BR112022007674A2 - Métodos para tratar doença de pompe, para melhorar a função muscular, para reduzir o acúmulo de glicogênio, para melhorar a função pulmonar e para aumentar a expressão de gaa, kits e usos de um vetor aav - Google Patents

Métodos para tratar doença de pompe, para melhorar a função muscular, para reduzir o acúmulo de glicogênio, para melhorar a função pulmonar e para aumentar a expressão de gaa, kits e usos de um vetor aav

Info

Publication number
BR112022007674A2
BR112022007674A2 BR112022007674A BR112022007674A BR112022007674A2 BR 112022007674 A2 BR112022007674 A2 BR 112022007674A2 BR 112022007674 A BR112022007674 A BR 112022007674A BR 112022007674 A BR112022007674 A BR 112022007674A BR 112022007674 A2 BR112022007674 A2 BR 112022007674A2
Authority
BR
Brazil
Prior art keywords
methods
improve
kits
function
aav vector
Prior art date
Application number
BR112022007674A
Other languages
English (en)
Portuguese (pt)
Inventor
T Gray John
Cunningham Justine
Rico Salvador
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 BR112022007674A2 publication Critical patent/BR112022007674A2/pt

Links

Classifications

    • 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
    • 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
    • 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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • Diabetes (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)
BR112022007674A 2019-10-25 2020-10-23 Métodos para tratar doença de pompe, para melhorar a função muscular, para reduzir o acúmulo de glicogênio, para melhorar a função pulmonar e para aumentar a expressão de gaa, kits e usos de um vetor aav BR112022007674A2 (pt)

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
BR112022007674A2 true BR112022007674A2 (pt) 2022-08-09

Family

ID=75620302

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022007674A BR112022007674A2 (pt) 2019-10-25 2020-10-23 Métodos para tratar doença de pompe, para melhorar a função muscular, para reduzir o acúmulo de glicogênio, para melhorar a função pulmonar e para aumentar a expressão de gaa, kits e usos de um vetor aav

Country Status (14)

Country Link
US (1) US20220387562A1 (https=)
EP (1) EP4048286A4 (https=)
JP (1) JP2022554141A (https=)
KR (1) KR20220105643A (https=)
CN (1) CN114828858A (https=)
AU (1) AU2020372429A1 (https=)
BR (1) BR112022007674A2 (https=)
CA (1) CA3158281A1 (https=)
CO (1) CO2022006772A2 (https=)
IL (1) IL292401A (https=)
MX (1) MX2022004799A (https=)
PH (1) PH12022550977A1 (https=)
TW (2) TWI885000B (https=)
WO (1) WO2021081338A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240145491A (ko) * 2022-02-03 2024-10-07 아스텔라스 진 테라피스, 인크. 폼페병의 개선된 치료를 위한 조성물 및 방법
JP2025527694A (ja) 2022-08-23 2025-08-22 マストバイオ株式会社 Ｉｌ２変異体及びそれを含むタンパク質複合体

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE437944T1 (de) * 2000-04-28 2009-08-15 Asklepios Biopharmaceutical In Für das dystrophin-minigen kodierende dna- sequenzen und verfahren zu deren verwendung
ATE400656T1 (de) * 2000-10-06 2008-07-15 Univ Michigan Mini-dystrophin nukleinsäure- und peptidsequenzen
RS61055B1 (sr) * 2012-06-19 2020-12-31 Univ Florida Kompozicije i postupci za lečenje bolesti
EA201890543A1 (ru) * 2015-09-17 2018-08-31 Рисёрч Инститьют Эт Нэйшнвайд Чилдрен'С Хоспитэл Способы и материалы для генной терапии galgt2
EP3293260A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
WO2019012336A2 (en) * 2017-03-17 2019-01-17 Newcastle University ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY
EP3648776A4 (en) * 2017-07-06 2021-04-07 The Medical College of Wisconsin, Inc. NEW IN-VITRO AND IN-VIVO ENRICHMENT STRATEGY AGAINST LYMPHOCYTE FROM VECTOR-TRANSDUCED HSC FOR THE TREATMENT OF DISEASES
US12296026B2 (en) * 2018-02-05 2025-05-13 Astellas Gene Therapies, Inc. Transcription regulatory elements and uses thereof
EP3749771A1 (en) * 2018-02-07 2020-12-16 Genethon Hybrid regulatory elements

