CO2023006811A2 - Mayor eficiencia del empaquetamiento de vectores para terapia génica cardíaca - Google Patents
Mayor eficiencia del empaquetamiento de vectores para terapia génica cardíacaInfo
- Publication number
- CO2023006811A2 CO2023006811A2 CONC2023/0006811A CO2023006811A CO2023006811A2 CO 2023006811 A2 CO2023006811 A2 CO 2023006811A2 CO 2023006811 A CO2023006811 A CO 2023006811A CO 2023006811 A2 CO2023006811 A2 CO 2023006811A2
- Authority
- CO
- Colombia
- Prior art keywords
- compositions
- methods
- cardiac
- gene therapy
- carc
- Prior art date
Links
- 230000000747 cardiac effect Effects 0.000 title abstract 3
- 239000013598 vector Substances 0.000 title abstract 3
- 238000001415 gene therapy Methods 0.000 title abstract 2
- 238000004806 packaging method and process Methods 0.000 title abstract 2
- 238000000034 method Methods 0.000 abstract 4
- 239000000203 mixture Substances 0.000 abstract 4
- 108700019146 Transgenes Proteins 0.000 abstract 2
- 239000013607 AAV vector Substances 0.000 abstract 1
- 229940088872 Apoptosis inhibitor Drugs 0.000 abstract 1
- 102000011727 Caspases Human genes 0.000 abstract 1
- 108010076667 Caspases Proteins 0.000 abstract 1
- 108700031547 S100A1 Proteins 0.000 abstract 1
- 239000000158 apoptosis inhibitor Substances 0.000 abstract 1
- 239000002299 complementary DNA Substances 0.000 abstract 1
- 230000007115 recruitment Effects 0.000 abstract 1
- 230000002195 synergetic effect Effects 0.000 abstract 1
- 230000008685 targeting Effects 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
- 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
-
- 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
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
-
- 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
- C07K14/4702—Regulators; Modulating activity
- C07K14/4703—Inhibitors; Suppressors
-
- 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
- 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
- C07K14/4747—Apoptosis related proteins
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
-
- 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
- C12N2830/50—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
- 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)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Gastroenterology & Hepatology (AREA)
- Biomedical Technology (AREA)
- Toxicology (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Hospice & Palliative Care (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Ultra Sonic Daignosis Equipment (AREA)
- Electrotherapy Devices (AREA)
Abstract
La presente descripción se relaciona con composiciones y métodos útiles en el tratamiento de afecciones cardíacas. Las composiciones y los métodos descritos se basan en terapias génicas que comprenden un vector AAV recombinante para administrar dos o más transgenes en el corazón de un sujeto, donde los transgenes codifican una proteína S100A1 y un inhibidor del Represor de la Apoptosis Cardiaca con Dominio de Reclutamiento de Caspasa (cARC), respectivamente. En diversas realizaciones, las composiciones y los métodos descritos en este documento comprenden vectores que comprenden secuencias de ADNc de S100A1 y/o cARC que tienen codones optimizados para la expresión en seres humanos. En diversas realizaciones, las composiciones y los métodos descritos en este documento comprenden vectores con eficiencias de empaquetamiento mejoradas. En algunos aspectos, enfocarse en múltiples fuentes de una o más afecciones cardíacas puede brindar beneficios sinérgicos durante el tratamiento.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202063130109P | 2020-12-23 | 2020-12-23 | |
PCT/US2021/064637 WO2022140402A1 (en) | 2020-12-23 | 2021-12-21 | Increased packaging efficiency of vector for cardiac gene therapy |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2023006811A2 true CO2023006811A2 (es) | 2023-05-29 |
Family
ID=80445607
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2023/0006811A CO2023006811A2 (es) | 2020-12-23 | 2023-05-25 | Mayor eficiencia del empaquetamiento de vectores para terapia génica cardíaca |
Country Status (14)
Country | Link |
---|---|
US (1) | US20240139343A1 (es) |
EP (1) | EP4267749A1 (es) |
JP (1) | JP2024501957A (es) |
KR (1) | KR20230124979A (es) |
CN (1) | CN116867512A (es) |
AR (1) | AR124506A1 (es) |
AU (1) | AU2021409806A1 (es) |
CA (1) | CA3205116A1 (es) |
CL (1) | CL2023001731A1 (es) |
CO (1) | CO2023006811A2 (es) |
IL (1) | IL303860A (es) |
MX (1) | MX2023007602A (es) |
TW (1) | TW202242120A (es) |
WO (1) | WO2022140402A1 (es) |
Family Cites Families (8)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
US5962313A (en) | 1996-01-18 | 1999-10-05 | Avigen, Inc. | Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme |
