PE20190802A1 - Variantes de alfa-glucosidasa acida y usos de las mismas - Google Patents

Variantes de alfa-glucosidasa acida y usos de las mismas

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Publication number
PE20190802A1
PE20190802A1 PE2019000499A PE2019000499A PE20190802A1 PE 20190802 A1 PE20190802 A1 PE 20190802A1 PE 2019000499 A PE2019000499 A PE 2019000499A PE 2019000499 A PE2019000499 A PE 2019000499A PE 20190802 A1 PE20190802 A1 PE 20190802A1
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Peru
Prior art keywords
gaa
nucleic acid
glucosidase
refers
protein
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PE2019000499A
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English (en)
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Federico Mingozzi
Giuseppe Rozitti
Pasqualina Colella
Francesco Puzzo
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Genethon
Univ Sorbonne
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Application filed by Genethon, Univ Sorbonne filed Critical Genethon
Publication of PE20190802A1 publication Critical patent/PE20190802A1/es

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    • C12Y302/0102Alpha-glucosidase (3.2.1.20)

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Abstract

Referido a una molecula de acido nucleico que codifica una proteina de alfa-glucosidasa acida (GAA) quimerica, que comprende un resto de peptido senal y un resto de GAA funcional; donde el resto de GAA corresponde a una forma truncada de una GAA parenteral; y el resto de peptido senal tiene una secuencia de aminoacidos seleccionada entre el grupo que consiste en SEQ ID NO: 2 a 4, preferentemente SEQ ID NO:2. Dicha molecula expresa y secreta niveles mas altos en comparacion con la proteina de GAA de tipo silvestre y producen una correccion mejorada de la acumulacion patologica de glucogeno en todo el cuerpo. Tambien se refiere a una celula transformada con la molecula de acido nucleico, una construccion y un vector que comprenden el citado acido nucleico; siendo util para tratar una enfermedad de almacenamiento de glucogeno, tal como enfermedad de von Gierke (FSDI), entre otros
PE2019000499A 2016-09-12 2017-09-12 Variantes de alfa-glucosidasa acida y usos de las mismas PE20190802A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP16306149.2A EP3293203A1 (en) 2016-09-12 2016-09-12 Acid-alpha glucosidase variants and uses thereof
PCT/EP2017/072945 WO2018046775A1 (en) 2016-09-12 2017-09-12 Acid-alpha glucosidase variants and uses thereof

Publications (1)

Publication Number Publication Date
PE20190802A1 true PE20190802A1 (es) 2019-06-10

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PE2019000499A PE20190802A1 (es) 2016-09-12 2017-09-12 Variantes de alfa-glucosidasa acida y usos de las mismas

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US (3) US20190390224A1 (es)
EP (3) EP3293203A1 (es)
JP (2) JP2019533991A (es)
KR (2) KR20230049763A (es)
CN (2) CN109843930B (es)
AU (1) AU2017322377B2 (es)
BR (1) BR112019004783A2 (es)
CA (1) CA3036368A1 (es)
CL (1) CL2019000610A1 (es)
CO (1) CO2019002248A2 (es)
DK (1) DK3510049T3 (es)
ES (1) ES2970293T3 (es)
FI (1) FI3510049T3 (es)
IL (2) IL302354B1 (es)
MX (1) MX2019002841A (es)
PE (1) PE20190802A1 (es)
PH (1) PH12019500376A1 (es)
PT (1) PT3510049T (es)
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WO (1) WO2018046775A1 (es)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
EP3293259A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
US11339406B2 (en) 2016-09-12 2022-05-24 Genethon Acid-alpha glucosidase variants and uses thereof
CN110809583A (zh) 2017-06-07 2020-02-18 瑞泽恩制药公司 用于内化酶的组合物和方法
BR112020023393A2 (pt) * 2018-05-16 2021-02-09 Spark Therapeutics, Inc. cassetes de expressão de a-glicosidase ácida otimizados por códon e métodos de uso dos mesmos
CN113747926A (zh) 2019-04-08 2021-12-03 吉尼松公司 用于肌肉表达的杂合启动子
EP3996696A1 (en) 2019-07-09 2022-05-18 Genethon Treatment of glycogen storage disease (gsd)
WO2021078834A1 (en) 2019-10-22 2021-04-29 Genethon Chimeric acid-alpha glucosidase polypeptides and uses thereof
JP2022553550A (ja) 2019-10-22 2022-12-23 ジェネトン キメラポリペプチド及びその使用
TW202136506A (zh) * 2019-12-20 2021-10-01 美商克迪科思股份有限公司 經工程處理之酸性α-葡萄糖苷酶變異體
CN117897492A (zh) 2021-08-04 2024-04-16 吉尼松公司 用于肌肉和cns中的基因表达的杂合启动子

