EA202192973A1 - COMPOSITIONS USEFUL IN THE TREATMENT OF POMPE DISEASE - Google Patents
COMPOSITIONS USEFUL IN THE TREATMENT OF POMPE DISEASEInfo
- Publication number
- EA202192973A1 EA202192973A1 EA202192973A EA202192973A EA202192973A1 EA 202192973 A1 EA202192973 A1 EA 202192973A1 EA 202192973 A EA202192973 A EA 202192973A EA 202192973 A EA202192973 A EA 202192973A EA 202192973 A1 EA202192973 A1 EA 202192973A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- treatment
- paav
- compositions useful
- pompe disease
- pompe
- Prior art date
Links
Abstract
Предлагается рекомбинантный аденоассоциированный вирус (рААВ), полезный для лечения болезни накопления гликогена (Помпе) типа II. рААВ содержит капсид ААВ, который нацелен на клетки по меньшей мере чего-то одного из мышц, сердца, почки и центральной нервной системы и в который упакован геном вектора, содержащий последовательность нуклеиновой кислоты, кодирующей белок кислой -глюкозидазы человека hGAA780I или слитый белок под контролем регуляторных последовательностей, направляющих его экспрессию. Также предусматриваются способы изготовления и применения такого рААВ.A recombinant adeno-associated virus (rAAV) is provided that is useful in the treatment of glycogen storage disease (Pompe) type II. pAAV contains an AAV capsid that targets cells of at least one of the muscles, heart, kidney, and central nervous system, and into which the vector genome is packaged, containing the nucleic acid sequence encoding the human acid β-glucosidase protein hGAA780I or a controlled fusion protein. regulatory sequences that direct its expression. Methods for making and using such a pAAV are also provided.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962913401P | 2019-10-10 | 2019-10-10 | |
| PCT/US2020/030493 WO2020223362A1 (en) | 2019-04-30 | 2020-04-29 | Compositions useful for treatment of pompe disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| EA202192973A1 true EA202192973A1 (en) | 2022-02-07 |
Family
ID=80631339
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EA202192971A EA202192971A1 (en) | 2019-10-10 | 2020-04-29 | COMPOSITIONS USEFUL IN THE TREATMENT OF POMPE DISEASE |
| EA202192973A EA202192973A1 (en) | 2019-10-10 | 2020-04-29 | COMPOSITIONS USEFUL IN THE TREATMENT OF POMPE DISEASE |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EA202192971A EA202192971A1 (en) | 2019-10-10 | 2020-04-29 | COMPOSITIONS USEFUL IN THE TREATMENT OF POMPE DISEASE |
Country Status (1)
| Country | Link |
|---|---|
| EA (2) | EA202192971A1 (en) |
-
2020
- 2020-04-29 EA EA202192971A patent/EA202192971A1/en unknown
- 2020-04-29 EA EA202192973A patent/EA202192973A1/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EA202192971A1 (en) | 2022-02-01 |
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