Also Published As

Publication number Publication date
EP4048286A1 (en) 2022-08-31
IL292401A (en) 2022-06-01
CN114828858A (zh) 2022-07-29
JP2022554141A (ja) 2022-12-28
TWI885000B (zh) 2025-06-01
CA3158281A1 (en) 2021-04-29
TW202600177A (zh) 2026-01-01
PH12022550977A1 (en) 2023-10-09
TW202116359A (zh) 2021-05-01
CO2022006772A2 (es) 2022-08-09
MX2022004799A (es) 2022-07-19
WO2021081338A1 (en) 2021-04-29
KR20220105643A (ko) 2022-07-27
US20220387562A1 (en) 2022-12-08
AU2020372429A1 (en) 2022-04-28
EP4048286A4 (en) 2023-12-06

Similar Documents

Publication Publication Date Title
Gulyaeva et al. Genetic and epigenetic control of adipose development
Del Sorbo et al. Mechanical ventilation in adults with acute respiratory distress syndrome. Summary of the experimental evidence for the clinical practice guideline
BR112022015979A2 (pt) Vetores de terapia genética para tratamento de doenças cardíacas
BR112021021156A2 (pt) Terapêuticos anticorpos totalmente humanos pós-tradução modificados
BR112021019880A2 (pt) Terapias gênicas para distúrbios lisossomais
ES2976559T3 (es) Terapia génica con conexina 43 para restaurar la función eléctrica y cardíaca y la estructura cardíaca, en la miocardiopatía arritmogénica del ventrículo derecho
CY1119295T1 (el) Γονιδιακη θεραπεια για ασθενειες λυσοσωμικης αποθhκευσης
MX2021000443A (es) Vectores de terapia génica para el tratamiento de la enfermedad de danon.
BRPI0410041A (pt) métodos para tratamento de dor de cabeça de sinusite
BR112021021908A2 (pt) Composições úteis no tratamento da leucodistrofia metacromática
BR112014031788A2 (pt) composições e métodos para o tratamento de diabetes
BR112022007674A2 (pt) Métodos para tratar doença de pompe, para melhorar a função muscular, para reduzir o acúmulo de glicogênio, para melhorar a função pulmonar e para aumentar a expressão de gaa, kits e usos de um vetor aav
WO2022035903A3 (en) Gene therapies for lysosomal disorders
BR112021015751A2 (pt) Vetores de terapia genética para tratamento da doença de danon
TW202012628A (zh) 用於帕金森氏症治療的組成物及方法
MX2021006253A (es) Terapias génicas para enfermedad neurodegenerativa.
Gould Gene doping: gene delivery for olympic victory
Mendell et al. 480. Gene therapy for spinal muscular atrophy type 1 shows potential to improve survival and motor functional outcomes
Senthilkumar et al. Reversal of neuropsychiatric comorbidities in an animal model of temporal lobe epilepsy following systemic administration of dental pulp stem cells and bone marrow mesenchymal stem cells
CN111542612B (zh) 用于治疗弗里德希氏共济失调的载体
MX2021009383A (es) Metodos para el tratamiento contra sintomas y trastornos asociados a enfermedades del almacenamiento lisosomico.
JOP20240081A1 (ar) مُعدِّلات الجزيئات الصغيرة لنشاط الجلوكوسيريبروسيد واستخداماتها
CN108888753B (zh) 酸性糖蛋白orm在制备治疗心力衰竭药物中的应用
Hoshikawa et al. In vitro and in vivo effects of D-allose: up-regulation of thioredoxin-interacting protein in head and neck cancer cells
ES2959188T3 (es) Procedimientos de tratamiento de trastornos inflamatorios neurológicos