AR122289A1 - Terapia genética cardíaca con aav para la cardiomiopatía en humanos - Google Patents
Terapia genética cardíaca con aav para la cardiomiopatía en humanosInfo
- Publication number
- AR122289A1 AR122289A1 ARP200102021A ARP200102021A AR122289A1 AR 122289 A1 AR122289 A1 AR 122289A1 AR P200102021 A ARP200102021 A AR P200102021A AR P200102021 A ARP200102021 A AR P200102021A AR 122289 A1 AR122289 A1 AR 122289A1
- Authority
- AR
- Argentina
- Prior art keywords
- raav
- compositions
- vector
- heart
- aav
- Prior art date
Links
- 230000000747 cardiac effect Effects 0.000 title abstract 3
- 238000001415 gene therapy Methods 0.000 title abstract 2
- 208000031229 Cardiomyopathies Diseases 0.000 title 1
- 239000000203 mixture Substances 0.000 abstract 5
- 238000000034 method Methods 0.000 abstract 4
- 102000021350 Caspase recruitment domains Human genes 0.000 abstract 3
- 108091011189 Caspase recruitment domains Proteins 0.000 abstract 3
- 239000002245 particle Substances 0.000 abstract 3
- 241000702421 Dependoparvovirus Species 0.000 abstract 2
- 108700019146 Transgenes Proteins 0.000 abstract 2
- 239000013598 vector Substances 0.000 abstract 2
- 239000013607 AAV vector Substances 0.000 abstract 1
- 241000282412 Homo Species 0.000 abstract 1
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 1
- 108700031547 S100A1 Proteins 0.000 abstract 1
- 230000001640 apoptogenic effect Effects 0.000 abstract 1
- 230000006907 apoptotic process Effects 0.000 abstract 1
- 210000000234 capsid Anatomy 0.000 abstract 1
- 239000002299 complementary DNA Substances 0.000 abstract 1
- 208000019622 heart disease Diseases 0.000 abstract 1
- 239000003112 inhibitor Substances 0.000 abstract 1
- 108091033319 polynucleotide Proteins 0.000 abstract 1
- 239000002157 polynucleotide Substances 0.000 abstract 1
- 102000040430 polynucleotide Human genes 0.000 abstract 1
- 239000013608 rAAV vector Substances 0.000 abstract 1
- 230000002195 synergetic effect Effects 0.000 abstract 1
- 230000008685 targeting Effects 0.000 abstract 1
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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
-
- 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
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
- 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
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4728—Calcium binding proteins, e.g. calmodulin
-
- 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)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6472—Cysteine endopeptidases (3.4.22)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/22—Cysteine endopeptidases (3.4.22)
- C12Y304/22061—Caspase-8 (3.4.22.61)
-
- 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
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
- C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Biophysics (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Toxicology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Marine Sciences & Fisheries (AREA)
- Immunology (AREA)
- Mycology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962876540P | 2019-07-19 | 2019-07-19 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR122289A1 true AR122289A1 (es) | 2022-08-31 |
Family
ID=74193792
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP200102021A AR122289A1 (es) | 2019-07-19 | 2020-07-17 | Terapia genética cardíaca con aav para la cardiomiopatía en humanos |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US20220265858A1 (OSRAM) |
| EP (1) | EP3999074A4 (OSRAM) |
| JP (1) | JP2022541793A (OSRAM) |
| KR (1) | KR20220034801A (OSRAM) |
| CN (1) | CN114206351A (OSRAM) |
| AR (1) | AR122289A1 (OSRAM) |
| AU (1) | AU2020316339A1 (OSRAM) |
| BR (1) | BR112022000898A2 (OSRAM) |
| CA (1) | CA3142534A1 (OSRAM) |
| CL (1) | CL2022000115A1 (OSRAM) |
| CO (1) | CO2022001686A2 (OSRAM) |
