MA55313A - Vecteurs d'adn non viraux et leurs utilisations pour exprimer des agents thérapeutiques du facteur viii - Google Patents

Vecteurs d'adn non viraux et leurs utilisations pour exprimer des agents thérapeutiques du facteur viii

Info

Publication number
MA55313A
MA55313A MA055313A MA55313A MA55313A MA 55313 A MA55313 A MA 55313A MA 055313 A MA055313 A MA 055313A MA 55313 A MA55313 A MA 55313A MA 55313 A MA55313 A MA 55313A
Authority
MA
Morocco
Prior art keywords
therapeutic agents
factor viii
viral dna
dna vectors
expressing factor
Prior art date
Application number
MA055313A
Other languages
English (en)
Inventor
Douglas Anthony Kerr
Debra Klatte
Phillip Samayoa
Nathaniel Silver
Original Assignee
Generation Bio Co
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 Generation Bio Co filed Critical Generation Bio Co
Publication of MA55313A publication Critical patent/MA55313A/fr

Links

Classifications

    • 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
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • 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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2820/00—Vectors comprising a special origin of replication system
    • C12N2820/60—Vectors comprising a special origin of replication system from viruses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
MA055313A 2019-03-13 2020-03-13 Vecteurs d'adn non viraux et leurs utilisations pour exprimer des agents thérapeutiques du facteur viii MA55313A (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962817904P 2019-03-13 2019-03-13
US201962856432P 2019-06-03 2019-06-03

Publications (1)

Publication Number Publication Date
MA55313A true MA55313A (fr) 2022-01-19

Family

ID=72426821

Family Applications (1)

Application Number Title Priority Date Filing Date
MA055313A MA55313A (fr) 2019-03-13 2020-03-13 Vecteurs d'adn non viraux et leurs utilisations pour exprimer des agents thérapeutiques du facteur viii

Country Status (13)

Country Link
US (2) US12338275B2 (fr)
EP (1) EP3938523A4 (fr)
JP (3) JP7795357B2 (fr)
KR (1) KR20210151785A (fr)
CN (1) CN113874513A (fr)
AU (1) AU2020234713A1 (fr)
BR (1) BR112021017853A2 (fr)
CA (1) CA3133255A1 (fr)
IL (1) IL286284A (fr)
MA (1) MA55313A (fr)
MX (1) MX2021011039A (fr)
SG (1) SG11202109850SA (fr)
WO (1) WO2020186207A2 (fr)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2020001593A (es) 2017-08-09 2020-07-13 Bioverativ Therapeutics Inc Moleculas de acido nucleico y usos de las mismas.
JP7602454B2 (ja) 2018-08-09 2024-12-18 バイオベラティブ セラピューティクス インコーポレイテッド 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
MX2023001109A (es) 2020-07-27 2023-04-24 Anjarium Biosciences Ag Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas.
US20220356490A1 (en) * 2020-08-23 2022-11-10 Bioverativ Therapeutics Inc. Baculovirus expression system
AU2022334714A1 (en) * 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Closed-end dna production with inverted terminal repeat sequences
WO2023028440A2 (fr) * 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Système d'expression de baculovirus
WO2023069948A1 (fr) 2021-10-18 2023-04-27 Flagship Pioneering Innovations Vii, Llc Compositions d'adn et procédés associés
WO2023078220A1 (fr) * 2021-11-02 2023-05-11 苏州华毅乐健生物科技有限公司 Molécule d'acide nucléique isolée et son utilisation
JP2023089774A (ja) * 2021-12-16 2023-06-28 株式会社三洋物産 遊技機
WO2023135273A2 (fr) * 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions de molécules d'adn codant pour le facteur viii, leurs procédés de préparation et leurs méthodes d'utilisation
EP4598590A4 (fr) * 2022-10-08 2026-05-27 Lingyi Biotech Co Ltd Construction pour améliorer l'expression génique
WO2025110984A1 (fr) * 2023-11-21 2025-05-30 Hydrogene Therapeutics, Inc. Compositions et méthodes pour le traitement de la maladie de wilson
KR20260011950A (ko) 2024-07-17 2026-01-26 이민형 수질 시료 채취 드론

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2220222A4 (fr) 2007-11-19 2011-10-12 Univ California Nouvelle analyse pour des inhibiteurs d'egfr
US20120263743A1 (en) * 2009-04-30 2012-10-18 Cytos Biotechnology Ag Influenza hemagglutinin compositions and uses thereof
EP2500434A1 (fr) * 2011-03-12 2012-09-19 Association Institut de Myologie Vecteurs AAV sans capside, compositions et procédés pour la production des vecteurs et la thérapie génique
AU2012340501A1 (en) * 2011-12-19 2013-07-11 Grifols, S.A. Compositions, methods, and kits for preparing sialylated recombinant proteins
WO2013151664A1 (fr) * 2012-04-02 2013-10-10 modeRNA Therapeutics Polynucléotides modifiés pour la production de protéines
RS61387B2 (sr) * 2013-02-15 2024-06-28 Bioverativ Therapeutics Inc Gen optimizovanog faktora viii
ES2813698T3 (es) * 2013-09-12 2021-03-24 Biomarin Pharm Inc Vectores de AAV que comprenden un gen que codifica el factor VIII
GB2526339A (en) 2014-05-21 2015-11-25 Imp Innovations Ltd Lentiviral vectors
WO2017021359A1 (fr) 2015-08-03 2017-02-09 Myodopa Limited Synthèse et régulation systémiques de la l-dopa
JP2018535929A (ja) 2015-09-24 2018-12-06 バイオマリン ファーマシューティカル インコーポレイテッド アデノ随伴ウイルス第ｖｉｉｉ因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤
US10143760B2 (en) * 2015-10-28 2018-12-04 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes and methods of use thereof
PE20231949A1 (es) * 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
IL261524B2 (en) * 2016-03-03 2023-10-01 Univ Massachusetts End-capped linear double-stranded DNA for non-viral gene transfer
PE20181922A1 (es) * 2016-04-15 2018-12-11 Univ Pennsylvania Terapia genica para tratar la hemofilia a
KR102219859B1 (ko) * 2016-06-24 2021-02-25 재단법인 목암생명과학연구소 재조합 단쇄 fviii 및 그 화학 접합물
US20200237930A1 (en) 2017-08-01 2020-07-30 Spark Therapeutics, Inc. Factor viii (fviii) gene therapy methods
MX2020001593A (es) * 2017-08-09 2020-07-13 Bioverativ Therapeutics Inc Moleculas de acido nucleico y usos de las mismas.

Also Published As

Publication number Publication date
WO2020186207A8 (fr) 2020-11-12
AU2020234713A1 (en) 2021-11-04
KR20210151785A (ko) 2021-12-14
US20220177545A1 (en) 2022-06-09
IL286284A (en) 2021-10-31
MX2021011039A (es) 2021-12-15
CN113874513A (zh) 2021-12-31
JP2025073123A (ja) 2025-05-12
US20250282848A1 (en) 2025-09-11
US12338275B2 (en) 2025-06-24
WO2020186207A2 (fr) 2020-09-17
JP2026002894A (ja) 2026-01-08
BR112021017853A2 (pt) 2021-11-30
CA3133255A1 (fr) 2020-09-17
WO2020186207A3 (fr) 2020-12-17
EP3938523A4 (fr) 2023-06-21
JP7795357B2 (ja) 2026-01-07
EP3938523A2 (fr) 2022-01-19
SG11202109850SA (en) 2021-10-28
JP2022524434A (ja) 2022-05-02

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