ES3057448T3 - Mutated beta-glucocerebrosidase with improved stability - Google Patents
Mutated beta-glucocerebrosidase with improved stabilityInfo
- Publication number
- ES3057448T3 ES3057448T3 ES21752123T ES21752123T ES3057448T3 ES 3057448 T3 ES3057448 T3 ES 3057448T3 ES 21752123 T ES21752123 T ES 21752123T ES 21752123 T ES21752123 T ES 21752123T ES 3057448 T3 ES3057448 T3 ES 3057448T3
- Authority
- ES
- Spain
- Prior art keywords
- gcase
- polypeptide
- modified
- mutation
- optionally
- Prior art date
- Legal status (The legal status 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 status listed.)
- Active
Links
Classifications
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Food Preservation Except Freezing, Refrigeration, And Drying (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB2011813.9A GB202011813D0 (en) | 2020-07-29 | 2020-07-29 | Polypeptide |
| GBGB2100648.1A GB202100648D0 (en) | 2021-01-18 | 2021-01-18 | Polypeptide |
| GBGB2105924.1A GB202105924D0 (en) | 2021-04-26 | 2021-04-26 | Polypeptide |
| PCT/GB2021/051969 WO2022023761A2 (en) | 2020-07-29 | 2021-07-29 | Polypeptide |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ES3057448T3 true ES3057448T3 (en) | 2026-03-02 |
Family
ID=77265101
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES21752123T Active ES3057448T3 (en) | 2020-07-29 | 2021-07-29 | Mutated beta-glucocerebrosidase with improved stability |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US20240261433A1 (https=) |
| EP (2) | EP4118200B1 (https=) |
| JP (1) | JP2023535808A (https=) |
| KR (1) | KR20230042513A (https=) |
| CN (1) | CN116322744A (https=) |
| AU (1) | AU2021316875B2 (https=) |
| BR (1) | BR112023001583A2 (https=) |
| CA (1) | CA3189801A1 (https=) |
| DK (1) | DK4118200T3 (https=) |
| ES (1) | ES3057448T3 (https=) |
| FI (1) | FI4118200T3 (https=) |
| HR (1) | HRP20251591T1 (https=) |
| IL (1) | IL299811A (https=) |
| MX (1) | MX2023001137A (https=) |
| PT (1) | PT4118200T (https=) |
| WO (1) | WO2022023761A2 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025122912A1 (en) | 2023-12-08 | 2025-06-12 | Denali Therapeutics Inc. | Modified glucocerebrosidase polypeptides and methods of use thereof |
| WO2025166045A1 (en) | 2024-01-31 | 2025-08-07 | Alector Llc | β-GLUCOCEREBROSIDASE ENZYMES, FUSION PROTEINS AND COMPLEXES COMPRISING THE SAME, AND METHODS OF USE THEREOF |
| CN118086341B (zh) * | 2024-04-25 | 2024-08-02 | 上海凌医生物科技有限公司 | 在肝脏中高表达人源葡萄糖脑苷脂酶基因的表达框 |
| WO2026006173A1 (en) | 2024-06-24 | 2026-01-02 | Denali Therapeutics Inc. | Fusion proteins comprising modified glucocerebrosidase polypeptides and methods thereof |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU1775901A (en) * | 1999-11-17 | 2001-05-30 | Avigen, Inc. | Recombinant adeno-associated virus virions for the treatment of lysosomal disorders |
| AU2352201A (en) * | 1999-12-30 | 2001-07-16 | Maxygen Aps | Improved lysosomal enzymes and lysosomal enzyme activators |
| EP2154969B1 (en) | 2007-05-16 | 2015-11-18 | The Brigham and Women's Hospital, Inc. | Treatment of synucleinopathies |
