LT3294323T - Adenoasocijuotas virusas, skirtas terapiniam pristatymui į centrinę nervų sistemą - Google Patents

Adenoasocijuotas virusas, skirtas terapiniam pristatymui į centrinę nervų sistemą

Info

Publication number
LT3294323T
LT3294323T LTEPPCT/US2016/032392T LT16032392T LT3294323T LT 3294323 T LT3294323 T LT 3294323T LT 16032392 T LT16032392 T LT 16032392T LT 3294323 T LT3294323 T LT 3294323T
Authority
LT
Lithuania
Prior art keywords
nervous system
central nervous
associated virus
therapeutic delivery
adeno associated
Prior art date
Application number
LTEPPCT/US2016/032392T
Other languages
English (en)
Inventor
R. Scott Mcivor
Lalitha R. BELUR
Karen Kozarsky
Original Assignee
Regenxbio Inc
Regents Of The University Of Minnesota
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=LT3294323(T) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Regenxbio Inc, Regents Of The University Of Minnesota filed Critical Regenxbio Inc
Publication of LT3294323T publication Critical patent/LT3294323T/lt

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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (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)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (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)
LTEPPCT/US2016/032392T 2015-05-15 2016-05-13 Adenoasocijuotas virusas, skirtas terapiniam pristatymui į centrinę nervų sistemą LT3294323T (lt)

Applications Claiming Priority (5)

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
PCT/US2016/032392 WO2016187017A1 (en) 2015-05-15 2016-05-13 Adeno-associated for therapeutic delivery to central nervous system

Publications (1)

Publication Number Publication Date
LT3294323T true LT3294323T (lt) 2022-02-25

Family

ID=57320277

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEPPCT/US2016/032392T LT3294323T (lt) 2015-05-15 2016-05-13 Adenoasocijuotas virusas, skirtas terapiniam pristatymui į centrinę nervų sistemą

Country Status (23)

Country Link
US (5) US20180289839A1 (lt)
EP (2) EP4000631A1 (lt)
JP (4) JP2018515615A (lt)
KR (3) KR20220082100A (lt)
CN (1) CN108367055A (lt)
AU (2) AU2016263119B2 (lt)
BR (1) BR112017024519A2 (lt)
CA (1) CA2986252A1 (lt)
CY (1) CY1125229T1 (lt)
DK (1) DK3294323T3 (lt)
ES (1) ES2901766T3 (lt)
HK (1) HK1256341A1 (lt)
HR (1) HRP20220050T1 (lt)
HU (1) HUE057795T2 (lt)
LT (1) LT3294323T (lt)
MX (2) MX2017014443A (lt)
PL (1) PL3294323T3 (lt)
PT (1) PT3294323T (lt)
RS (1) RS63927B1 (lt)
RU (1) RU2751952C2 (lt)
SI (1) SI3294323T1 (lt)
SM (1) SMT202200022T1 (lt)
WO (1) WO2016187017A1 (lt)

