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PE20231570A1 - Vector neurod1 - Google Patents

Vector neurod1

Info

Publication number
PE20231570A1
PE20231570A1 PE2023001241A PE2023001241A PE20231570A1 PE 20231570 A1 PE20231570 A1 PE 20231570A1 PE 2023001241 A PE2023001241 A PE 2023001241A PE 2023001241 A PE2023001241 A PE 2023001241A PE 20231570 A1 PE20231570 A1 PE 20231570A1
Authority
PE
Peru
Prior art keywords
neurod1
vector
neurogliocytes
neurons
compositions
Prior art date
Application number
PE2023001241A
Other languages
English (en)
Inventor
Jie Xu
Original Assignee
Neuexcell Therapeutics Inc
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 Neuexcell Therapeutics Inc filed Critical Neuexcell Therapeutics Inc
Publication of PE20231570A1 publication Critical patent/PE20231570A1/es

Links

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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0619—Neurons
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • 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
    • C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
    • C12N2506/08—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from cells of the nervous 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
    • 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
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurosurgery (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

La presente divulgacion se refiere a vectores AAV, composiciones y metodos relacionados con la conversion de neurogliocitos en neuronas mediante el uso de una secuencia codificante de NeuroD1 en un vector AAV
PE2023001241A 2020-09-29 2021-09-28 Vector neurod1 PE20231570A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063084908P 2020-09-29 2020-09-29
US202163246545P 2021-09-21 2021-09-21
PCT/US2021/052299 WO2022072308A1 (en) 2020-09-29 2021-09-28 Neurod1 vector

Publications (1)

Publication Number Publication Date
PE20231570A1 true PE20231570A1 (es) 2023-10-04

Family

ID=80931167

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2023001241A PE20231570A1 (es) 2020-09-29 2021-09-28 Vector neurod1

Country Status (13)

Country Link
US (1) US20220106613A1 (es)
EP (1) EP4222270A4 (es)
JP (1) JP2023544068A (es)
KR (1) KR20230123460A (es)
AU (1) AU2021355362A1 (es)
CA (1) CA3197321A1 (es)
CL (1) CL2023000919A1 (es)
CO (1) CO2023005298A2 (es)
IL (1) IL301749A (es)
MX (1) MX2023003655A (es)
PE (1) PE20231570A1 (es)
PH (1) PH12023550861A1 (es)
WO (1) WO2022072308A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024222929A1 (en) * 2023-04-28 2024-10-31 Neuexcell Therapeutics (Suzhou) Co., Ltd. Gene therapy compositions and methods for reprogramming glial cells
WO2025108407A2 (en) * 2023-11-23 2025-05-30 Neuexcell Therapeutics (Suzhou) Co., Ltd. Gene therapy compositions and methods for treating glioma
WO2025113676A1 (en) * 2023-11-29 2025-06-05 Neuexcell Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treating stroke in primates

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9388427B2 (en) * 2002-12-02 2016-07-12 Biovec, Llc In vivo and ex vivo gene transfer into renal tissue using gutless adenovirus vectors
CA2683469C (en) * 2007-04-12 2019-11-26 The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Genetic suppression and replacement
EP1995309A1 (en) * 2007-05-21 2008-11-26 Vivalis Recombinant protein production in avian EBx® cells
CA2855653A1 (en) * 2011-11-18 2013-05-23 Universite Laval Methods and products for increasing frataxin levels and uses thereof
CA2876293C (en) * 2012-06-27 2023-10-10 Arthrogen B.V. Combination for treating an inflammatory disorder
CN113512565A (zh) * 2012-07-19 2021-10-19 宾夕法尼亚州研究基金会 再生用于在神经系统中治疗疾病和损伤的功能性神经元
US20150065376A1 (en) * 2013-08-12 2015-03-05 Holger Knaut Expression constructs encoding g protein coupled receptors and methods of use thereof
KR101712247B1 (ko) * 2014-09-29 2017-03-06 단국대학교 산학협력단 암 특이적 트랜스-스플라이싱 라이보자임 및 이의 용도
JP6754361B2 (ja) * 2014-12-16 2020-09-09 ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ 若年型バッテン病のための遺伝子療法
WO2016172155A1 (en) * 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
US10973930B2 (en) * 2016-02-18 2021-04-13 The Penn State Research Foundation Generating GABAergic neurons in brains
ES2718192T3 (es) * 2016-02-25 2019-06-28 Provecs Medical Gmbh Sistema vector inmunoestimulante novedoso
CN110177803A (zh) * 2016-11-22 2019-08-27 T细胞受体治疗公司 用于使用融合蛋白进行tcr重新编程的组合物和方法
GB201720948D0 (en) * 2017-12-15 2018-01-31 Autolus Ltd Plasmid system
KR20210087462A (ko) * 2018-10-05 2021-07-12 유니버시티 오브 매사추세츠 Gm1 및 gm2 강글리오시드증의 치료를 위한 raav 벡터
GB2593353B (en) * 2018-10-16 2023-03-01 Blueallele Corp Methods for targeted insertion of DNA in genes
CN113227368B (zh) * 2018-10-22 2023-07-07 因思科瑞普特公司 工程化酶
EA202191418A1 (ru) * 2018-11-21 2021-08-05 Страйдбайо, Инк. Рекомбинантные вирусные векторы и нуклеиновые кислоты для их получения

Also Published As

Publication number Publication date
IL301749A (en) 2023-05-01
JP2023544068A (ja) 2023-10-19
PH12023550861A1 (en) 2023-11-20
WO2022072308A1 (en) 2022-04-07
EP4222270A4 (en) 2025-02-26
CL2023000919A1 (es) 2023-11-17
AU2021355362A9 (en) 2023-07-06
CA3197321A1 (en) 2022-04-07
EP4222270A1 (en) 2023-08-09
KR20230123460A (ko) 2023-08-23
MX2023003655A (es) 2023-06-22
AU2021355362A1 (en) 2023-05-11
CO2023005298A2 (es) 2023-07-10
US20220106613A1 (en) 2022-04-07

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