AU2017227803B2 - Therapy for frontotemporal dementia - Google Patents

Therapy for frontotemporal dementia Download PDF

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Publication number
AU2017227803B2
AU2017227803B2 AU2017227803A AU2017227803A AU2017227803B2 AU 2017227803 B2 AU2017227803 B2 AU 2017227803B2 AU 2017227803 A AU2017227803 A AU 2017227803A AU 2017227803 A AU2017227803 A AU 2017227803A AU 2017227803 B2 AU2017227803 B2 AU 2017227803B2
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AU
Australia
Prior art keywords
mammal
grn
aav
progranulin
raav
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AU2017227803A
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English (en)
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AU2017227803A1 (en
Inventor
Defne AMADO
Alice CHEN PLOTKIN
Beverly L. Davidson
Julianne REIDERS
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Childrens Hospital of Philadelphia CHOP
University of Pennsylvania Penn
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Childrens Hospital of Philadelphia CHOP
University of Pennsylvania Penn
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Publication of AU2017227803A1 publication Critical patent/AU2017227803A1/en
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    • 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
    • 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
    • A61K48/0075—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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C07K14/4711—Alzheimer's disease; Amyloid plaque core protein
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Organic Chemistry (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Zoology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
AU2017227803A 2016-03-02 2017-03-02 Therapy for frontotemporal dementia Active AU2017227803B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662302525P 2016-03-02 2016-03-02
US62/302,525 2016-03-02
PCT/US2017/020397 WO2017151884A1 (en) 2016-03-02 2017-03-02 Therapy for frontotemporal dementia

Publications (2)

Publication Number Publication Date
AU2017227803A1 AU2017227803A1 (en) 2018-10-25
AU2017227803B2 true AU2017227803B2 (en) 2024-05-02

Family

ID=59743281

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2017227803A Active AU2017227803B2 (en) 2016-03-02 2017-03-02 Therapy for frontotemporal dementia

Country Status (6)

Country Link
US (1) US20190328906A1 (enExample)
EP (1) EP3423109A4 (enExample)
JP (2) JP7436089B2 (enExample)
AU (1) AU2017227803B2 (enExample)
CA (1) CA3016314A1 (enExample)
WO (1) WO2017151884A1 (enExample)

Families Citing this family (28)

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CN107106689A (zh) 2014-11-05 2017-08-29 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
US20190328906A1 (en) * 2016-03-02 2019-10-31 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
US20240002459A1 (en) * 2016-07-14 2024-01-04 Emory University Granulin Compositions and Uses Related Thereto
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
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
BR112020006633A2 (pt) 2017-10-03 2020-10-06 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
CN111819281B (zh) * 2017-10-23 2024-12-31 普利维尔治疗公司 用于神经变性疾病的基因疗法
JP7401432B2 (ja) * 2017-12-01 2023-12-19 エンコーデッド セラピューティクス， インコーポレイテッド 操作されたｄｎａ結合タンパク質
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
CN113383010B (zh) 2018-09-28 2025-09-09 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
CN113164627B (zh) * 2018-10-16 2024-10-29 戴纳立制药公司 治疗和监测颗粒体蛋白前体相关病症的方法
EP3917539A4 (en) * 2019-02-01 2022-11-23 Avrobio, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF NEUROCOGNITIVE DISORDERS
MX2021010134A (es) * 2019-02-22 2021-09-23 Univ Pennsylvania Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn.
US11999974B2 (en) 2019-04-10 2024-06-04 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
KR20220006527A (ko) 2019-04-10 2022-01-17 프리베일 테라퓨틱스, 인크. 리소좀 장애에 대한 유전자 요법
WO2020243651A1 (en) 2019-05-29 2020-12-03 Encoded Therapeutics, Inc. Compositions and methods for selective gene regulation
GB201913974D0 (en) 2019-09-27 2019-11-13 King S College London Vector
MX2022004812A (es) * 2019-10-22 2023-02-23 Applied Genetic Tech Corporation Sistemas de virus adeno-asociados (aav) para el tratamiento de enfermedades o trastornos neurodegenerativos asociados a la progranulina.
EP4146797A1 (en) * 2020-05-06 2023-03-15 Orchard Therapeutics (Europe) Limited Treatment for neurodegenerative diseases
US20230227847A1 (en) * 2020-05-27 2023-07-20 Universität Zürich Viral vectors expressing therapeutic proteins specifically in myeloid cells and microglia
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
PH12023550145A1 (en) * 2020-08-12 2024-03-18 UCB Biopharma SRL Gene therapy using nucleic acid constructs comprising methyl cpg binding protein 2 (mecp2) promoter sequences
AR123358A1 (es) * 2020-08-26 2022-11-23 Univ Pennsylvania Virus adenoasociado recombinante para el tratamiento de la neurodegeneración de inicio adulto asociada a la grn
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2024035649A1 (en) * 2022-08-08 2024-02-15 Shape Therapeutics Inc. Compositions for progranulin expression and methods of use thereof
GB202216168D0 (en) 2022-10-31 2022-12-14 UCB Biopharma SRL Route of administration

Citations (4)

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WO2007146046A2 (en) * 2006-06-07 2007-12-21 Genzyme Corporation Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
WO2010129021A1 (en) * 2009-05-02 2010-11-11 Genzyme Corporation Gene therapy for neurodegenerative disorders
US20100324127A1 (en) * 2008-01-16 2010-12-23 Kay Denis G Treating neurodegenerative diseases with progranulin
US20110203007A1 (en) * 2010-02-16 2011-08-18 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Assays of neurodegenerative disorders, including frontotemporal dementia and amyotrophic lateral sclerosis

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190328906A1 (en) 2016-03-02 2019-10-31 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007146046A2 (en) * 2006-06-07 2007-12-21 Genzyme Corporation Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
US20100324127A1 (en) * 2008-01-16 2010-12-23 Kay Denis G Treating neurodegenerative diseases with progranulin
WO2010129021A1 (en) * 2009-05-02 2010-11-11 Genzyme Corporation Gene therapy for neurodegenerative disorders
US20110203007A1 (en) * 2010-02-16 2011-08-18 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Assays of neurodegenerative disorders, including frontotemporal dementia and amyotrophic lateral sclerosis

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
De Muynck, L. & Van Damme, P., Frontiers of Clinical Drug research - Alzheimer Disorders, (2015), vol. 3, pages 231-291, doi: 10.2174/97816810806801150301 *

Also Published As

Publication number Publication date
JP2019511570A (ja) 2019-04-25
JP7507808B2 (ja) 2024-06-28
JP2022071067A (ja) 2022-05-13
EP3423109A4 (en) 2019-08-14
CA3016314A1 (en) 2017-09-08
AU2017227803A1 (en) 2018-10-25
EP3423109A1 (en) 2019-01-09
US20190328906A1 (en) 2019-10-31
JP7436089B2 (ja) 2024-02-21
WO2017151884A1 (en) 2017-09-08

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