KR20220012266A - 영아 악성 골화석증을 위한 유전자 요법 벡터 - Google Patents

영아 악성 골화석증을 위한 유전자 요법 벡터 Download PDF

Info

Publication number
KR20220012266A
KR20220012266A KR1020217040611A KR20217040611A KR20220012266A KR 20220012266 A KR20220012266 A KR 20220012266A KR 1020217040611 A KR1020217040611 A KR 1020217040611A KR 20217040611 A KR20217040611 A KR 20217040611A KR 20220012266 A KR20220012266 A KR 20220012266A
Authority
KR
South Korea
Prior art keywords
seq
expression cassette
transfer plasmid
tcirg1
lentiviral
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
KR1020217040611A
Other languages
English (en)
Korean (ko)
Inventor
브라이언 비어드
데이비드 릭스
라이 프라바카
Original Assignee
스페이스크래프트 세븐, 엘엘씨
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 스페이스크래프트 세븐, 엘엘씨 filed Critical 스페이스크래프트 세븐, 엘엘씨
Publication of KR20220012266A publication Critical patent/KR20220012266A/ko
Ceased legal-status Critical Current

Links

Images

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
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0387—Animal model for diseases of the immune 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
    • 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)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020217040611A 2019-05-23 2020-05-22 영아 악성 골화석증을 위한 유전자 요법 벡터 Ceased KR20220012266A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962852216P 2019-05-23 2019-05-23
US62/852,216 2019-05-23
PCT/US2020/034394 WO2020237219A1 (en) 2019-05-23 2020-05-22 Gene therapy vectors for infantile malignant osteopetrosis

Publications (1)

Publication Number Publication Date
KR20220012266A true KR20220012266A (ko) 2022-02-03

Family

ID=73458718

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217040611A Ceased KR20220012266A (ko) 2019-05-23 2020-05-22 영아 악성 골화석증을 위한 유전자 요법 벡터

Country Status (12)

Country Link
US (1) US20220218844A1 (https=)
EP (1) EP3973066A4 (https=)
JP (2) JP7623299B2 (https=)
KR (1) KR20220012266A (https=)
CN (1) CN113994008A (https=)
AU (1) AU2020278046A1 (https=)
BR (1) BR112021023473A2 (https=)
CA (1) CA3137700A1 (https=)
IL (1) IL288193A (https=)
MX (1) MX2021014337A (https=)
SG (1) SG11202112347RA (https=)
WO (1) WO2020237219A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE055862T2 (hu) 2016-04-20 2021-12-28 Centro De Investig Energeticas Készítmények és eljárások PKLR fokozott génexpressziójára
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
WO2019200167A1 (en) 2018-04-11 2019-10-17 Rocket Pharmaceuticals, Ltd. Compositions and methods for stem cell transplant
MX2020011386A (es) 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
KR20230043869A (ko) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
CN116042719B (zh) * 2022-12-28 2025-11-11 蓝图生物医药(广州)有限公司 一种重组腺相关病毒载体及其应用

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
GB0202771D0 (en) * 2002-02-06 2002-03-27 Univ Aberdeen Genetic markers for bone mass
CA2650507A1 (en) * 2006-04-27 2007-11-08 Universite De Montreal Assessment and reduction of risk of graft-versus-host disease
AU2013309488A1 (en) * 2012-08-29 2015-03-05 Nature Technology Corporation DNA plasmids with improved expression
WO2014077863A1 (en) * 2012-11-19 2014-05-22 Nature Technology Corporation Replicative minicircle vectors with improved expression
MX374532B (es) * 2013-06-17 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos.
CN116064678A (zh) * 2016-01-15 2023-05-05 美国基因技术国际有限公司 用于活化γ-δT细胞的方法和组合物
CN121102467A (zh) * 2016-04-22 2025-12-12 克莱格医学有限公司 用于细胞免疫疗法的组合物和方法
KR20240151726A (ko) 2016-09-08 2024-10-18 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. 판코니 빈혈 환자를 위한 유전자 치료

Also Published As

Publication number Publication date
EP3973066A1 (en) 2022-03-30
SG11202112347RA (en) 2021-12-30
MX2021014337A (es) 2022-03-17
EP3973066A4 (en) 2023-07-26
JP2025063200A (ja) 2025-04-15
WO2020237219A1 (en) 2020-11-26
JP2022532802A (ja) 2022-07-19
CA3137700A1 (en) 2020-11-26
CN113994008A (zh) 2022-01-28
AU2020278046A1 (en) 2022-01-27
US20220218844A1 (en) 2022-07-14
JP7623299B2 (ja) 2025-01-28
IL288193A (en) 2022-01-01
BR112021023473A2 (pt) 2022-02-01

Similar Documents

Publication Publication Date Title
JP2025063200A (ja) 乳児性悪性大理石骨病に対する遺伝子療法ベクター
AU2020260491B2 (en) Gene therapies for lysosomal disorders
RU2650860C2 (ru) Векторы для экспрессии простатоассоциированных антигенов
AU2020205228B2 (en) Gene therapies for lysosomal disorders
AU2015263150B2 (en) Lentiviral vectors
CN111372943B (zh) 腺病毒及其用途
KR20180069081A (ko) 심장 이상 및 기타 병리 이상의 치료를 위한 복수의 생물학적으로 활성화된 폴리펩티드를 단일 벡터로부터 발현하기 위한 조성물 및 방법
EP4028033A1 (en) Compositions and methods for tcr reprogramming using fusion proteins
KR20220112283A (ko) Hunter 질환 치료용 아데노-연합된 바이러스 벡터
AU2023265204A1 (en) Virus-derived construct plasmid library and construction of same
KR20080030956A (ko) 개선된 조절 발현 체계를 사용한 질병의 치료
TW202233830A (zh) 使用下一代工程化t細胞療法治療癌症之組合物及方法
RU2731513C2 (ru) Генотерапевтический ДНК-вектор на основе генотерапевтического ДНК-вектора VTvaf17, несущий целевой ген, выбранный из группы генов NOS2, NOS3, VIP, KCNMA1, CGRP, для повышения уровня экспрессии этих целевых генов, способ его получения и применения, штамм Escherichia coli SCS110-AF/VTvaf17-NOS2, или Escherichia coli SCS110-AF/VTvaf17-NOS3, или Escherichia coli SCS110-AF/VTvaf17-VIP, или Escherichia coli SCS110-AF/VTvaf17-KCNMA1, или Escherichia coli SCS110-AF/VTvaf17-CGRP, несущий генотерапевтический ДНК-вектор, способ его получения, способ производства в промышленных масштабах генотерапевтического ДНК-вектора
HK1116092A (en) Remedy for disease associated with apoptotic degeneration in ocular cell tissue with the use of siv-pedf vector
HK1122837A (en) Treatment of disease using an improved regulated expression system

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

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

D21 Rejection of application intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D21-EXM-PE0902 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0902 Notice of grounds for rejection

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

B15 Application refused following examination

Free format text: ST27 STATUS EVENT CODE: N-2-6-B10-B15-EXM-PE0601 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0601 Decision on rejection of patent

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