MX2022005916A - Virus adenoasociado terapeutico que comprende promotores especificos del higado para el tratamiento de la enfermedad de pompe y los trastornos liposomales. - Google Patents

Virus adenoasociado terapeutico que comprende promotores especificos del higado para el tratamiento de la enfermedad de pompe y los trastornos liposomales.

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Publication number
MX2022005916A
MX2022005916A MX2022005916A MX2022005916A MX2022005916A MX 2022005916 A MX2022005916 A MX 2022005916A MX 2022005916 A MX2022005916 A MX 2022005916A MX 2022005916 A MX2022005916 A MX 2022005916A MX 2022005916 A MX2022005916 A MX 2022005916A
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MX
Mexico
Prior art keywords
liver
gaa
polypeptide
disease
igf2
Prior art date
Application number
MX2022005916A
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English (en)
Inventor
Anna Tretiakova
Michael Roberts
Juan Manuel Iglesias
Michael W O´Callaghan
Achille Francois
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Asklepios Biopharmaceutical 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.)
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Publication date
Application filed by Asklepios Biopharmaceutical Inc filed Critical Asklepios Biopharmaceutical Inc
Publication of MX2022005916A publication Critical patent/MX2022005916A/es

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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405—Glucanases
    • C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
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    • 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
    • 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
    • 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
    • C07K14/475—Growth factors; Growth regulators
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    • 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
    • C07K14/575—Hormones
    • C07K14/62—Insulins
    • 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
    • C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
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    • 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)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
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    • 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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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/80—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates
    • C12N2810/85—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian
    • C12N2810/851—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian from growth factors; from growth regulators
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/38—Vector systems having a special element relevant for transcription being a stuffer
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Diabetes (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Endocrinology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Vectores AAV recombinantes (rAAV) que comprenden un genoma rAVV que comprende un ácido nucleico heterólogo que codifica una proteína lisosomal, por ejemplo, el polipéptido alfa-glucosidasa ácida (GAA), y opcionalmente un péptido de señal y/u opcionalmente una secuencia de dirección, por ejemplo, el péptido de dirección IGF2, operativamente enlazado a un promotor específico del hígado (LSP), lo que permite al polipéptido GAA ser secretado del hígado y dirigido a los lisosomas. Las modalidades particulares se relacionan a un vector AAV recombinante (rAAV) que codifica un polipéptido alfa-glucosidasa (GAA), que tiene un péptido de señal secretorio del hígado y un péptido de dirección IGF2 que enlaza el receptor de manosa-6-fosfato independiente de catión (CI-MPR) humano o al receptor IGF2, que permite la localización subcelular apropiada del polipéptido GAA a los lisosomas. También se abarcan células, y métodos para tratar una enfermedad lisosomal, por ejemplo una enfermedad por almacenamiento de glucógeno tipo II (GSD-II) y/o la Enfermedad de Pompe con el vector rAAV.
MX2022005916A 2019-11-19 2020-11-19 Virus adenoasociado terapeutico que comprende promotores especificos del higado para el tratamiento de la enfermedad de pompe y los trastornos liposomales. MX2022005916A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962937583P 2019-11-19 2019-11-19
US201962937556P 2019-11-19 2019-11-19
US202063023570P 2020-05-12 2020-05-12
PCT/US2020/061223 WO2021102107A1 (en) 2019-11-19 2020-11-19 Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders

Publications (1)

Publication Number Publication Date
MX2022005916A true MX2022005916A (es) 2022-08-04

Family

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Application Number Title Priority Date Filing Date
MX2022005916A MX2022005916A (es) 2019-11-19 2020-11-19 Virus adenoasociado terapeutico que comprende promotores especificos del higado para el tratamiento de la enfermedad de pompe y los trastornos liposomales.

Country Status (11)

Country Link
US (1) US20230038520A1 (es)
EP (1) EP4061946A4 (es)
JP (1) JP2023503046A (es)
KR (1) KR20220098384A (es)
CN (1) CN116096895A (es)
AU (1) AU2020388634A1 (es)
CA (1) CA3159018A1 (es)
IL (1) IL293068A (es)
MX (1) MX2022005916A (es)
TW (1) TW202132570A (es)
WO (1) WO2021102107A1 (es)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20200042935A (ko) * 2017-08-25 2020-04-24 오비드 테라퓨틱스 인크. 재조합 아데노-관련된 벡터들
WO2020102645A1 (en) * 2018-11-16 2020-05-22 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease
WO2023288301A1 (en) * 2021-07-15 2023-01-19 Cornell University Engineered biomolecules for nutrient reprogramming
WO2023139496A1 (en) * 2022-01-21 2023-07-27 Pfizer Inc. Gene therapy for gaucher disease
WO2023164060A2 (en) * 2022-02-25 2023-08-31 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease with long term cessation of gaa enzyme replacement therapy
WO2023172975A1 (en) * 2022-03-08 2023-09-14 University Of Massachusetts Methods of raav packaging
TW202403048A (zh) * 2022-03-18 2024-01-16 美商艾司科皮歐斯生物製藥有限公司 用於治療龐貝症之具有訊號肽修飾之以密碼子最佳化核酸編碼α-葡萄糖苷酶(GAA)之治療性腺相關病毒
WO2024055272A1 (zh) * 2022-09-16 2024-03-21 复旦大学附属中山医院 能高效表达目的基因的mRNA载体系统、其构建及应用

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE521701T1 (de) * 2003-01-22 2011-09-15 Univ Duke Verbesserte konstrukte zur expression lysosomaler polypeptide
US7521211B2 (en) * 2005-04-05 2009-04-21 Regeneron Pharmaceuticals, Inc IGF-1 and IGF-2 chimeric polypeptides and therapeutic uses thereof
CA2687141C (en) * 2007-05-22 2014-04-01 Amgen Inc. Compositions and methods for producing bioactive fusion proteins
WO2012145644A1 (en) * 2011-04-22 2012-10-26 Genzyme Corporation Modified acid alpha glucosidase with accelerated processing
WO2017100671A1 (en) * 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
CN111936621A (zh) * 2018-02-05 2020-11-13 奥登茨治疗公司 转录调控元件和它的用途
CN112040985A (zh) * 2018-02-07 2020-12-04 瑞泽恩制药公司 用于递送治疗性蛋白质的方法和组合物
BR112020021962A2 (pt) * 2018-04-30 2021-01-26 Amicus Therapeutics, Inc. construtos para terapia gênica e métodos de uso
WO2020102645A1 (en) * 2018-11-16 2020-05-22 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease

Also Published As

Publication number Publication date
US20230038520A1 (en) 2023-02-09
CN116096895A (zh) 2023-05-09
WO2021102107A1 (en) 2021-05-27
EP4061946A4 (en) 2024-03-06
TW202132570A (zh) 2021-09-01
IL293068A (en) 2022-07-01
JP2023503046A (ja) 2023-01-26
AU2020388634A1 (en) 2022-06-23
CA3159018A1 (en) 2021-05-27
KR20220098384A (ko) 2022-07-12
EP4061946A1 (en) 2022-09-28

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