BR112022021479A2 - Variantes, formulações e métodos adeno-associados para entrega pulmonar - Google Patents

Variantes, formulações e métodos adeno-associados para entrega pulmonar

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Publication number
BR112022021479A2
BR112022021479A2 BR112022021479A BR112022021479A BR112022021479A2 BR 112022021479 A2 BR112022021479 A2 BR 112022021479A2 BR 112022021479 A BR112022021479 A BR 112022021479A BR 112022021479 A BR112022021479 A BR 112022021479A BR 112022021479 A2 BR112022021479 A2 BR 112022021479A2
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BR
Brazil
Prior art keywords
adeno
variants
formulations
pulmonary delivery
associated methods
Prior art date
Application number
BR112022021479A
Other languages
English (en)
Inventor
Kotterman Melissa
Francis Peter
Calton Melissa
Gonzales Johnny
Croze Roxanne
Schmitt Christopher
Original Assignee
4D Molecular 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 4D Molecular Therapeutics Inc filed Critical 4D Molecular Therapeutics Inc
Publication of BR112022021479A2 publication Critical patent/BR112022021479A2/pt

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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
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
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    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material

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  • Health & Medical Sciences (AREA)
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  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
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  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Pulmonology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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Abstract

VARIANTES, FORMULAÇÕES E MÉTODOS ADENO-ASSOCIADOS PARA ENTREGA PULMONAR. A presente revelação fornece uma proteína do capsídeo de AAV variante que confere tropismo às células pulmonares e vírus adeno-associados recombinantes que compreendem a variante AAV e composições farmacêuticas que compreendem as mesmas e seu uso na entrega de ácidos nucleicos heterólogos às células para o tratamento de doenças pulmonares.
BR112022021479A 2020-04-27 2021-04-26 Variantes, formulações e métodos adeno-associados para entrega pulmonar BR112022021479A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063016246P 2020-04-27 2020-04-27
US202063088432P 2020-10-06 2020-10-06
PCT/US2021/029253 WO2021222148A2 (en) 2020-04-27 2021-04-26 Adeno-associated variants, formulations and methods for pulmonary delivery

Publications (1)

Publication Number Publication Date
BR112022021479A2 true BR112022021479A2 (pt) 2022-12-13

Family

ID=78332150

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BR112022021479A BR112022021479A2 (pt) 2020-04-27 2021-04-26 Variantes, formulações e métodos adeno-associados para entrega pulmonar

Country Status (12)

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US (3) US11499166B2 (pt)
EP (1) EP4100539A4 (pt)
JP (2) JP7393565B2 (pt)
KR (1) KR20230026985A (pt)
CN (1) CN115552021A (pt)
AU (2) AU2021265768B2 (pt)
BR (1) BR112022021479A2 (pt)
CA (1) CA3172260A1 (pt)
IL (1) IL296445A (pt)
MX (1) MX2022013462A (pt)
WO (1) WO2021222148A2 (pt)
ZA (1) ZA202210026B (pt)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7393565B2 (ja) * 2020-04-27 2023-12-06 ４ディー モレキュラー セラピューティクス インコーポレイテッド 肺送達のためのアデノ随伴バリアント、製剤および方法
WO2023172873A2 (en) * 2022-03-07 2023-09-14 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids with improved lung tropism and uses thereof

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005056762A2 (en) 2003-12-05 2005-06-23 University Of Iowa Research Foundation Truncated cmv promoters and vectors containing same
WO2006036465A2 (en) 2004-09-03 2006-04-06 University Of Florida Compositions and methods for treating cystic fibrosis
CA2907799A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
WO2017201121A1 (en) * 2016-05-17 2017-11-23 University Of Florida Research Foundation, Inc. Recombinant aav for gene therapy in lungs
MA47603A (fr) 2017-02-27 2020-01-01 Translate Bio Inc Nouvel arnm cftr à codons optimisés
WO2018213476A1 (en) 2017-05-16 2018-11-22 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr
WO2020014479A1 (en) 2018-07-11 2020-01-16 Baxalta Incorporated Aav compositions
MX2021006646A (es) * 2018-12-05 2021-12-10 Abeona Therapeutics Inc Vector viral adenoasociado recombinante para el suministro de genes.
JP2022529002A (ja) * 2019-04-19 2022-06-16 レジェンクスバイオ インコーポレーテッド アデノ随伴ウイルスベクター製剤及び方法
JP7393565B2 (ja) * 2020-04-27 2023-12-06 ４ディー モレキュラー セラピューティクス インコーポレイテッド 肺送達のためのアデノ随伴バリアント、製剤および方法

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AU2023202289A1 (en) 2023-05-11
US11807868B2 (en) 2023-11-07
MX2022013462A (es) 2022-11-16
US20210395772A1 (en) 2021-12-23
EP4100539A2 (en) 2022-12-14
JP2023123668A (ja) 2023-09-05
AU2021265768A1 (en) 2022-11-03
AU2021265768B2 (en) 2023-03-30
US11499166B2 (en) 2022-11-15
EP4100539A4 (en) 2023-11-22
CN115552021A (zh) 2022-12-30
WO2021222148A2 (en) 2021-11-04
CA3172260A1 (en) 2021-11-04
ZA202210026B (en) 2024-01-31
WO2021222148A3 (en) 2021-12-02
US20230257772A1 (en) 2023-08-17
JP7393565B2 (ja) 2023-12-06
US20240067987A1 (en) 2024-02-29
KR20230026985A (ko) 2023-02-27
IL296445A (en) 2022-11-01
JP2023516223A (ja) 2023-04-18

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