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6858425B1 (en) 1998-12-04 2005-02-22 Genzyme Corporation Human acid alpha glucosidase gene and bovine alpha-S1 casein gene sequences
CA2353522A1 (en) 1998-12-07 2000-06-15 Pharming Intellectual Property B.V. Treatment of pompe's disease
US7629309B2 (en) * 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
WO2004064750A2 (en) 2003-01-22 2004-08-05 Duke University Improved constructs for expressing lysosomal polypeptides
JP4914224B2 (ja) * 2004-02-10 2012-04-11 バイオマリン ファーマシューティカル インコーポレイテッド 酸性αグルコシダーゼおよびそのフラグメント
US8633006B2 (en) * 2004-06-29 2014-01-21 Novozymes, Inc. Polypeptides having alpha-glucosidase activity and polynucleotides encoding same
EP3290513B1 (en) 2004-06-01 2022-09-07 Genzyme Corporation Compositions and methods to prevent aav vector aggregation
HUE025212T2 (en) 2008-07-08 2016-03-29 Univ Duke A method for treating glycogen storage disease
WO2012085622A1 (en) * 2010-12-22 2012-06-28 Fondazione Telethon Therapeutic strategies to treat cns pathology in mucopolysaccharidoses
MX2013012345A (es) 2011-04-22 2015-05-07 Genzyme Corp Alfa glucosidasa acida modificada con procesamiento acelerado.
WO2013013017A2 (en) * 2011-07-21 2013-01-24 Alnylam Pharmaceuticals, Inc. Compositions and methods for modifying the glycosylation of lysosomal storage disorder therapeutics
US9540657B2 (en) * 2012-05-25 2017-01-10 California Institute Of Technology Expression of secreted and cell-surface polypeptides
CN110606874B (zh) 2013-07-22 2024-04-19 费城儿童医院 用于基因转移到细胞、器官和组织中的变异aav和组合物、方法及用途
EP3851537B1 (en) 2014-04-25 2023-12-27 Genethon Treatment of hyperbilirubinemia
US10202591B2 (en) * 2014-06-13 2019-02-12 Valerion Therapeutics, Llc Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders
WO2015196179A1 (en) 2014-06-20 2015-12-23 University Of Florida Research Foundation, Inc. Methods of packaging multiple adeno-associated virus vectors
WO2016065319A1 (en) * 2014-10-24 2016-04-28 Shire Human Genetic Therapies, Inc. Lysosomal targeting of enzymes, and uses thereof

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IL302354A (en) 2023-06-01
RU2019110772A3 (es) 2020-11-25
KR20230049763A (ko) 2023-04-13
US20240043867A1 (en) 2024-02-08
CN109843930B (zh) 2023-11-17
PH12019500376A1 (en) 2019-05-15
KR20190056388A (ko) 2019-05-24
US20190390224A1 (en) 2019-12-26
DK3510049T3 (da) 2024-01-29
ES2970293T3 (es) 2024-05-27
SG11201901430WA (en) 2019-03-28
RU2019110772A (ru) 2020-10-12
AU2017322377B2 (en) 2024-05-16
IL265170A (en) 2019-05-30
EP3510049B1 (en) 2023-11-08
EP3510049B8 (en) 2023-12-20
MX2019002841A (es) 2019-08-29
IL302354B1 (en) 2024-05-01
JP2019533991A (ja) 2019-11-28
IL265170B1 (en) 2023-06-01
JP2022180543A (ja) 2022-12-06
CN117512014A (zh) 2024-02-06
AU2017322377A1 (en) 2019-03-28
FI3510049T3 (fi) 2024-02-02
CN109843930A (zh) 2019-06-04
BR112019004783A2 (pt) 2019-06-04
CO2019002248A2 (es) 2019-05-21
CL2019000610A1 (es) 2019-07-05
EP3510049A1 (en) 2019-07-17
WO2018046775A1 (en) 2018-03-15
EP4361178A2 (en) 2024-05-01
PT3510049T (pt) 2024-01-30
EP3293203A1 (en) 2018-03-14
IL265170B2 (en) 2023-10-01
US20210040503A1 (en) 2021-02-11
CA3036368A1 (en) 2018-03-15

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