MX2021008503A - Tratamiento de enfermedades que involucran deficiencia de enpp1 o enpp3. - Google Patents
Tratamiento de enfermedades que involucran deficiencia de enpp1 o enpp3.Info
- Publication number
- MX2021008503A MX2021008503A MX2021008503A MX2021008503A MX2021008503A MX 2021008503 A MX2021008503 A MX 2021008503A MX 2021008503 A MX2021008503 A MX 2021008503A MX 2021008503 A MX2021008503 A MX 2021008503A MX 2021008503 A MX2021008503 A MX 2021008503A
- Authority
- MX
- Mexico
- Prior art keywords
- enpp1
- enpp3
- treatment
- diseases involving
- deficiency
- Prior art date
Links
- 201000010099 disease Diseases 0.000 title abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title abstract 2
- 230000007812 deficiency Effects 0.000 title 1
- 101000812677 Homo sapiens Nucleotide pyrophosphatase Proteins 0.000 abstract 1
- 101000897042 Homo sapiens Nucleotide pyrophosphatase Proteins 0.000 abstract 1
- 102100021969 Nucleotide pyrophosphatase Human genes 0.000 abstract 1
- 102100039306 Nucleotide pyrophosphatase Human genes 0.000 abstract 1
- 230000002308 calcification Effects 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 230000011164 ossification Effects 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
-
- 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)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
- A01K67/02—Breeding vertebrates
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
- A01K67/027—New or modified breeds of vertebrates
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/04—Phosphoric diester hydrolases (3.1.4)
- C12Y301/04001—Phosphodiesterase I (3.1.4.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
- C12Y306/01—Hydrolases acting on acid anhydrides (3.6) in phosphorus-containing anhydrides (3.6.1)
- C12Y306/01009—Nucleotide diphosphatase (3.6.1.9), i.e. nucleotide-pyrophosphatase
-
- 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
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/31—Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
-
- 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
- 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
- C12N2750/14151—Methods of production or purification of viral material
-
- 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
- C12N2750/14171—Demonstrated in vivo effect
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Molecular Biology (AREA)
- Virology (AREA)
- Immunology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Environmental Sciences (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physical Education & Sports Medicine (AREA)
- Urology & Nephrology (AREA)
- Cell Biology (AREA)
- Gastroenterology & Hepatology (AREA)
- Biodiversity & Conservation Biology (AREA)
- Animal Husbandry (AREA)
- Rheumatology (AREA)
- Orthopedic Medicine & Surgery (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962794450P | 2019-01-18 | 2019-01-18 | |
| US201962821692P | 2019-03-21 | 2019-03-21 | |
| US201962877044P | 2019-07-22 | 2019-07-22 | |
| PCT/US2020/014296 WO2020150716A1 (en) | 2019-01-18 | 2020-01-20 | Treatment of diseases involving deficiency of enpp1 or enpp3 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2021008503A true MX2021008503A (es) | 2021-10-13 |
Family
ID=71613451
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2021008503A MX2021008503A (es) | 2019-01-18 | 2020-01-20 | Tratamiento de enfermedades que involucran deficiencia de enpp1 o enpp3. |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US20210187067A1 (https=) |
| EP (1) | EP3911153A4 (https=) |
| JP (2) | JP7724156B2 (https=) |
| KR (1) | KR20210142599A (https=) |
| CN (1) | CN113631033B (https=) |
| AU (1) | AU2020207967A1 (https=) |
| BR (1) | BR112021013941A2 (https=) |
| CA (1) | CA3126839A1 (https=) |
| IL (1) | IL284699A (https=) |
| MX (1) | MX2021008503A (https=) |