US20070015238A1 (en) | 2002-06-05 | 2007-01-18 | Snyder Richard O | Production of pseudotyped recombinant AAV virions |
US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
US20200308582A1 (en) * | 2017-10-12 | 2020-10-01 | Universität Heidelberg | Kcnk3-based gene therapy of cardiac arrhythmia |
BR112020024935A2 (pt) | 2018-06-08 | 2021-03-09 | University Of Florida Research Foundation, Incorporated | Terapia gênica cardíaca com aav para cardiomiopatia |
AU2020229382A1 (en) * | 2019-02-28 | 2021-09-16 | Encoded Therapeutics, Inc. | Compositions and methods for treating laminopathies |
CN114206351A (zh) * | 2019-07-19 | 2022-03-18 | 佛罗里达大学研究基金会有限公司 | 用于人中心肌病的aav心脏基因治疗 |
-
2021
- 2021-12-21 CA CA3205116A patent/CA3205116A1/en active Pending
- 2021-12-21 US US18/269,089 patent/US20240139343A1/en active Pending
- 2021-12-21 WO PCT/US2021/064637 patent/WO2022140402A1/en active Application Filing
- 2021-12-21 IL IL303860A patent/IL303860A/en unknown
- 2021-12-21 EP EP21854906.1A patent/EP4267749A1/en active Pending
- 2021-12-21 KR KR1020237024492A patent/KR20230124979A/ko unknown
- 2021-12-21 MX MX2023007602A patent/MX2023007602A/es unknown
- 2021-12-21 AU AU2021409806A patent/AU2021409806A1/en active Pending
- 2021-12-21 CN CN202180086464.7A patent/CN116867512A/zh active Pending
- 2021-12-21 JP JP2023538963A patent/JP2024501957A/ja active Pending
- 2021-12-22 TW TW110148254A patent/TW202242120A/zh unknown
- 2021-12-23 AR ARP210103661A patent/AR124506A1/es unknown
-
2023
- 2023-05-25 CO CONC2023/0006811A patent/CO2023006811A2/es unknown
- 2023-06-14 CL CL2023001731A patent/CL2023001731A1/es unknown
Also Published As
Publication number | Publication date |
---|---|
EP4267749A1 (en) | 2023-11-01 |
KR20230124979A (ko) | 2023-08-28 |
CL2023001731A1 (es) | 2023-12-15 |
JP2024501957A (ja) | 2024-01-17 |
AU2021409806A9 (en) | 2024-02-08 |
TW202242120A (zh) | 2022-11-01 |
US20240139343A1 (en) | 2024-05-02 |
CN116867512A (zh) | 2023-10-10 |
CA3205116A1 (en) | 2022-06-30 |
AU2021409806A1 (en) | 2023-06-15 |
AR124506A1 (es) | 2023-04-05 |
WO2022140402A1 (en) | 2022-06-30 |
IL303860A (en) | 2023-08-01 |
MX2023007602A (es) | 2023-09-21 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
CL2022000115A1 (es) | Terapia genética cardíaca con aav para la cardiomiopatía en humanos. | |
CO2020016718A2 (es) | Terapia génica cardíaca con aav para cardiomiopatía | |
CO2021008120A2 (es) | Vectores virales recombinantes y ácidos nucleicos para producirlos | |
CL2022000900A1 (es) | Proteínas heterodiméricas fusionadas con fc. (divisional de solicitud 202101058) | |
BR112018002046A2 (pt) | métodos de tratamento da síndrome de lennox-gastaut usando fenfluramina | |
Grzech-Lesniak | Making use of lasers in periodontal treatment: a new gold standard | |
PE20181922A1 (es) | Terapia genica para tratar la hemofilia a | |
AR116700A1 (es) | Generación de una mejor fenilalanina hidroxilasa hepática humana para el tratamiento de la fenilcetonuria severa por terapia génica de reemplazo directo en el hígado | |
ES2722623T3 (es) | Conjuntos de adaptador para la interconexión de conjuntos de mango electromecánico y unidades de carga quirúrgica | |
CO2023006811A2 (es) | Mayor eficiencia del empaquetamiento de vectores para terapia génica cardíaca | |
AR118997A1 (es) | Partículas virales modificadas y usos de estas | |
NI201500057A (es) | Métodos para reducir riesgos asociados con insuficiencia cardiaca y factores asociados | |
BR112022007324A2 (pt) | Dispositivos ou métodos para modular atividade de sistema imunológico em um paciente com câncer ou tratar câncer | |
ECSP20055797A (es) | Métodos y composiciones para tratar enfermedades pulmonares crónicas | |
CO2021016487A2 (es) | Vectores aav con el promotor de la proteína cero de mielina y usos de los mismos para el tratamiento de enfermedades asociadas a las células de schwann como la enfermedad de charcot-marie-tooth | |
JP2021514725A5 (es) | ||
CL2023001876A1 (es) | Proteínas de la cápside viral con especificidad para las células del tejido cardiaco | |
AR117877A1 (es) | Rhabdovirus recombinante que codifica ccl21 | |
Kazemikhoo et al. | Effects of photobiomodulation and split-thickness skin grafting in the prognosis of wound healing in children with deep burn ulcers | |
EP4171411A1 (en) | Split biphasic waveform for embolic reduction | |
ES2654845A1 (es) | Dispositivo que combina tecnología laser con ozono y su uso en un método de tratamiento de infecciones micóticas | |
Wang et al. | Comparison of rectilinear biphasic waveform with biphasic truncated exponential waveform in a pediatric defibrillation model | |
Wollina et al. | The use of biophysical technologies in chronic wound management | |
Na et al. | Low-level green and red laser treatment of Shaochong (HT9)· Dadun (LR1) and Shaohai (HT3)· Yingu (KI10) acupoints in a rat model of focal cerebral ischemia | |
JP2022099207A (ja) | 貧血治療薬 |