EA202192818A1 - COMPOSITIONS AND METHODS FOR THE TREATMENT OF CYSTIC FISSIDOSIS - Google Patents
COMPOSITIONS AND METHODS FOR THE TREATMENT OF CYSTIC FISSIDOSISInfo
- 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
Links
Landscapes
- 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.
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 |
Family
ID=81077479
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)
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 |
-
2020
- 2020-04-15 EA EA202192818A patent/EA202192818A1/en unknown
Cited By (2)
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 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
MX2021012681A (en) | Compositions and methods for treatment of cystic fibrosis. | |
PH12020550094A1 (en) | Adeno-associated virus variant capsids and methods of use thereof | |
CL2020002200A1 (en) | New vectors of adeno-associated virus (aav), vectors that have reduced capsid deamination and their uses. | |
CL2020002201A1 (en) | Novel adeno-associated virus (aav) vectors, aav vectors that have reduced capsid deamidation, and uses for these. | |
EA202192818A1 (en) | COMPOSITIONS AND METHODS FOR THE TREATMENT OF CYSTIC FISSIDOSIS | |
CL2019002474A1 (en) | Vector clade f of adeno-associated virus (vaa) and related uses. | |
PH12020550706A1 (en) | Adeno-associated virus variant capsids and use for inhibiting angiogenesis | |
WO2019222136A3 (en) | Liver targeting adeno-associated viral vectors | |
MX2023009627A (en) | Adeno-associated virus variant capsids and methods of use thereof. | |
BR112021020054A2 (en) | Adeno-associated virus (aav) capsids and compositions containing the same | |
MX2021005517A (en) | Gene therapy for neuronal ceroid lipofuscinoses. | |
WO2018022608A3 (en) | Novel adeno-associated virus capsid proteins | |
BR112021018776A2 (en) | Recombinant adeno-associated virus vectors | |
BR112021020957A2 (en) | Variant adeno-associated virus (aav) capsids for intravitreal delivery | |
BR112019023303A2 (en) | GENE THERAPY FOR NEURONAL CEROID LIPOFUSCINOSES | |
WO2020219766A8 (en) | Compositions useful in treatment of rett syndrome | |
MX2021012682A (en) | Methods and compositions for transgene expression. | |
EA202192819A1 (en) | METHODS AND COMPOSITIONS FOR TRANSGENE EXPRESSION | |
EA202192160A1 (en) | DELIVERY OF POLYNUCLEOTIDE CLN3 USING AN ADENO-ASSOCIATED VIRUS | |
MX2022000551A (en) | Modified aav capsid proteins for treatment of arthritic disease. | |
EA202092046A1 (en) | NEW VECTORS BASED ON THE ADENO-ASSOCIATED VIRUS (AAV), AAV VECTORS WITH REDUCED CAPSID DESAMIDATION, AS WELL AS THEIR APPLICATION | |
EA202092015A1 (en) | NEW VECTORS BASED ON THE ADENO-ASSOCIATED VIRUS (AAV), AAV VECTORS WITH REDUCED CAPSID DESAMIDATION, AND THEIR APPLICATION | |
AR122261A1 (en) | COMPOSITIONS AND METHODS FOR THE TREATMENT OF TAUPATHY | |
EA202192936A1 (en) | AAV CAPSID VARIANTS FOR DELIVERY TO THE VIRTUE | |
EA202192973A1 (en) | COMPOSITIONS USEFUL IN THE TREATMENT OF POMPE DISEASE |