MA55625A - Compositions et méthodes d'administration de produits thérapeutiques - Google Patents

Compositions et méthodes d'administration de produits thérapeutiques

Info

Publication number
MA55625A
MA55625A MA055625A MA55625A MA55625A MA 55625 A MA55625 A MA 55625A MA 055625 A MA055625 A MA 055625A MA 55625 A MA55625 A MA 55625A MA 55625 A MA55625 A MA 55625A
Authority
MA
Morocco
Prior art keywords
administration
compositions
methods
therapeutic products
therapeutic
Prior art date
Application number
MA055625A
Other languages
English (en)
Inventor
Archana Belle
Stephanie Tagliatela
Original Assignee
Encoded Therapeutics Inc
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 Encoded Therapeutics Inc filed Critical Encoded Therapeutics Inc
Publication of MA55625A publication Critical patent/MA55625A/fr

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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A61K48/005—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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • 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
    • A61P25/28—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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • Virology (AREA)
  • Dermatology (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
MA055625A 2019-04-12 2020-04-10 Compositions et méthodes d'administration de produits thérapeutiques MA55625A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962833447P 2019-04-12 2019-04-12

Publications (1)

Publication Number Publication Date
MA55625A true MA55625A (fr) 2022-02-16

Family

ID=72750879

Family Applications (1)

Application Number Title Priority Date Filing Date
MA055625A MA55625A (fr) 2019-04-12 2020-04-10 Compositions et méthodes d'administration de produits thérapeutiques

Country Status (17)

Country Link
US (1) US20220168449A1 (fr)
EP (1) EP3952924A4 (fr)
JP (1) JP2022526425A (fr)
KR (1) KR20220007601A (fr)
CN (1) CN114430684A (fr)
AU (1) AU2020272980A1 (fr)
BR (1) BR112021020421A2 (fr)
CA (1) CA3136646A1 (fr)
CL (1) CL2021002635A1 (fr)
CO (1) CO2021013548A2 (fr)
EA (1) EA202192801A1 (fr)
IL (1) IL287137A (fr)
MA (1) MA55625A (fr)
MX (1) MX2021012527A (fr)
SG (1) SG11202111195VA (fr)
TW (1) TW202104596A (fr)
WO (1) WO2020210633A1 (fr)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11149256B2 (en) 2018-09-26 2021-10-19 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
WO2022174000A2 (fr) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Compositions d'arni de superoxyde dismutase 1 (sod1) et procédés d'utilisation correspondants pour traiter ou prévenir des maladies neurodégénératives associées à la superoxyde dismutase 1 (sod1)

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007044627A2 (fr) * 2005-10-06 2007-04-19 Sloan Kettering Institute For Cancer Research Compositions et methodes d'administration d'arn interferant
CN101460621B (zh) * 2006-06-07 2016-01-27 建新公司 肌萎缩性侧索硬化和其他脊髓失调症的基因治疗
ES2635726T3 (es) * 2007-06-06 2017-10-04 Genzyme Corporation Terapia génica para enfermedades de almacenamiento lisosómico
WO2010129021A1 (fr) * 2009-05-02 2010-11-11 Genzyme Corporation Thérapie génique pour les maladies neurodégénératives
MX2016001026A (es) * 2013-07-26 2016-08-03 Univ Iowa Res Found Metodos y composiciones para tratar enfermedades del cerebro.
CN112375760A (zh) * 2014-11-14 2021-02-19 沃雅戈治疗公司 调节性多核苷酸
IL266862B2 (en) * 2016-12-01 2024-01-01 Sangamo Therapeutics Inc Tau modulators and methods and preparations for their administration
US20200040061A1 (en) * 2017-02-22 2020-02-06 Crispr Therapeutics Ag Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders
WO2018209205A1 (fr) * 2017-05-11 2018-11-15 The Trustees Of The University Of Pennsylvania Thérapie génique de céroïdes-lipofuscinoses neuronales
TWI811219B (zh) * 2017-05-19 2023-08-11 美商編碼製藥公司 高活性調控元件
KR20220066225A (ko) * 2019-05-29 2022-05-24 엔코디드 테라퓨틱스, 인크. 선택적 유전자 조절을 위한 조성물 및 방법

Also Published As

Publication number Publication date
JP2022526425A (ja) 2022-05-24
TW202104596A (zh) 2021-02-01
AU2020272980A1 (en) 2021-11-04
WO2020210633A8 (fr) 2021-09-30
US20220168449A1 (en) 2022-06-02
EA202192801A1 (ru) 2022-02-24
CN114430684A (zh) 2022-05-03
KR20220007601A (ko) 2022-01-18
CA3136646A1 (fr) 2020-10-15
SG11202111195VA (en) 2021-11-29
CL2021002635A1 (es) 2022-07-15
BR112021020421A2 (pt) 2021-12-21
CO2021013548A2 (es) 2022-01-28
EP3952924A4 (fr) 2023-05-24
EP3952924A1 (fr) 2022-02-16
IL287137A (en) 2021-12-01
MX2021012527A (es) 2022-01-06
WO2020210633A1 (fr) 2020-10-15

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