EA202192818A1 - COMPOSITIONS AND METHODS FOR THE TREATMENT OF CYSTIC FISSIDOSIS - Google Patents

COMPOSITIONS AND METHODS FOR THE TREATMENT OF CYSTIC FISSIDOSIS

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Publication number
EA202192818A1
EA202192818A1 EA202192818A EA202192818A EA202192818A1 EA 202192818 A1 EA202192818 A1 EA 202192818A1 EA 202192818 A EA202192818 A EA 202192818A EA 202192818 A EA202192818 A EA 202192818A EA 202192818 A1 EA202192818 A1 EA 202192818A1
Authority
EA
Eurasian Patent Office
Prior art keywords
treatment
methods
cystic
compositions
fissidosis
Prior art date
Application number
EA202192818A
Other languages
Russian (ru)
Inventor
Джон Ф. Энгельхард
Цзыин Янь
Инхуа Тан
Эрик Юэнь
Шэнь Линь
Original Assignee
Юниверсити Оф Айова Рисерч Фаундейшн
Спайровант Сайенсиз, Инк.
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 Юниверсити Оф Айова Рисерч Фаундейшн, Спайровант Сайенсиз, Инк. filed Critical Юниверсити Оф Айова Рисерч Фаундейшн
Priority claimed from PCT/US2020/028264 external-priority patent/WO2020214668A1/en
Publication of EA202192818A1 publication Critical patent/EA202192818A1/en

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  • 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)

Abstract

Изобретение относится к полинуклеотидам, векторам rAAV, фармацевтическим композициям и способам их получения и применения, например, для лечения муковисцидоза (CF). Например, изобретение относится к рекомбинантному аденоассоциированному вирусу (rAAV), который содержит, в одном варианте осуществления, капсидный белок AV.TL65 и полинуклеотид, который содержит энхансер F5 и промотор tg83, функционально связанный с мини-геном CFTRR, к его фармацевтическим композициям и способам их применения, например, для лечения CF.The invention relates to polynucleotides, rAAV vectors, pharmaceutical compositions and methods for their preparation and use, for example, in the treatment of cystic fibrosis (CF). For example, the invention relates to a recombinant adeno-associated virus (rAAV) that contains, in one embodiment, an AV.TL65 capsid protein and a polynucleotide that contains an F5 enhancer and a tg83 promoter operably linked to a CFTRR minigene, to pharmaceutical compositions and methods thereof. their use, for example, in the treatment of CF.

EA202192818A 2020-01-29 2020-04-15 COMPOSITIONS AND METHODS FOR THE TREATMENT OF CYSTIC FISSIDOSIS EA202192818A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202062967214P 2020-01-29 2020-01-29
PCT/US2020/028264 WO2020214668A1 (en) 2019-04-15 2020-04-15 Compositions and methods for treatment of cystic fibrosis

Publications (1)

Publication Number Publication Date
EA202192818A1 true EA202192818A1 (en) 2022-03-03

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Family Applications (1)

Application Number Title Priority Date Filing Date
EA202192818A EA202192818A1 (en) 2020-01-29 2020-04-15 COMPOSITIONS AND METHODS FOR THE TREATMENT OF CYSTIC FISSIDOSIS

Country Status (1)

Country Link
EA (1) EA202192818A1 (en)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11684679B2 (en) 2016-03-07 2023-06-27 University Of Iowa Research Foundation AAV-mediated expression using a synthetic promoter and enhancer
US11702672B2 (en) 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11702672B2 (en) 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
US11684679B2 (en) 2016-03-07 2023-06-27 University Of Iowa Research Foundation AAV-mediated expression using a synthetic promoter and enhancer

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