MX2023009030A - Composiciones y métodos para el tratamiento de enfermedad de niemann pick tipo a. - Google Patents
Composiciones y métodos para el tratamiento de enfermedad de niemann pick tipo a.Info
- Publication number
- MX2023009030A MX2023009030A MX2023009030A MX2023009030A MX2023009030A MX 2023009030 A MX2023009030 A MX 2023009030A MX 2023009030 A MX2023009030 A MX 2023009030A MX 2023009030 A MX2023009030 A MX 2023009030A MX 2023009030 A MX2023009030 A MX 2023009030A
- Authority
- MX
- Mexico
- Prior art keywords
- compositions
- disease
- treatment
- methods
- pick type
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 3
- 201000010099 disease Diseases 0.000 title abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title abstract 2
- 239000013598 vector Substances 0.000 abstract 3
- 101000785978 Homo sapiens Sphingomyelin phosphodiesterase Proteins 0.000 abstract 2
- 108091026890 Coding region Proteins 0.000 abstract 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 102100026263 Sphingomyelin phosphodiesterase Human genes 0.000 abstract 1
- 102000048866 human SMPD1 Human genes 0.000 abstract 1
- 108091033319 polynucleotide Proteins 0.000 abstract 1
- 102000040430 polynucleotide Human genes 0.000 abstract 1
- 239000002157 polynucleotide Substances 0.000 abstract 1
- 230000001105 regulatory effect Effects 0.000 abstract 1
Classifications
-
- 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
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
-
- 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)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
-
- 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)
- C12Y301/04012—Sphingomyelin phosphodiesterase (3.1.4.12)
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- 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
- C12N2830/42—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
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)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Dermatology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163144103P | 2021-02-01 | 2021-02-01 | |
| US202163286939P | 2021-12-07 | 2021-12-07 | |
| PCT/US2022/014742 WO2022165421A1 (en) | 2021-02-01 | 2022-02-01 | Compositions and methods for treatment of niemann pick type a disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2023009030A true MX2023009030A (es) | 2023-10-16 |
Family
ID=82654923
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2023009030A MX2023009030A (es) | 2021-02-01 | 2022-02-01 | Composiciones y métodos para el tratamiento de enfermedad de niemann pick tipo a. |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20240115733A1 (https=) |
| EP (1) | EP4284442A4 (https=) |
| JP (1) | JP2024506860A (https=) |
| AU (1) | AU2022214529A1 (https=) |
| CA (1) | CA3205351A1 (https=) |
| MX (1) | MX2023009030A (https=) |
| WO (1) | WO2022165421A1 (https=) |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5773278A (en) * | 1991-05-03 | 1998-06-30 | Mount Sinai Medical Center | Acid sphingomyelinase gene |
| EP1620133B1 (en) * | 2003-05-01 | 2015-12-09 | Genzyme Corporation | Gene therapy for neurometabolic disorders |
| US7527956B2 (en) * | 2003-06-12 | 2009-05-05 | Genzyme Corporation | Modified human acid sphingomyelinase having increased activity, and methods for making the same |
| AR059089A1 (es) * | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| AR059371A1 (es) * | 2006-02-08 | 2008-03-26 | Genzyme Corp | Terapia genica para la enfermedad de niemann-pick tipo a |
| ES2635726T3 (es) * | 2007-06-06 | 2017-10-04 | Genzyme Corporation | Terapia génica para enfermedades de almacenamiento lisosómico |
| MX2018012537A (es) * | 2016-04-15 | 2019-02-25 | Univ Pennsylvania | Terapia de genes para tratar hemofilia a. |
| WO2017191274A2 (en) * | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
| BR112020006661A2 (pt) * | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| PY1939780A (es) * | 2018-05-25 | 2019-12-04 | Genzyme Corp | Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida |
-
2022
- 2022-02-01 EP EP22746872.5A patent/EP4284442A4/en active Pending
- 2022-02-01 MX MX2023009030A patent/MX2023009030A/es unknown
- 2022-02-01 WO PCT/US2022/014742 patent/WO2022165421A1/en not_active Ceased
- 2022-02-01 AU AU2022214529A patent/AU2022214529A1/en active Pending
- 2022-02-01 JP JP2023546548A patent/JP2024506860A/ja active Pending
- 2022-02-01 US US18/263,085 patent/US20240115733A1/en active Pending
- 2022-02-01 CA CA3205351A patent/CA3205351A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2022165421A1 (en) | 2022-08-04 |
| CA3205351A1 (en) | 2022-08-04 |
| JP2024506860A (ja) | 2024-02-15 |
| AU2022214529A1 (en) | 2023-08-03 |
| US20240115733A1 (en) | 2024-04-11 |
| EP4284442A1 (en) | 2023-12-06 |
| EP4284442A4 (en) | 2025-04-23 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| WO2022076803A8 (en) | Compositions and methods for treatment of fabry disease | |
| ZA202300505B (en) | Compositions useful for treatment of charcot-marie-tooth disease | |
| MY209860A (en) | Gene therapy vectors for treating heart disease | |
| SA519402565B1 (ar) | F ناقل فرع حيوي لفيروس مرتبط بالغدة جديد واستخداماته | |
| SA519402566B1 (ar) | تركيبات نافعة في معالجة ضمور العضل النخاعي | |
| CL2022003757A1 (es) | Vectores de aav que tienen una desamidación reducida de la cápside y sus usos (divisional solic 202002200). | |
| WO2021163357A3 (en) | Gene therapy vectors for treating heart disease | |
| NZ603348A (en) | Simian adenovirus nucleic acid- and amino acid-sequences, vectors containing same, and uses thereof | |
| MX2021013420A (es) | Composiciones utiles en el tratamiento de leucodistrofia metacromatica. | |
| MX2021010356A (es) | SUMINISTRO DE ß-SARCOGLICANO MEDIANTE VECTORES DE VIRUS ADENOASOCIADO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR. | |
| JPWO2019156137A5 (https=) | ||
| MX2021005345A (es) | Composiciones y metodos. | |
| WO2020219766A8 (en) | Compositions useful in treatment of rett syndrome | |
| MX2025009545A (es) | Metodos y composiciones para la administracion de sistemas de vector dual otoferlin | |
| WO2023205300A3 (en) | Therapeutic adeno-associated virus using codon optimized nucleic acid encoding factor viii | |
| MX2025012376A (es) | Métodos y composiciones farmacéuticas de apoe para el tratamiento y la prevención de enfermedad de alzheimer | |
| MX2023009030A (es) | Composiciones y métodos para el tratamiento de enfermedad de niemann pick tipo a. | |
| CO2025004844A2 (es) | Vectores de aav recombinantes para tratamiento de distrofia muscular | |
| MX2022007546A (es) | Interferon-alfa-2 modificado que tiene inmunogenicidad reducida. | |
| RU2019100990A (ru) | Укороченный фисферлин для лечения фисферлинопатии | |
| BR0318594A (pt) | proteìna recombinante que possui um efeito anticancerìgeno, seu gene de codificação e usos desta | |
| MX2025000200A (es) | Metodos y composiciones farmaceuticas para el tratamiento y la prevencion de la enfermedad de alzheimer | |
| MX2024004398A (es) | Peptidos y fragmentos para tratar diabetes y enfermedades metabolicas asociadas. | |
| WO2021150713A3 (en) | Human immunogenic epitopes of h, k, and e human endogenous retroviruses (hervs) | |
| RU2006119452A (ru) | Рекомбинантный белок, обладающий противораковым действием, кодирующий его ген и его применение |