MX2022016528A - Composiciones y métodos para el tratamiento de pacientes de terapia génica. - Google Patents
Composiciones y métodos para el tratamiento de pacientes de terapia génica.Info
- Publication number
- MX2022016528A MX2022016528A MX2022016528A MX2022016528A MX2022016528A MX 2022016528 A MX2022016528 A MX 2022016528A MX 2022016528 A MX2022016528 A MX 2022016528A MX 2022016528 A MX2022016528 A MX 2022016528A MX 2022016528 A MX2022016528 A MX 2022016528A
- Authority
- MX
- Mexico
- Prior art keywords
- gene therapy
- compositions
- treatment
- methods
- therapy patients
- Prior art date
Links
- 238000001415 gene therapy Methods 0.000 title abstract 3
- 239000000203 mixture Substances 0.000 title abstract 3
- 238000000034 method Methods 0.000 title 1
- 102100026120 IgG receptor FcRn large subunit p51 Human genes 0.000 abstract 2
- 101710177940 IgG receptor FcRn large subunit p51 Proteins 0.000 abstract 2
- 210000000234 capsid Anatomy 0.000 abstract 1
- 239000003446 ligand Substances 0.000 abstract 1
- 230000003472 neutralizing effect Effects 0.000 abstract 1
- 230000009870 specific binding Effects 0.000 abstract 1
- 230000003612 virological effect Effects 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
- C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
-
- 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
- A61K38/19—Cytokines; Lymphokines; Interferons
- A61K38/21—Interferons [IFN]
- A61K38/217—IFN-gamma
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
-
- 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
-
- 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
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
-
- 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
-
- 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
-
- 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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
- C07K14/70503—Immunoglobulin superfamily
- C07K14/70535—Fc-receptors, e.g. CD16, CD32, CD64 (CD2314/705F)
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
- C07K2317/55—Fab or Fab'
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
- C07K2317/56—Immunoglobulins specific features characterized by immunoglobulin fragments variable (Fv) region, i.e. VH and/or VL
- C07K2317/565—Complementarity determining region [CDR]
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
- C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
- C07K2317/622—Single chain antibody (scFv)
-
- 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10041—Use of virus, viral particle or viral elements as a vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Immunology (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Biotechnology (AREA)
- Gastroenterology & Hepatology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Dermatology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Mycology (AREA)
- Toxicology (AREA)
- Plant Pathology (AREA)
- Cell Biology (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
Se proporcionan composiciones útiles para su administración conjunta con un vector de terapia génica a un paciente que tenga anticuerpos neutralizantes preexistentes contra la fuente viral de la cápside del vector de terapia génica. Las composiciones comprenden un ligando FcRn que inhibe la unión específica entre FcRn e IgG.
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202063040381P | 2020-06-17 | 2020-06-17 | |
US202163135998P | 2021-01-11 | 2021-01-11 | |
US202163152085P | 2021-02-22 | 2021-02-22 | |
PCT/US2021/037575 WO2021257668A1 (en) | 2020-06-17 | 2021-06-16 | Compositions and methods for treatment of gene therapy patients |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2022016528A true MX2022016528A (es) | 2023-06-02 |
Family
ID=77127052
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2022016528A MX2022016528A (es) | 2020-06-17 | 2021-06-16 | Composiciones y métodos para el tratamiento de pacientes de terapia génica. |
Country Status (11)
Country | Link |
---|---|
US (1) | US20230220069A1 (es) |
EP (1) | EP4171738A1 (es) |
JP (1) | JP2023531451A (es) |
KR (1) | KR20230025000A (es) |
CN (1) | CN115968302A (es) |
AU (1) | AU2021292200A1 (es) |
CA (1) | CA3183153A1 (es) |
IL (1) | IL299167A (es) |
MX (1) | MX2022016528A (es) |
TW (1) | TW202214695A (es) |
WO (1) | WO2021257668A1 (es) |
Families Citing this family (8)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20220259308A1 (en) * | 2019-08-01 | 2022-08-18 | Janssen Biotech, Inc. | Fcrn antibodies and methods of use thereof |
WO2021151004A1 (en) * | 2020-01-22 | 2021-07-29 | Spark Therapeutics, Inc. | Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins |
EP4334334A1 (en) | 2021-04-23 | 2024-03-13 | The Trustees of The University of Pennsylvania | Novel compositions with brain-specific targeting motifs and compositions containing same |
TW202325845A (zh) | 2021-10-02 | 2023-07-01 | 賓州大學委員會 | 新穎aav衣殼及含其之組成物 |
WO2023196893A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating her2 positive metastatic breast cancer and other cancers |
