SG11202010061PA - Gene therapy for cns degeneration - Google Patents

Gene therapy for cns degeneration

Info

Publication number
SG11202010061PA
SG11202010061PA SG11202010061PA SG11202010061PA SG11202010061PA SG 11202010061P A SG11202010061P A SG 11202010061PA SG 11202010061P A SG11202010061P A SG 11202010061PA SG 11202010061P A SG11202010061P A SG 11202010061PA SG 11202010061P A SG11202010061P A SG 11202010061PA
Authority
SG
Singapore
Prior art keywords
gene therapy
cns degeneration
cns
degeneration
therapy
Prior art date
Application number
SG11202010061PA
Other languages
English (en)
Inventor
Annahita Keravala
Paven Battiprolu
Raj Prabhakar
Roderick Wong
Piratip Pratumsuwan
Naveen Yalamanchi
Original Assignee
Rocket Pharmaceuticals Ltd
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 Rocket Pharmaceuticals Ltd filed Critical Rocket Pharmaceuticals Ltd
Publication of SG11202010061PA publication Critical patent/SG11202010061PA/en

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    • 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
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
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    • 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
    • C—CHEMISTRY; METALLURGY
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    • 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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • 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
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N9/10—Transferases (2.)
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    • C12N9/104—Aminoacyltransferases (2.3.2)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/93—Ligases (6)
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Animal Behavior & Ethology (AREA)
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  • Epidemiology (AREA)
  • Psychology (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
SG11202010061PA 2018-04-27 2019-04-29 Gene therapy for cns degeneration SG11202010061PA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862664006P 2018-04-27 2018-04-27
PCT/US2019/029744 WO2019210325A1 (en) 2018-04-27 2019-04-29 Gene therapy for cns degeneration

Publications (1)

Publication Number Publication Date
SG11202010061PA true SG11202010061PA (en) 2020-11-27

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ID=68295821

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Application Number Title Priority Date Filing Date
SG11202010061PA SG11202010061PA (en) 2018-04-27 2019-04-29 Gene therapy for cns degeneration

Country Status (13)

Country Link
US (1) US12378576B2 (https=)
EP (1) EP3810781A4 (https=)
JP (2) JP2021522273A (https=)
KR (1) KR20210005889A (https=)
CN (1) CN112368390B (https=)
AU (1) AU2019261646A1 (https=)
BR (1) BR112020021903A2 (https=)
CA (1) CA3096293A1 (https=)
IL (1) IL278213B2 (https=)
MX (1) MX2020011386A (https=)
RU (1) RU2020134965A (https=)
SG (1) SG11202010061PA (https=)
WO (1) WO2019210325A1 (https=)

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EP3720507A4 (en) * 2017-12-07 2021-08-25 Ramot at Tel-Aviv University Ltd. TREATMENT OF PARKINSON PATIENTS WITH MUTATIONS IN THE LRRK2 GENE
BR112020020841A2 (pt) 2018-04-11 2021-01-19 Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus Composições e métodos para transplante de célulastronco
CA3096293A1 (en) 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration
JP2023535121A (ja) * 2020-05-20 2023-08-16 スペースクラフト セブン リミテッド ライアビリティ カンパニー 操作されたパーキン及びその使用
JP2023526449A (ja) * 2020-05-21 2023-06-21 ニスノバイオ ジーティー，エルエルシー パーキンソン病を治療するための、増加した活性を有するパーキン変異体の遺伝子治療送達
CA3190720A1 (en) * 2020-08-03 2022-02-10 Prevail Therapeutics, Inc. Aav vectors encoding parkin and uses thereof
JP2023538519A (ja) 2020-08-07 2023-09-08 スペースクラフト セブン リミテッド ライアビリティ カンパニー Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療
WO2022040530A2 (en) * 2020-08-21 2022-02-24 Capsida, Inc. Virus compositions for treating mucopolysaccharidosis ii
WO2022066911A1 (en) * 2020-09-23 2022-03-31 Seelos Therapeutics, Inc. Compositions and methods for inhibiting alpha-synuclein aggregation
CN113846122B (zh) * 2021-08-16 2023-08-25 中国医学科学院医学生物学研究所 一种过表达snca的腺相关病毒载体aav-snca、制备方法及其应用
CN115029360B (zh) * 2022-05-30 2024-08-02 上海勉亦生物科技有限公司 用于治疗粘多糖贮积症iiia型的转基因表达盒
KR20250142386A (ko) * 2023-02-03 2025-09-30 얀센 파마슈티카 엔브이 파킨슨병에 사용하기 위한 유전자 요법 벡터
WO2025065011A1 (en) * 2023-09-22 2025-03-27 The Regents Of The University Of Colorado, A Body Corporate Gene therapy for diabetic retinopathy
CN119193701B (zh) * 2024-09-13 2025-07-15 暨南大学 帕金森动物模型的构建方法与特异性靶向Parkin基因的gRNA及其应用

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