| WO (1) | WO2020150716A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2022076848A1 (en) * | 2020-10-08 | 2022-04-14 | Inozyme Pharma, Inc. | Liver specific production of enpp1 or enpp3 |
| AU2021383830A1 (en) * | 2020-11-19 | 2023-06-22 | Biomarin Pharmaceutical Inc. | Treatment of enpp1 deficiency and abcc6 deficiency |
| WO2023049864A1 (en) * | 2021-09-24 | 2023-03-30 | Inozyme Pharma, Inc. | Lyophilized enpp1 polypeptide formulations and uses thereof |
| KR20240167914A (ko) * | 2022-04-04 | 2024-11-28 | 이노자임 파마, 인코포레이티드 | Enpp1 결핍증 및 abcc6 결핍증의 치료 |
| WO2025213139A1 (en) * | 2024-04-04 | 2025-10-09 | The General Hospital Corporation | Enpp1 gene therapy for the treatment of vascular disease |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6894022B1 (en) * | 1998-08-27 | 2005-05-17 | Eidgenossische Technische Hochschule Zurich | Growth factor modified protein matrices for tissue engineering |
| CA2462653C (en) * | 2001-11-07 | 2016-06-07 | Aya Jakobovits | Nucleic acid and corresponding protein entitled 161p2f10b useful in treatment and detection of cancer |
| EP1736553A1 (en) * | 2005-06-17 | 2006-12-27 | Centre National De La Recherche Scientifique | ENPP1 (PC-1) gene haplotype associated with the risk of obesity and type 2 diabetes and their applications |
| US8168586B1 (en) * | 2007-11-21 | 2012-05-01 | Celera Corporation | Cancer targets and uses thereof |
| WO2010037408A1 (en) * | 2008-09-30 | 2010-04-08 | Curevac Gmbh | Composition comprising a complexed (m)rna and a naked mrna for providing or enhancing an immunostimulatory response in a mammal and uses thereof |
| EP3470089A1 (en) * | 2013-12-12 | 2019-04-17 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components |
| CA2960912A1 (en) * | 2014-09-16 | 2016-03-24 | Universitat Autonoma De Barcelona | Adeno-associated viral vectors for the gene therapy of metabolic diseases |
| EP3234116B1 (en) * | 2014-12-19 | 2021-09-08 | Alexion Pharmaceuticals, Inc. | Methods of treating tissue calcification |
| KR20250078598A (ko) * | 2015-05-19 | 2025-06-02 | 예일 유니버시티 | 병리학적 석회화 병태를 치료하기 위한 조성물, 및 이의 사용 방법 |
| BR112019002355A2 (pt) * | 2016-08-05 | 2019-07-16 | Yale University | composições e métodos para prevenção de acidente vascular cerebral em pacientes pediátricos com anemia falciforme |
| EP3844280A4 (en) * | 2018-08-31 | 2022-09-14 | Yale University | ENPP1 POLYPEPTIDES AND METHODS OF USE |
-
2020
- 2020-01-20 KR KR1020217025330A patent/KR20210142599A/ko not_active Ceased
- 2020-01-20 CA CA3126839A patent/CA3126839A1/en active Pending
- 2020-01-20 JP JP2021541712A patent/JP7724156B2/ja active Active
- 2020-01-20 WO PCT/US2020/014296 patent/WO2020150716A1/en not_active Ceased
- 2020-01-20 CN CN202080009084.9A patent/CN113631033B/zh active Active
- 2020-01-20 EP EP20741209.9A patent/EP3911153A4/en active Pending
- 2020-01-20 MX MX2021008503A patent/MX2021008503A/es unknown
- 2020-01-20 AU AU2020207967A patent/AU2020207967A1/en active Pending
- 2020-01-20 BR BR112021013941-9A patent/BR112021013941A2/pt unknown
-
2021
- 2021-02-17 US US17/178,127 patent/US20210187067A1/en not_active Abandoned
- 2021-07-08 IL IL284699A patent/IL284699A/en unknown
-
2022
- 2022-08-25 US US17/822,245 patent/US20230031809A1/en active Pending
-
2025
- 2025-02-21 JP JP2025027352A patent/JP2025084821A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| IL284699A (en) | 2021-08-31 |
| BR112021013941A2 (pt) | 2021-09-21 |
| CA3126839A1 (en) | 2020-07-23 |
| CN113631033A (zh) | 2021-11-09 |
| US20210187067A1 (en) | 2021-06-24 |
| EP3911153A4 (en) | 2022-12-21 |
| US20230031809A1 (en) | 2023-02-02 |
| CN113631033B (zh) | 2024-10-15 |
| JP2022517435A (ja) | 2022-03-08 |
| WO2020150716A1 (en) | 2020-07-23 |
| JP2025084821A (ja) | 2025-06-03 |
| EP3911153A1 (en) | 2021-11-24 |
| KR20210142599A (ko) | 2021-11-25 |
| JP7724156B2 (ja) | 2025-08-15 |
| AU2020207967A1 (en) | 2021-07-29 |
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