CL2024002307A1 - Composiciones y métodos para el tratamiento mejorado de la enfermedad de pompe. - Google Patents
Composiciones y métodos para el tratamiento mejorado de la enfermedad de pompe.Info
- Publication number
- CL2024002307A1 CL2024002307A1 CL2024002307A CL2024002307A CL2024002307A1 CL 2024002307 A1 CL2024002307 A1 CL 2024002307A1 CL 2024002307 A CL2024002307 A CL 2024002307A CL 2024002307 A CL2024002307 A CL 2024002307A CL 2024002307 A1 CL2024002307 A1 CL 2024002307A1
- Authority
- CL
- Chile
- Prior art keywords
- methods
- pompe disease
- compositions
- improved treatment
- provides methods
- Prior art date
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
- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
- A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
- A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
-
- 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
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- 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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
- 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)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Toxicology (AREA)
- Diabetes (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Hematology (AREA)
- Emergency Medicine (AREA)
- Endocrinology (AREA)
- Obesity (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263306361P | 2022-02-03 | 2022-02-03 | |
| US202263402850P | 2022-08-31 | 2022-08-31 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2024002307A1 true CL2024002307A1 (es) | 2024-12-27 |
Family
ID=87552969
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2024002307A CL2024002307A1 (es) | 2022-02-03 | 2024-07-31 | Composiciones y métodos para el tratamiento mejorado de la enfermedad de pompe. |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20250135038A1 (https=) |
| EP (1) | EP4472658A4 (https=) |
| JP (1) | JP2025505575A (https=) |
| KR (1) | KR20240145491A (https=) |
| AU (1) | AU2023216268A1 (https=) |
| CA (1) | CA3250992A1 (https=) |
| CL (1) | CL2024002307A1 (https=) |
| IL (1) | IL314586A (https=) |
| MX (1) | MX2024009519A (https=) |
| TW (1) | TW202342092A (https=) |
| WO (1) | WO2023150688A2 (https=) |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2003224880A1 (en) * | 2002-04-05 | 2003-10-27 | Genzyme Corporation | Methods of enhancing lysosomal storage disease therapy |
| WO2021081338A1 (en) * | 2019-10-25 | 2021-04-29 | Audentes Therapeutics, Inc. | Compositions and methods for treating glycogen storage disorders |
| CA3159018A1 (en) * | 2019-11-19 | 2021-05-27 | Michael W. O'CALLAGHAN | Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders |
| EP4103725A4 (en) * | 2020-02-14 | 2024-03-27 | Asklepios Biopharmaceutical, Inc. | Method of treating gene therapy associated toxicity with antibiotics |
| WO2021202599A2 (en) * | 2020-04-01 | 2021-10-07 | Valiant Biosciences, Llc | Adeno-associated virus based compositions and related methods for inducing humoral immunity |
-
2023
- 2023-02-03 KR KR1020247029043A patent/KR20240145491A/ko active Pending
- 2023-02-03 IL IL314586A patent/IL314586A/en unknown
- 2023-02-03 US US18/835,480 patent/US20250135038A1/en active Pending
- 2023-02-03 WO PCT/US2023/061951 patent/WO2023150688A2/en not_active Ceased
- 2023-02-03 CA CA3250992A patent/CA3250992A1/en active Pending
- 2023-02-03 JP JP2024545922A patent/JP2025505575A/ja active Pending
- 2023-02-03 EP EP23750442.8A patent/EP4472658A4/en active Pending
- 2023-02-03 TW TW112103875A patent/TW202342092A/zh unknown
- 2023-02-03 MX MX2024009519A patent/MX2024009519A/es unknown
- 2023-02-03 AU AU2023216268A patent/AU2023216268A1/en active Pending
-
2024
- 2024-07-31 CL CL2024002307A patent/CL2024002307A1/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| IL314586A (en) | 2024-09-01 |
| WO2023150688A8 (en) | 2023-11-16 |
| JP2025505575A (ja) | 2025-02-28 |
| WO2023150688A3 (en) | 2023-09-28 |
| EP4472658A2 (en) | 2024-12-11 |
| TW202342092A (zh) | 2023-11-01 |
| WO2023150688A2 (en) | 2023-08-10 |
| KR20240145491A (ko) | 2024-10-07 |
| CA3250992A1 (en) | 2023-08-10 |
| AU2023216268A1 (en) | 2024-08-15 |
| MX2024009519A (es) | 2024-08-14 |
| US20250135038A1 (en) | 2025-05-01 |
| EP4472658A4 (en) | 2026-01-14 |
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