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HK1252871A1 - 用於治療傳遞給中樞神經系統的腺相關病毒 - Google Patents

用於治療傳遞給中樞神經系統的腺相關病毒

Info

Publication number
HK1252871A1
HK1252871A1 HK18112215.4A HK18112215A HK1252871A1 HK 1252871 A1 HK1252871 A1 HK 1252871A1 HK 18112215 A HK18112215 A HK 18112215A HK 1252871 A1 HK1252871 A1 HK 1252871A1
Authority
HK
Hong Kong
Prior art keywords
adeno
nervous system
central nervous
therapeutic delivery
therapeutic
Prior art date
Application number
HK18112215.4A
Other languages
English (en)
Inventor
R Scott Mcivor
Lalitha R Belur
Karen Kozarsky
Original Assignee
R Scott Mcivor
Lalitha R Belur
Karen Kozarsky
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=57320277&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HK1252871(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by R Scott Mcivor, Lalitha R Belur, Karen Kozarsky filed Critical R Scott Mcivor
Publication of HK1252871A1 publication Critical patent/HK1252871A1/zh

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
    • A61K48/0075—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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/66—Phosphorus compounds
    • A61K31/675—Phosphorus compounds having nitrogen as a ring hetero atom, e.g. pyridoxal phosphate
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K48/0083—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 administration regime
    • 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
    • A61K9/0043—Nose
    • 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
    • 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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6806—Preparing nucleic acids for analysis, e.g. for polymerase chain reaction [PCR] assay
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Analytical Chemistry (AREA)
  • Otolaryngology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
HK18112215.4A 2015-05-15 2018-09-21 用於治療傳遞給中樞神經系統的腺相關病毒 HK1252871A1 (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562162174P 2015-05-15 2015-05-15
US201562252055P 2015-11-06 2015-11-06
US201662301980P 2016-03-01 2016-03-01
US201662331156P 2016-05-03 2016-05-03

Publications (1)

Publication Number Publication Date
HK1252871A1 true HK1252871A1 (zh) 2019-06-06

Family

ID=57320277

Family Applications (2)

Application Number Title Priority Date Filing Date
HK18112215.4A HK1252871A1 (zh) 2015-05-15 2018-09-21 用於治療傳遞給中樞神經系統的腺相關病毒
HK18115428.0A HK1256341A1 (zh) 2015-05-15 2018-12-03 用於治療性遞送到中樞神經系統的腺相關物

Family Applications After (1)

Application Number Title Priority Date Filing Date
HK18115428.0A HK1256341A1 (zh) 2015-05-15 2018-12-03 用於治療性遞送到中樞神經系統的腺相關物

Country Status (22)

Country Link
US (5) US20180289839A1 (zh)
EP (2) EP4000631A1 (zh)
JP (4) JP2018515615A (zh)
KR (2) KR102406615B1 (zh)
CN (1) CN108367055A (zh)
AU (2) AU2016263119B2 (zh)
BR (1) BR112017024519A2 (zh)
CA (1) CA2986252A1 (zh)
CY (1) CY1125229T1 (zh)
DK (1) DK3294323T3 (zh)
ES (1) ES2901766T3 (zh)
HK (2) HK1252871A1 (zh)
HR (1) HRP20220050T1 (zh)
HU (1) HUE057795T2 (zh)
LT (1) LT3294323T (zh)
MX (2) MX2017014443A (zh)
PL (1) PL3294323T3 (zh)
PT (1) PT3294323T (zh)
RS (1) RS63927B1 (zh)
RU (1) RU2751952C2 (zh)
SI (1) SI3294323T1 (zh)
WO (1) WO2016187017A1 (zh)

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US12121567B2 (en) 2013-05-15 2024-10-22 Regents Of The University Of Minnesota Methods to treat mucopolysaccharidosis type II or deficiency in iduronate-2-sulfatase using a recombinant adeno-associated virus (AAV) vector encoding iduronate-2-sulfatase