| IL (1) | IL289667A (OSRAM) |
| MX (1) | MX2022000767A (OSRAM) |
| SG (1) | SG11202112140SA (OSRAM) |
| TW (1) | TW202117017A (OSRAM) |
| WO (1) | WO2021016126A1 (OSRAM) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2024501957A (ja) * | 2020-12-23 | 2024-01-17 | ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． | 心臓遺伝子治療のためのベクターの増大したパッケージング効率 |
| WO2023147304A1 (en) | 2022-01-25 | 2023-08-03 | The Trustees Of The University Of Pennsylvania | Aav capsids for improved heart transduction and detargeting of liver |
| WO2024130070A2 (en) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof |
| WO2024130067A2 (en) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same |
| AR133098A1 (es) | 2023-06-29 | 2025-08-27 | Univ Pennsylvania | Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen |
| WO2025106661A1 (en) | 2023-11-14 | 2025-05-22 | The Trustees Of The University Of Pennsylvania | Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE19915485A1 (de) * | 1999-04-07 | 2000-10-19 | Hugo A Katus | Therapie der Herzinsuffizienz |
| CA2412847A1 (en) * | 2000-06-30 | 2002-01-10 | Collateral Therapeutics, Inc. | Dual recombinant gene therapy compositions and methods of use |
| US8383601B2 (en) * | 2006-10-30 | 2013-02-26 | Thomas Jefferson University | Tissue specific gene therapy treatment |
| EP2295072A1 (en) * | 2009-09-15 | 2011-03-16 | Max-Delbrück-Centrum für Molekulare Medizin (MDC) | Use of ARC for inhibiting cell death during liver failure |
| WO2015049277A1 (en) * | 2013-10-01 | 2015-04-09 | Ruprecht-Karls-Universität Heidelberg | S100 based treatment of cardiac power failure |
| WO2017191274A2 (en) * | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
| WO2019073058A1 (en) * | 2017-10-12 | 2019-04-18 | Universität Heidelberg | GENE THERAPY OF CARDIAC ARRHYTHMIA BASED ON KCNK3 |
| SG11202011061SA (en) * | 2018-06-08 | 2020-12-30 | University Of Florida Research Foundation Incorporated | Aav cardiac gene therapy for cardiomyopathy |
-
2020
- 2020-07-17 WO PCT/US2020/042663 patent/WO2021016126A1/en not_active Ceased
- 2020-07-17 BR BR112022000898A patent/BR112022000898A2/pt unknown
- 2020-07-17 SG SG11202112140SA patent/SG11202112140SA/en unknown
- 2020-07-17 JP JP2022503802A patent/JP2022541793A/ja active Pending
- 2020-07-17 CN CN202080052456.6A patent/CN114206351A/zh active Pending
- 2020-07-17 CA CA3142534A patent/CA3142534A1/en active Pending
- 2020-07-17 MX MX2022000767A patent/MX2022000767A/es unknown
- 2020-07-17 US US17/628,139 patent/US20220265858A1/en active Pending
- 2020-07-17 EP EP20844818.3A patent/EP3999074A4/en active Pending
- 2020-07-17 KR KR1020227002673A patent/KR20220034801A/ko not_active Ceased
- 2020-07-17 AR ARP200102021A patent/AR122289A1/es not_active Application Discontinuation
- 2020-07-17 TW TW109124287A patent/TW202117017A/zh unknown
- 2020-07-17 AU AU2020316339A patent/AU2020316339A1/en not_active Abandoned
-
2022
- 2022-01-06 IL IL289667A patent/IL289667A/en unknown
- 2022-01-17 CL CL2022000115A patent/CL2022000115A1/es unknown
- 2022-02-17 CO CONC2022/0001686A patent/CO2022001686A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| US20220265858A1 (en) | 2022-08-25 |
| AU2020316339A2 (en) | 2021-11-25 |
| TW202117017A (zh) | 2021-05-01 |
| EP3999074A1 (en) | 2022-05-25 |
| SG11202112140SA (en) | 2021-11-29 |
| EP3999074A4 (en) | 2023-08-16 |
| WO2021016126A8 (en) | 2021-09-10 |
| CA3142534A1 (en) | 2021-01-28 |
| CL2022000115A1 (es) | 2022-10-07 |
| BR112022000898A2 (pt) | 2022-06-07 |
| JP2022541793A (ja) | 2022-09-27 |
| MX2022000767A (es) | 2022-04-25 |
| IL289667A (en) | 2022-03-01 |
| KR20220034801A (ko) | 2022-03-18 |
| CN114206351A (zh) | 2022-03-18 |
| WO2021016126A1 (en) | 2021-01-28 |
| CO2022001686A2 (es) | 2022-03-29 |
| AU2020316339A1 (en) | 2021-11-18 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FA | Abandonment or withdrawal |