MX2023008362A - Composiciones y metodos para el tratamiento de la enfermedad de fabry. - Google Patents
Composiciones y metodos para el tratamiento de la enfermedad de fabry.Info
- Publication number
- MX2023008362A MX2023008362A MX2023008362A MX2023008362A MX2023008362A MX 2023008362 A MX2023008362 A MX 2023008362A MX 2023008362 A MX2023008362 A MX 2023008362A MX 2023008362 A MX2023008362 A MX 2023008362A MX 2023008362 A MX2023008362 A MX 2023008362A
- Authority
- MX
- Mexico
- Prior art keywords
- methods
- fabry disease
- compositions
- polynucleotides
- treating fabry
- Prior art date
Links
- 208000024720 Fabry Disease Diseases 0.000 title abstract 2
- 239000000203 mixture Substances 0.000 title 1
- 102000040430 polynucleotide Human genes 0.000 abstract 3
- 108091033319 polynucleotide Proteins 0.000 abstract 3
- 239000002157 polynucleotide Substances 0.000 abstract 3
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
Classifications
-
- 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)
- C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
-
- 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
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
-
- 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
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
-
- 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
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- 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)
- C12Y302/01022—Alpha-galactosidase (3.2.1.22)
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)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Physics & Mathematics (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163137235P | 2021-01-14 | 2021-01-14 | |
| US202163264356P | 2021-11-19 | 2021-11-19 | |
| PCT/US2022/070184 WO2022155665A1 (en) | 2021-01-14 | 2022-01-13 | Compositions and methods for treating fabry disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2023008362A true MX2023008362A (es) | 2023-10-04 |
Family
ID=82448637
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2023008362A MX2023008362A (es) | 2021-01-14 | 2022-01-13 | Composiciones y metodos para el tratamiento de la enfermedad de fabry. |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20240301380A1 (https=) |
| EP (1) | EP4277988A4 (https=) |
| JP (1) | JP2024504611A (https=) |
| KR (1) | KR20230131246A (https=) |
| AU (1) | AU2022207185A1 (https=) |
| CA (1) | CA3208153A1 (https=) |
| CL (1) | CL2023002015A1 (https=) |
| CO (1) | CO2023010599A2 (https=) |
| IL (1) | IL304273A (https=) |
| MX (1) | MX2023008362A (https=) |
| PE (1) | PE20240915A1 (https=) |
| WO (1) | WO2022155665A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4426846A4 (en) * | 2021-11-03 | 2025-11-05 | Sangamo Therapeutics Inc | METHODS OF USING VIRAL VECTOR CONSTRUCTIONS FOR THE TREATMENT OF FABRY DISEASE |
| KR20250161626A (ko) * | 2023-03-20 | 2025-11-17 | 스파크 테라퓨틱스, 인코포레이티드 | Ppt1 유전자 치료 |
| US20240360431A1 (en) * | 2023-04-30 | 2024-10-31 | Sichuan Real&Best Biotech Co., Ltd. | Engineered alpha-galactosidase a (a-gal a) peptides and functional variants thereof and associated methods of treating fabry disease |
| CN117551636B (zh) * | 2023-04-30 | 2024-11-08 | 四川至善唯新生物科技有限公司 | 工程化α-GAL A肽及其功能变体和治疗法布里病的相关方法 |
| WO2025264565A1 (en) * | 2024-06-17 | 2025-12-26 | Spark Therapeutics, Inc. | Promoters |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013151665A2 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins associated with human disease |
| WO2017201328A1 (en) * | 2016-05-18 | 2017-11-23 | Modernatx, Inc. | POLYNUCLEOTIDES ENCODING α-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE |
| KR102712926B1 (ko) * | 2016-10-20 | 2024-10-07 | 상가모 테라퓨틱스, 인코포레이티드 | 파브리병의 치료를 위한 방법 및 조성물 |
| MY207355A (en) * | 2018-05-16 | 2025-02-21 | Spark Therapeutics Inc | Codon-optimized acid lpha-glucosidase expression cassettes and methods of using same |
| KR20220022107A (ko) * | 2018-12-05 | 2022-02-24 | 아베오나 테라퓨틱스 인코퍼레이티드 | 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터 |
| US12163425B2 (en) * | 2020-03-13 | 2024-12-10 | Baker Hughes Oilfield Operations Llc | Automated geosteering based on a distance to oil-water contact |
-
2022
- 2022-01-13 MX MX2023008362A patent/MX2023008362A/es unknown
- 2022-01-13 KR KR1020237027400A patent/KR20230131246A/ko not_active Withdrawn
- 2022-01-13 CA CA3208153A patent/CA3208153A1/en active Pending
- 2022-01-13 PE PE2023002056A patent/PE20240915A1/es unknown
- 2022-01-13 JP JP2023542653A patent/JP2024504611A/ja not_active Withdrawn
- 2022-01-13 WO PCT/US2022/070184 patent/WO2022155665A1/en not_active Ceased
- 2022-01-13 US US18/261,285 patent/US20240301380A1/en active Pending
- 2022-01-13 AU AU2022207185A patent/AU2022207185A1/en not_active Abandoned
- 2022-01-13 EP EP22740258.3A patent/EP4277988A4/en not_active Withdrawn
-
2023
- 2023-07-05 IL IL304273A patent/IL304273A/en unknown
- 2023-07-11 CL CL2023002015A patent/CL2023002015A1/es unknown
- 2023-08-14 CO CONC2023/0010599A patent/CO2023010599A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| AU2022207185A9 (en) | 2024-07-25 |
| EP4277988A1 (en) | 2023-11-22 |
| WO2022155665A1 (en) | 2022-07-21 |
| US20240301380A1 (en) | 2024-09-12 |
| CL2023002015A1 (es) | 2024-03-08 |
| IL304273A (en) | 2023-09-01 |
| CO2023010599A2 (es) | 2023-09-18 |
| KR20230131246A (ko) | 2023-09-12 |
| EP4277988A4 (en) | 2024-12-11 |
| CA3208153A1 (en) | 2022-07-21 |
| JP2024504611A (ja) | 2024-02-01 |
| PE20240915A1 (es) | 2024-04-30 |
| AU2022207185A1 (en) | 2023-07-13 |
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