AU2021325717A1 - Gene therapy using nucleic acid constructs comprising methyl CpG binding protein 2 (MeCP2) promoter sequences - Google Patents

Gene therapy using nucleic acid constructs comprising methyl CpG binding protein 2 (MeCP2) promoter sequences Download PDF

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Publication number
AU2021325717A1
AU2021325717A1 AU2021325717A AU2021325717A AU2021325717A1 AU 2021325717 A1 AU2021325717 A1 AU 2021325717A1 AU 2021325717 A AU2021325717 A AU 2021325717A AU 2021325717 A AU2021325717 A AU 2021325717A AU 2021325717 A1 AU2021325717 A1 AU 2021325717A1
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AU
Australia
Prior art keywords
mecp2
nucleic acid
binding protein
gene therapy
promoter sequences
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Pending
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AU2021325717A
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English (en)
Inventor
Nikita DALAL
Patrick Mark Downey
Ami KABADI
Toshal Rohit Patel
Amulya Nidhi Shrivastava
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UCB Biopharma SRL
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UCB Biopharma SRL
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=77655525&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=AU2021325717(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by UCB Biopharma SRL filed Critical UCB Biopharma SRL
Publication of AU2021325717A1 publication Critical patent/AU2021325717A1/en
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • 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
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    • 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
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hospice & Palliative Care (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2021325717A 2020-08-12 2021-08-11 Gene therapy using nucleic acid constructs comprising methyl CpG binding protein 2 (MeCP2) promoter sequences Pending AU2021325717A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063064431P 2020-08-12 2020-08-12
US63/064,431 2020-08-12
PCT/EP2021/072365 WO2022034130A1 (en) 2020-08-12 2021-08-11 Gene therapy using nucleic acid constructs comprising methyl cpg binding protein 2 (mecp2) promoter sequences

Publications (1)

Publication Number Publication Date
AU2021325717A1 true AU2021325717A1 (en) 2023-03-02

Family

ID=77655525

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021325717A Pending AU2021325717A1 (en) 2020-08-12 2021-08-11 Gene therapy using nucleic acid constructs comprising methyl CpG binding protein 2 (MeCP2) promoter sequences

Country Status (19)

Country Link
US (1) US20230295657A1 (https=)
EP (1) EP4196171A1 (https=)
JP (1) JP2023537980A (https=)
KR (1) KR20230044506A (https=)
CN (1) CN116113441A (https=)
AR (1) AR123206A1 (https=)
AU (1) AU2021325717A1 (https=)
BR (1) BR112023002374A2 (https=)
CA (1) CA3188748A1 (https=)
CL (1) CL2023000419A1 (https=)
CO (1) CO2023000444A2 (https=)
EC (1) ECSP23016688A (https=)
IL (1) IL300294A (https=)
MX (1) MX2023001701A (https=)
PE (1) PE20230914A1 (https=)
PH (1) PH12023550145A1 (https=)
TW (1) TWI902867B (https=)
WO (1) WO2022034130A1 (https=)
ZA (1) ZA202300378B (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20240033332A1 (en) * 2020-12-17 2024-02-01 Kyoto University Agent for activating a neuron
GB202216168D0 (en) 2022-10-31 2022-12-14 UCB Biopharma SRL Route of administration
WO2024163823A1 (en) * 2023-02-02 2024-08-08 Shape Therapeutics Inc. Tissue-specific enhancers for regulating transcription
JP2026509301A (ja) * 2023-03-14 2026-03-17 ユーシービー バイオファルマ エスアールエル 遺伝子治療
CN121335978A (zh) * 2023-06-16 2026-01-13 舒泰神（北京）生物制药股份有限公司 一种人源MeCp2启动子及其用途
WO2025213097A1 (en) * 2024-04-05 2025-10-09 The Children's Hospital Of Philadelphia Novel gene therapy constructs for stxbp1 haploinsufficiency

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3075858T3 (pl) * 2007-12-21 2020-07-13 Novartis Ag Ssaczy wektor ekspresyjny
EA201492116A1 (ru) * 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
WO2016077687A1 (en) * 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
EP4344741A3 (en) * 2014-11-21 2024-08-28 The University of North Carolina at Chapel Hill Aav vectors targeted to the central nervous system
WO2016100575A1 (en) * 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
US20190328906A1 (en) * 2016-03-02 2019-10-31 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
TWI601821B (zh) * 2016-04-15 2017-10-11 中央研究院 治療神經發展性疾病
CN113166781A (zh) * 2018-10-15 2021-07-23 沃雅戈治疗公司 在杆状病毒/Sf9系统中大规模生产rAAV的表达载体

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Publication number Publication date
KR20230044506A (ko) 2023-04-04
TW202221018A (zh) 2022-06-01
CL2023000419A1 (es) 2023-07-21
US20230295657A1 (en) 2023-09-21
CO2023000444A2 (es) 2023-01-26
EP4196171A1 (en) 2023-06-21
IL300294A (en) 2023-04-01
BR112023002374A2 (pt) 2023-03-21
AR123206A1 (es) 2022-11-09
TWI902867B (zh) 2025-11-01
MX2023001701A (es) 2023-03-09
CN116113441A (zh) 2023-05-12
PH12023550145A1 (en) 2024-03-18
WO2022034130A1 (en) 2022-02-17
ZA202300378B (en) 2024-04-24
JP2023537980A (ja) 2023-09-06
CA3188748A1 (en) 2022-02-17
PE20230914A1 (es) 2023-06-02
ECSP23016688A (es) 2023-04-28

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