 Change to representative recorded

St.27 status event code: A-3-3-R10-R17-oth-X000

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

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

E601 Decision to refuse application
PE0601 Decision on rejection of patent

St.27 status event code: N-2-6-B10-B15-exm-PE0601

R18 Changes to party contact information recorded

Free format text: ST27 STATUS EVENT CODE: A-3-3-R10-R18-OTH-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000