AU2020278046A1 - Gene therapy vectors for infantile malignant osteopetrosis - Google Patents

Gene therapy vectors for infantile malignant osteopetrosis Download PDF

Info

Publication number
AU2020278046A1
AU2020278046A1 AU2020278046A AU2020278046A AU2020278046A1 AU 2020278046 A1 AU2020278046 A1 AU 2020278046A1 AU 2020278046 A AU2020278046 A AU 2020278046A AU 2020278046 A AU2020278046 A AU 2020278046A AU 2020278046 A1 AU2020278046 A1 AU 2020278046A1
Authority
AU
Australia
Prior art keywords
seq
transfer plasmid
expression cassette
lentiviral
tcirg1
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.)
Abandoned
Application number
AU2020278046A
Other languages
English (en)
Inventor
Brian Beard
Raj PRABHAKAR
David RICKS
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Spacecraft Seven LLC
Original Assignee
Spacecraft Seven LLC
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 Spacecraft Seven LLC filed Critical Spacecraft Seven LLC
Publication of AU2020278046A1 publication Critical patent/AU2020278046A1/en
Abandoned 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
    • 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)
AU2020278046A 2019-05-23 2020-05-22 Gene therapy vectors for infantile malignant osteopetrosis Abandoned AU2020278046A1 (en)

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
AU2020278046A1 true AU2020278046A1 (en) 2022-01-27

Family

ID=73458718

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020278046A Abandoned AU2020278046A1 (en) 2019-05-23 2020-05-22 Gene therapy vectors for infantile malignant osteopetrosis

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
KR20220012266A (ko) 2022-02-03
JP2022532802A (ja) 2022-07-19
CA3137700A1 (en) 2020-11-26
CN113994008A (zh) 2022-01-28
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
JP7623299B2 (ja) 乳児性悪性大理石骨病に対する遺伝子療法ベクター
JP7586810B2 (ja) ダノン病治療用遺伝子療法ベクター
EP4103214B1 (en) Gene therapy vectors for treating heart disease
JP2018519827A (ja) 逆配向のヒトユビキチンｃプロモーターを含むレトロウイルスベクター
US20250270587A1 (en) Vector
KR20240025507A (ko) 미성숙 종결 코돈-매개 장애를 치료하기 위한 방법 및 조성물
TW202129002A (zh) 用於myh7關聯之心肌病之基因療法組合物及治療
US20220170045A1 (en) Augmentations to lentiviral vectors (cclc-mgata/ank-core lcr-beta-as3-fb) to increase expression
EP4158030A1 (en) Improved lentiviral vector transfer plasmid and methods of use
JP7197901B2 (ja) Ｈｕｎｔｅｒ症候群の遺伝子治療に使用されるベクタープラスミド、レンチウイルスベクターシステム、細胞、及び、細胞製剤
WO2024243073A1 (en) Anti-codon engineered suppressor transfer rnas
TW202337476A (zh) 用於與肌肉萎縮蛋白有關之心肌病之基因療法組合物及治療
JP2023507885A5 (https=)
WO2019228525A1 (en) Lentiviral vector used for treatment of x-scid, lentivirus, and preparation method and application thereof
Maurya et al. Retroviral vectors and gene therapy: an update
RU2808459C2 (ru) Геннотерапевтические векторы для лечения болезни данона
CN114507692B (zh) 用于治疗法布里病的腺相关病毒载体及其用途

Legal Events

Date Code Title Description
MK1 Application lapsed section 142(2)(a) - no request for examination in relevant period