IL287608A - Intrathecal and intravenous combination gene therapy for the treatment of infantile batten disease - Google Patents

Intrathecal and intravenous combination gene therapy for the treatment of infantile batten disease

Info

Publication number
IL287608A
IL287608A IL287608A IL28760821A IL287608A IL 287608 A IL287608 A IL 287608A IL 287608 A IL287608 A IL 287608A IL 28760821 A IL28760821 A IL 28760821A IL 287608 A IL287608 A IL 287608A
Authority
IL
Israel
Prior art keywords
intraspinal
treatment
combined
gene therapies
batten disease
Prior art date
Application number
IL287608A
Other languages
English (en)
Hebrew (he)
Inventor
Timothy J Miller
Original Assignee
Univ North Carolina Chapel Hill
Timothy J Miller
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 Univ North Carolina Chapel Hill, Timothy J Miller filed Critical Univ North Carolina Chapel Hill
Publication of IL287608A publication Critical patent/IL287608A/en

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
    • A61K48/005—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
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61P25/08—Antiepileptics; Anticonvulsants
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
    • 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
    • C12N2750/14123—Virus like particles [VLP]
    • 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)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pain & Pain Management (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Dermatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
IL287608A 2019-04-29 2021-10-27 Intrathecal and intravenous combination gene therapy for the treatment of infantile batten disease IL287608A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962840360P 2019-04-29 2019-04-29
PCT/US2020/030427 WO2020223322A1 (en) 2019-04-29 2020-04-29 Intrathecal and intravenous combination gene therapy for the treatment of infantile batten disease

Publications (1)

Publication Number Publication Date
IL287608A true IL287608A (en) 2021-12-01

Family

ID=73029200

Family Applications (1)

Application Number Title Priority Date Filing Date
IL287608A IL287608A (en) 2019-04-29 2021-10-27 Intrathecal and intravenous combination gene therapy for the treatment of infantile batten disease

Country Status (12)

Country Link
US (1) US20220193268A1 (enExample)
EP (1) EP3963081A4 (enExample)
JP (1) JP7788594B2 (enExample)
KR (1) KR20220046513A (enExample)
CN (1) CN114269935A (enExample)
AU (1) AU2020264438A1 (enExample)
BR (1) BR112021021632A8 (enExample)
CA (1) CA3138274A1 (enExample)
IL (1) IL287608A (enExample)
MX (1) MX2021013275A (enExample)
SG (1) SG11202111908XA (enExample)
WO (1) WO2020223322A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4551695A1 (en) * 2022-07-06 2025-05-14 Research Institute at Nationwide Children's Hospital Adeno-associated virus delivery of cln1 polynucleotide

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20130010121A (ko) * 2010-03-23 2013-01-25 인트렉손 코포레이션 치료적 단백질을 조건부로 발현하는 벡터，상기 벡터를 포함하는 숙주 세포 및 이의 용도
AU2015364636B9 (en) * 2014-12-16 2021-12-02 Board Of Regents Of The University Of Nebraska Gene therapy for Juvenile Batten Disease
US11504435B2 (en) * 2016-06-13 2022-11-22 The University Of North Carolina At Chapel Hill Optimized CLN1 genes and expression cassettes and their use
CN111065741A (zh) * 2017-07-08 2020-04-24 吉尼松公司 脊髓性肌萎缩症的治疗
US20210115453A1 (en) * 2017-08-31 2021-04-22 Poseida Therapeutics, Inc. Transposon system and methods of use

Also Published As

Publication number Publication date
SG11202111908XA (en) 2021-11-29
KR20220046513A (ko) 2022-04-14
BR112021021632A8 (pt) 2022-06-28
JP7788594B2 (ja) 2025-12-19
EP3963081A4 (en) 2023-07-26
AU2020264438A1 (en) 2021-12-16
BR112021021632A2 (enExample) 2021-12-21
WO2020223322A1 (en) 2020-11-05
CA3138274A1 (en) 2020-11-05
MX2021013275A (es) 2022-03-17
EP3963081A1 (en) 2022-03-09
JP2022530264A (ja) 2022-06-28
CN114269935A (zh) 2022-04-01
US20220193268A1 (en) 2022-06-23

Similar Documents

Publication Publication Date Title
IL285072A (en) Compositions and methods for treating, preventing or reversing age-associated inflammation and disorders
IL279918A (en) Gene therapy vectors for treatment of danon disease
EP3873335A4 (en) CHARACTERIZATION OF THERAPEUTIC EFFECTIVENESS OF NEUROSTIMULATION
IL285238A (en) Gene therapy vectors for treatment of danon disease
PL3673080T3 (pl) Oligomery antysensowne do leczenia stanów i chorób
EP3960766A4 (en) ANTI-TUMOR THERAPEUTIC AGENT AND USE THEREOF
EP4041212C0 (en) MEK INHIBITORS FOR THE TREATMENT OF HANTAVIRUS INFECTIONS
EP3880235A4 (en) Gene therapy for neuronal ceroid lipofuscinoses
PL3658140T3 (pl) Połączenia aprocitentanu z dodatkowymi składnikami czynnymi do leczenia opornego nadciśnienia
EP3802557A4 (en) THERAPEUTIC TREATMENT OF MICROSATELLITE-UNSTABLE CANCER
IL287904A (en) Combination treatment of arthritic disease
IL281244A (en) Combination therapy for the treatment of liver disease
EP4051324A4 (en) Gene therapy vectors
EP3641888A4 (en) PLASMINOGEN TREATMENT OF DISEASES RELATED TO PAI-1 OVEREXPRESSION
EP3968991C0 (en) PROTEIN KINASE INHIBITORS AND THEIR USES FOR THE TREATMENT OF DISEASES AND HEALTH PROBLEMS
PT3990005T (pt) Cistationina beta-sintetase peguilada para terapia enzimática para tratamento de homocistinúria
IL310870A (en) Quinazoline compounds for treatment of disease
DK3419672T3 (da) Genterapi til behandling af retinal degenerationssygdom
SG11202107659SA (en) New recombinant diamine oxidase and its use for the treatment of diseases characterized by excess histamine
IL257876A (en) Co-therapy comprising canagliflozin and phentermine for the treatment of obesity and obesity related disorders
EP4028123A4 (en) Delivery of therapeutic neuromodulation
IL280395A (en) Tetrahydroquinolino derivatives for the treatment of metastatic and chemoresistant cancers
EP3923963A4 (en) TREATMENT OF HEART FAILURE
IL289251A (en) Methods and compositions for treatment of pancreatic cancer
IL287608A (en) Intrathecal and intravenous combination gene therapy for the treatment of infantile batten disease