CO2022006772A2 - Composiciones y métodos para el tratamiento de los trastornos de almacenamiento de glucógeno - Google Patents

Composiciones y métodos para el tratamiento de los trastornos de almacenamiento de glucógeno

Info

Publication number
CO2022006772A2
CO2022006772A2 CONC2022/0006772A CO2022006772A CO2022006772A2 CO 2022006772 A2 CO2022006772 A2 CO 2022006772A2 CO 2022006772 A CO2022006772 A CO 2022006772A CO 2022006772 A2 CO2022006772 A2 CO 2022006772A2
Authority
CO
Colombia
Prior art keywords
compositions
methods
glycogen storage
treatment
storage disorders
Prior art date
Application number
CONC2022/0006772A
Other languages
English (en)
Inventor
John T Gray
Justine Cunningham
Salvador Rico
Original Assignee
Audentes 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 Audentes Therapeutics Inc filed Critical Audentes Therapeutics Inc
Publication of CO2022006772A2 publication Critical patent/CO2022006772A2/es

Links

Classifications

    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083—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 administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • 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
    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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

Landscapes

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

Abstract

La presente divulgación se refiere a composiciones y métodos útiles para tratar trastornos de almacenamiento de glucógeno, como el trastorno de almacenamiento de glucógeno de tipo II, que también se denomina en la presente enfermedad de Pompe. Al usar las composiciones y métodos de la divulgación, a un paciente (p. ej., un paciente mamífero, como un paciente humano) que tiene la enfermedad de Pompe se le puede administrar un vector viral, como un vector viral adenoasociado (AAV), que contiene un transgén que codifica la alfa-glucosidasa ácida.
CONC2022/0006772A 2019-10-25 2022-05-23 Composiciones y métodos para el tratamiento de los trastornos de almacenamiento de glucógeno CO2022006772A2 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962926282P 2019-10-25 2019-10-25
US202063083349P 2020-09-25 2020-09-25
PCT/US2020/057081 WO2021081338A1 (en) 2019-10-25 2020-10-23 Compositions and methods for treating glycogen storage disorders

Publications (1)

Publication Number Publication Date
CO2022006772A2 true CO2022006772A2 (es) 2022-08-09

Family

ID=75620302

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2022/0006772A CO2022006772A2 (es) 2019-10-25 2022-05-23 Composiciones y métodos para el tratamiento de los trastornos de almacenamiento de glucógeno

Country Status (13)

Country Link
US (1) US20220387562A1 (es)
EP (1) EP4048286A4 (es)
JP (1) JP2022554141A (es)
KR (1) KR20220105643A (es)
CN (1) CN114828858A (es)
AU (1) AU2020372429A1 (es)
BR (1) BR112022007674A2 (es)
CA (1) CA3158281A1 (es)
CO (1) CO2022006772A2 (es)
IL (1) IL292401A (es)
MX (1) MX2022004799A (es)
TW (1) TW202116359A (es)
WO (1) WO2021081338A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023150688A2 (en) * 2022-02-03 2023-08-10 Astellas Gene Therapies, Inc. Compositions and methods for improved treatment of pompe disease

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2013277215A1 (en) * 2012-06-19 2015-02-05 University Of Florida Research Foundation, Inc. Compositions and methods for treating diseases
EP3293260A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
JP7467356B2 (ja) * 2018-02-07 2024-04-15 ジェネトン ハイブリッド調節要素

Also Published As

Publication number Publication date
CA3158281A1 (en) 2021-04-29
BR112022007674A2 (pt) 2022-08-09
IL292401A (en) 2022-06-01
EP4048286A1 (en) 2022-08-31
CN114828858A (zh) 2022-07-29
KR20220105643A (ko) 2022-07-27
EP4048286A4 (en) 2023-12-06
AU2020372429A1 (en) 2022-04-28
US20220387562A1 (en) 2022-12-08
JP2022554141A (ja) 2022-12-28
WO2021081338A1 (en) 2021-04-29
TW202116359A (zh) 2021-05-01
MX2022004799A (es) 2022-07-19

Similar Documents

Publication Publication Date Title
MX2020003965A (es) Terapias genicas para los trastornos lisosomales.
CL2021002881A1 (es) Composiciones útiles en el tratamiento de leucodistrofia metacromática
MX2020003888A (es) Anticuerpos terapeuticos postraduccionalmente modificados completamente humanos.
MX2020004005A (es) Terapias genicas para los trastornos lisosomales.
BR112017017867A2 (pt) métodos e composições para tratamento de doenças oculares genéticas
CL2017002028A1 (es) Mejora del suministro de partículas virales al cuerpo estriado y al córtex.
CO2018012082A2 (es) Administración de vectores del virus adenoasociado de β-sarcoglicano y microarn-29 y el tratamiento de la distrofia muscular
AR114971A1 (es) Compuestos de purinona y su uso en el tratamiento del cáncer
CO2022012917A2 (es) Vectores de genoterapia para tratar enfermedades cardíacas
BR112021013140A2 (pt) Construções de terapia gênica para tratar doença de wilson
EA201491081A1 (ru) Композиции и способы для лечения заболеваний и расстройств печени
CO2020014193A2 (es) Terapia genética para las enfermedades causadas por piscinas de nucleótidos desequilibradas, incluyendo los síndromes de depleción del adn mitocondrial
CO2021008877A2 (es) Uso de vectores lentivirales que expresan el factor ix
CL2023000629A1 (es) Partículas virales modificadas y usos de estas
CR20200224A (es) Composiciones y métodos de tratamiento de la insuficiencia cardiaca con fracción de eyección reducida con dapagliflozina
CO2022006772A2 (es) Composiciones y métodos para el tratamiento de los trastornos de almacenamiento de glucógeno
BR112018075692A2 (pt) genes cln1 otimizados e cassetes de expressão e seu uso
MX2021006253A (es) Terapias génicas para enfermedad neurodegenerativa.
BR112022008214A2 (pt) Composições e métodos para tratar perda auditiva neurossensorial usando sistemas de vetor duplo de otoferlina
CO2021016487A2 (es) Vectores aav con el promotor de la proteína cero de mielina y usos de los mismos para el tratamiento de enfermedades asociadas a las células de schwann como la enfermedad de charcot-marie-tooth
BR112022003659A2 (pt) Composições e métodos de tratamento de doenças vasculares
CO2022016956A2 (es) Composiciones útiles en el tratamiento del trastorno de deficiencia de cdkl5 (cdd)
Seo et al. Early immunomodulation by intravenously transplanted mesenchymal stem cells promotes functional recovery in spinal cord injured rats
BR112022021468A2 (pt) Genes gla otimizados por códon e usos dos mesmos
BR112022000898A2 (pt) Terapia de gene cardíaco aav para cardiomiopatia em seres humanos