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GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
EP4000631A1 (en) * 2015-05-15 2022-05-25 REGENXBIO Inc. Adeno-associated for therapeutic delivery to central nervous system
IL260959B2 (en) 2016-02-03 2024-09-01 Univ Pennsylvania Gene therapy for the treatment of mucoid polysaccharomyces type I
KR20190008237A (ko) * 2016-04-15 2019-01-23 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
EP3541946A1 (en) * 2016-11-15 2019-09-25 Regents Of The University Of Minnesota Method for improving neurological function in mpsi and mpsii and other neurological disorders
BR112019021569A2 (pt) * 2017-04-14 2020-05-12 Regenxbio Inc. Precursor de iduronato-2-sulfatase humana recombinante glicosilada (ids), método para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo ii (mps ii)
JP2020517692A (ja) * 2017-04-27 2020-06-18 イミュソフト コーポレーション 治療剤のｉｎ ｖｉｖｏ送達のためのＢ細胞およびその投薬量
SG11201912631PA (en) * 2017-07-06 2020-01-30 Univ Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
WO2019060662A1 (en) * 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSE TYPE II
BR112020006671A2 (pt) 2017-10-03 2020-12-01 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
KR102693318B1 (ko) 2017-10-03 2024-08-07 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
EP3692158A4 (en) 2017-10-03 2021-08-04 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMIAL DISORDERS
AU2018375163A1 (en) * 2017-11-30 2020-06-11 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis IIIB
US11738093B2 (en) 2018-04-09 2023-08-29 Hope For Annabel AAV-mediated delivery of ATP1A3 genes to central nervous system
EP3823590A4 (en) * 2018-07-18 2022-04-20 REGENXBIO Inc. TREATMENT OF MUCOPOLYSACCHARIDOSIS I WITH ALL-HUMAN GLYCOSYLATED ALPHA-L-IDURONIDASE (IDUS)
KR20220006527A (ko) 2019-04-10 2022-01-17 프리베일 테라퓨틱스, 인크. 리소좀 장애에 대한 유전자 요법
CA3135539A1 (en) * 2019-05-03 2020-11-12 Juliette HORDEAUX Compositions useful in treatment of metachromatic leukodystrophy
KR20220035107A (ko) * 2019-06-10 2022-03-21 호몰로지 메디슨, 인크. Arsa 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법
EP4072595A1 (en) 2019-12-10 2022-10-19 Takeda Pharmaceutical Company Limited Adeno associated virus vectors for the treatment of hunter disease
MX2023001562A (es) * 2020-08-10 2023-05-03 Prevail Therapeutics Inc Terapias genicas para trastornos lisosomales.
CN117642173A (zh) * 2020-12-07 2024-03-01 华东理工大学 用于诱导对基因递送靶媒剂的免疫耐受性的方法和试剂盒
AU2022214429A1 (en) 2021-02-01 2023-09-14 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
BR112023016075A2 (pt) 2021-02-10 2023-11-21 Regenxbio Inc Método para tratar hepatoesplenomegalia, método de distribuição de um raav que codifica hids, método para tratar um sujeito humano, método de tratamento de um sujeito humano, método para determinar a eficácia do tratamento, método para identificar um sujeito como tendo mps ii
WO2022212616A1 (en) * 2021-04-01 2022-10-06 The University Of North Carolina At Chapel Hill Aav-ids vectors for treatment of mucopolysaccharidosis ii

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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12121567B2 (en) 2013-05-15 2024-10-22 Regents Of The University Of Minnesota Methods to treat mucopolysaccharidosis type II or deficiency in iduronate-2-sulfatase using a recombinant adeno-associated virus (AAV) vector encoding iduronate-2-sulfatase

Also Published As

Publication number Publication date
EP3294323A1 (en) 2018-03-21
JP2024147704A (ja) 2024-10-16
KR20220082100A (ko) 2022-06-16
AU2016263119A1 (en) 2018-01-18
RU2017143640A (ru) 2019-06-17
MX2017014443A (es) 2018-08-01
RU2020125073A (ru) 2020-08-27
RU2751952C2 (ru) 2021-07-21
EP4000631A1 (en) 2022-05-25
AU2021266344A1 (en) 2021-12-09
RU2017143640A3 (zh) 2019-11-22
MX2022012524A (es) 2022-11-16
CN108367055A (zh) 2018-08-03
CA2986252A1 (en) 2016-11-24
JP2021167332A (ja) 2021-10-21
HRP20220050T1 (hr) 2022-10-14
DK3294323T3 (da) 2022-01-10
SI3294323T1 (sl) 2022-06-30
US20230226225A1 (en) 2023-07-20
AU2016263119B2 (en) 2021-08-12
HUE057795T2 (hu) 2022-06-28
KR20180118508A (ko) 2018-10-31
US20210369871A1 (en) 2021-12-02
AU2021266344B2 (en) 2024-08-22
LT3294323T (lt) 2022-02-25
EP3294323B1 (en) 2021-10-13
EP3294323A4 (en) 2019-01-16
HK1256341A1 (zh) 2019-09-20
PT3294323T (pt) 2022-01-06
JP2018515615A (ja) 2018-06-14
US20180071373A1 (en) 2018-03-15
RS63927B1 (sr) 2023-02-28
CY1125229T1 (el) 2023-06-09
BR112017024519A2 (pt) 2018-07-24
JP2022137261A (ja) 2022-09-21
WO2016187017A1 (en) 2016-11-24
KR102406615B1 (ko) 2022-06-08
US20220211875A1 (en) 2022-07-07
US20180289839A1 (en) 2018-10-11
ES2901766T3 (es) 2022-03-23
PL3294323T3 (pl) 2022-06-20

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