| WO2011122950A1 (en) | 2010-04-01 | 2011-10-06 | Amsterdam Molecular Therapeutics (Amt) Ip B.V. | Monomeric duplex aav vectors |
| SI2638152T1 (sl) * | 2010-11-08 | 2016-12-30 | Amicus Therapeutics, Inc. | Variantni in rekombinantni proteini beta-glukocerebrozidaze s povišano stabilnostjo in povišano ohranjeno katalitično aktivnostjo |
| AU2013243948A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
| US9574184B2 (en) * | 2013-09-25 | 2017-02-21 | Children's Hospital Medical Center | Lysosomal protein targeting sequence and therapeutic applications of same |
| KR102450833B1 (ko) * | 2016-04-15 | 2022-10-05 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 혈우병 a 치료용 유전자 요법 |
| EP3692075A4 (en) | 2017-10-03 | 2022-02-09 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISEASES |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| WO2020012149A1 (en) | 2018-07-13 | 2020-01-16 | Ucl Business Ltd | Glucocerebrosidase gene therapy |
| WO2020012164A1 (en) | 2018-07-13 | 2020-01-16 | Ucl Business Ltd | Glucocerebrosidase gene therapy |
| US20220125892A1 (en) * | 2019-02-01 | 2022-04-28 | OXYRANE UK Ltd. | Glucocerebrosidase polypeptides |
-
2021
- 2021-07-29 EP EP21752123.6A patent/EP4118200B1/en active Active
- 2021-07-29 KR KR1020237007296A patent/KR20230042513A/ko active Pending
- 2021-07-29 CA CA3189801A patent/CA3189801A1/en active Pending
- 2021-07-29 ES ES21752123T patent/ES3057448T3/es active Active
- 2021-07-29 MX MX2023001137A patent/MX2023001137A/es unknown
- 2021-07-29 DK DK21752123.6T patent/DK4118200T3/da active
- 2021-07-29 US US18/017,158 patent/US20240261433A1/en active Pending
- 2021-07-29 PT PT217521236T patent/PT4118200T/pt unknown
- 2021-07-29 HR HRP20251591TT patent/HRP20251591T1/hr unknown
- 2021-07-29 JP JP2023506111A patent/JP2023535808A/ja active Pending
- 2021-07-29 WO PCT/GB2021/051969 patent/WO2022023761A2/en not_active Ceased
- 2021-07-29 CN CN202180066422.7A patent/CN116322744A/zh active Pending
- 2021-07-29 IL IL299811A patent/IL299811A/en unknown
- 2021-07-29 FI FIEP21752123.6T patent/FI4118200T3/fi active
- 2021-07-29 BR BR112023001583A patent/BR112023001583A2/pt unknown
- 2021-07-29 AU AU2021316875A patent/AU2021316875B2/en active Active
- 2021-07-29 EP EP25199900.9A patent/EP4643868A3/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| KR20230042513A (ko) | 2023-03-28 |
| WO2022023761A2 (en) | 2022-02-03 |
| EP4643868A3 (en) | 2026-04-08 |
| JP2023535808A (ja) | 2023-08-21 |
| FI4118200T3 (fi) | 2025-12-12 |
| CN116322744A (zh) | 2023-06-23 |
| EP4118200B1 (en) | 2025-10-08 |
| IL299811A (en) | 2023-03-01 |
| EP4643868A2 (en) | 2025-11-05 |
| BR112023001583A2 (pt) | 2023-02-14 |
| PT4118200T (pt) | 2026-01-08 |
| CA3189801A1 (en) | 2022-02-03 |
| MX2023001137A (es) | 2023-02-22 |
| DK4118200T3 (da) | 2025-12-08 |
| WO2022023761A3 (en) | 2022-03-10 |
| EP4118200A2 (en) | 2023-01-18 |
| US20240261433A1 (en) | 2024-08-08 |
| AU2021316875A1 (en) | 2023-02-23 |
| AU2021316875B2 (en) | 2026-04-02 |
| HRP20251591T1 (hr) | 2026-01-30 |
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