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JP6591956B2 (ja) 2013-03-15 2019-10-16 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Ｍｐｓ１を治療するための組成物および方法
KR20160010526A (ko) * 2013-05-15 2016-01-27 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
CA2986252A1 (en) 2015-05-15 2016-11-24 Regents Of The University Of Minnesota Adeno-associated virus vector for delivery of iduronate-2-sulfatase to the central nervous system
AU2017215211C1 (en) 2016-02-03 2023-11-16 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
AU2017250298B2 (en) * 2016-04-15 2024-10-03 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type II
IL266639B2 (en) * 2016-11-15 2024-06-01 Univ Minnesota Method for improving neurological function in mpsi and mpsii and other neurological disorders
AU2018253303B2 (en) 2017-04-14 2024-11-21 Regenxbio Inc. Treatment of mucopolysaccharidosis II with recombinant human iduronate-2 sulfatase (IDS) produced by human neural or glial cells
CN110709090A (zh) * 2017-04-27 2020-01-17 益缪索夫特公司 用于在体内递送治疗剂的b细胞及其剂量
EP3676385A1 (en) * 2017-07-06 2020-07-08 The Trustees of The University of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
JP7449223B2 (ja) * 2017-09-22 2024-03-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症ｉｉ型を治療するための遺伝子療法
BR112020006633A2 (pt) 2017-10-03 2020-10-06 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
CN112553210B (zh) 2017-10-03 2024-09-24 普利维尔治疗公司 用于溶酶体障碍的基因疗法
CA3078501A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
EA202091352A1 (ru) * 2017-11-30 2020-08-27 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Генотерапия мукополисахаридоза iiib
WO2019199841A1 (en) * 2018-04-09 2019-10-17 Cure Ahc, Inc. Aav-mediated delivery of atp1a3 genes to central nervous system
IL280198B2 (en) * 2018-07-18 2026-01-01 Regenxbio Inc Treatment of mucopolysaccharidosis I with fully glycosylated human iduronidase-alpha-L (idus)
SG11202108480UA (en) * 2019-02-26 2021-09-29 Univ Pennsylvania Compositions useful in treatment of krabbe disease
AU2020245415B2 (en) * 2019-03-22 2025-06-26 Rush University Medical Center Combination of nasal gene delivery and oral cinnamic acid, oleamide or gemfibrozil for lysosomal stoarge disorders
KR20220006527A (ko) 2019-04-10 2022-01-17 프리베일 테라퓨틱스, 인크. 리소좀 장애에 대한 유전자 요법
AU2020270421A1 (en) * 2019-05-03 2021-11-18 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
JP2022536338A (ja) * 2019-06-10 2022-08-15 ホモロジー・メディシンズ・インコーポレイテッド Ａｒｓａ遺伝子移入のためのアデノ随伴ウイルス組成物およびその使用方法
CA3155154A1 (en) * 2019-10-23 2021-04-29 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
MX2022007135A (es) 2019-12-10 2022-09-19 Takeda Pharmaceuticals Co Vectores de virus adenoasociados para el tratamiento del síndrome de hunter.
MX2023001562A (es) * 2020-08-10 2023-05-03 Prevail Therapeutics Inc Terapias genicas para trastornos lisosomales.
CN116437968A (zh) * 2020-08-10 2023-07-14 普利维尔治疗公司 用于神经退行性病症的基因疗法
WO2022121860A1 (en) * 2020-12-07 2022-06-16 East China University Of Science And Technology Methods and kits for inducing immunotolerance to gene delivering target vehicle
EP4284335A1 (en) 2021-02-01 2023-12-06 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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KR20160010526A (ko) 2013-05-15 2016-01-27 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
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CA2986252A1 (en) * 2015-05-15 2016-11-24 Regents Of The University Of Minnesota Adeno-associated virus vector for delivery of iduronate-2-sulfatase to the central nervous system
IL266639B2 (en) * 2016-11-15 2024-06-01 Univ Minnesota Method for improving neurological function in mpsi and mpsii and other neurological disorders
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

Also Published As

Publication number Publication date
BR112017024519A2 (en) 2018-07-24
SI3294323T1 (sl) 2022-06-30
EP3294323A4 (en) 2019-01-16
RU2017143640A (ru) 2019-06-17
US20220211875A1 (en) 2022-07-07
AU2021266344A1 (en) 2021-12-09
EP3294323B1 (en) 2021-10-13
RU2017143640A3 (lt) 2019-11-22
CY1125229T1 (el) 2023-06-09
JP2021167332A (ja) 2021-10-21
MX2022012524A (es) 2022-11-16
JP2024147704A (ja) 2024-10-16
EP3294323A1 (en) 2018-03-21
HUE057795T2 (hu) 2022-06-28
MX2017014443A (es) 2018-08-01
CN108367055A (zh) 2018-08-03
KR102406615B1 (ko) 2022-06-08
AU2016263119B2 (en) 2021-08-12
US20180289839A1 (en) 2018-10-11
RU2020125073A (ru) 2020-08-27
KR20180118508A (ko) 2018-10-31
US20210369871A1 (en) 2021-12-02
DK3294323T3 (da) 2022-01-10
PL3294323T3 (pl) 2022-06-20
RU2751952C2 (ru) 2021-07-21
SMT202200022T1 (it) 2022-03-21
EP4000631A1 (en) 2022-05-25
HK1252871A1 (en) 2019-06-06
JP2018515615A (ja) 2018-06-14
WO2016187017A1 (en) 2016-11-24
CA2986252A1 (en) 2016-11-24
ES2901766T3 (es) 2022-03-23
KR20220082100A (ko) 2022-06-16
AU2021266344B2 (en) 2024-08-22
US20230226225A1 (en) 2023-07-20
HRP20220050T1 (hr) 2022-10-14
HK1256341A1 (zh) 2019-09-20
JP2022137261A (ja) 2022-09-21
US20180071373A1 (en) 2018-03-15
KR20250150167A (ko) 2025-10-17
PT3294323T (pt) 2022-01-06
RS63927B1 (sr) 2023-02-28
AU2016263119A1 (en) 2018-01-18

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