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 |
CN117126270B (zh) * | 2023-10-25 | 2024-02-13 | 首都儿科研究所 | 一种2型人博卡病毒型别特异性抗体及其应用 |
Family Cites Families (34)
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US5786464C1 (en) | 1994-09-19 | 2012-04-24 | Gen Hospital Corp | Overexpression of mammalian and viral proteins |
US6506379B1 (en) | 1995-06-07 | 2003-01-14 | Ariad Gene Therapeutics, Inc. | Intramuscular delivery of recombinant AAV |
US6406861B1 (en) * | 1998-10-07 | 2002-06-18 | Cell Genesys, Inc. | Methods of enhancing effectiveness of therapeutic viral immunogenic agent administration |
CA2379166C (en) | 1999-08-09 | 2013-03-26 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs |
NZ600121A (en) | 2001-11-13 | 2013-12-20 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
SI2292780T1 (sl) | 2003-09-30 | 2017-12-29 | The Trustees Of The University Of Pennsylvania | Genotipske skupine, zaporedja, vektorji, ki vsebujejo le-te z adenovirusi povezanega virusa (aav) in njihova uporaba |
ES2525143T3 (es) | 2005-04-07 | 2014-12-18 | The Trustees Of The University Of Pennsylvania | Cápsides de AAVrh.64 modificado, composiciones que las contienen y usos de las mismas |
EP1879920A2 (en) * | 2005-04-29 | 2008-01-23 | The Jackson Laboratory | Fcrn antibodies and uses thereof |
US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
US20070027438A1 (en) | 2005-07-26 | 2007-02-01 | Frieder Loesel | System and method for compensating a corneal dissection |
TW200745163A (en) | 2006-02-17 | 2007-12-16 | Syntonix Pharmaceuticals Inc | Peptides that block the binding of IgG to FcRn |
EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
US20070292922A1 (en) | 2006-03-31 | 2007-12-20 | Cell Genesys, Inc. | Regulated expression of recombinant proteins from adeno-associated viral vectors |
EP2982695B1 (en) | 2008-07-09 | 2019-04-03 | Biogen MA Inc. | Compositions comprising antibodies to lingo or fragments thereof |
US8969353B2 (en) | 2008-11-07 | 2015-03-03 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
MX342858B (es) | 2010-03-29 | 2016-10-13 | The Trustees Of The Univ Of Pennsylvania * | Sistema de ablacion transgenica inducida farmacologicamente. |
JP6104897B2 (ja) | 2011-06-02 | 2017-04-05 | ダイアックス コーポレーション | Fcレセプター結合タンパク質 |
EP2717893B1 (en) | 2011-06-08 | 2019-05-08 | Translate Bio, Inc. | Lipid nanoparticle compositions and methods for mrna delivery |
KR20150020288A (ko) | 2012-06-08 | 2015-02-25 | 에트리스 게엠베하 | 메신저 rna의 폐전달 |
CA2901328C (en) | 2013-03-15 | 2023-08-01 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating mps1 |
WO2014205072A2 (en) | 2013-06-18 | 2014-12-24 | The Brigham And Women's Hospital, Inc. | FC RECEPTOR (FcRn) BINDING PEPTIDES AND USES THEREOF |
US20170000899A1 (en) | 2013-11-26 | 2017-01-05 | The Brigham And Women's Hospital, Inc. | Receptor-targeted nanoparticles for enhanced transcytosis mediated drug delivery |
US20180000043A1 (en) | 2015-02-04 | 2018-01-04 | Tony Gnann | Method for production of milk with a high content of native vitamin d |
PL3268391T3 (pl) * | 2015-03-09 | 2022-01-03 | Argenx Bvba | Sposoby zmniejszania poziomów środków zawierających Fc w surowicy z zastosowaniem antagonistów FcRn |
CA3006569A1 (en) | 2015-12-02 | 2017-06-08 | Voyager Therapeutics, Inc. | Assays for the detection of aav neutralizing antibodies |
WO2017160360A2 (en) | 2015-12-11 | 2017-09-21 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
WO2017106244A1 (en) | 2015-12-14 | 2017-06-22 | The Trustees Of The University Of Pennsylvania | Compositions and methods for regulatable antibody expression |
KR20200098604A (ko) * | 2017-12-13 | 2020-08-20 | 모멘타 파머슈티컬스 인코포레이티드 | FcRn 항체 및 이의 사용 방법 |
EP3768695A4 (en) | 2018-02-27 | 2022-04-06 | The Trustees of the University of Pennsylvania | NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF |
US20200407750A1 (en) | 2018-02-27 | 2020-12-31 | The Trustees Of The University Of Pennsylvania | Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor |
EP3908326A4 (en) | 2018-12-21 | 2022-10-26 | The Trustees of The University of Pennsylvania | COMPOSITIONS FOR DRG-SPECIFIC REDUCTION OF TRANSGENE EXPRESSION |
BR112021020054A2 (pt) | 2019-04-29 | 2021-12-07 | Univ Pennsylvania | Capsídeos de vírus adeno-associado (aav) e composições contendo os mesmos |
KR20220008280A (ko) | 2019-04-30 | 2022-01-20 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 폼페병의 치료에 유용한 조성물 |
-
2021
- 2021-06-16 MX MX2022016528A patent/MX2022016528A/es unknown
- 2021-06-16 IL IL299167A patent/IL299167A/en unknown
- 2021-06-16 EP EP21748696.8A patent/EP4171738A1/en active Pending
- 2021-06-16 CA CA3183153A patent/CA3183153A1/en active Pending
- 2021-06-16 AU AU2021292200A patent/AU2021292200A1/en active Pending
- 2021-06-16 CN CN202180050281.XA patent/CN115968302A/zh active Pending
- 2021-06-16 KR KR1020237001712A patent/KR20230025000A/ko active Search and Examination
- 2021-06-16 WO PCT/US2021/037575 patent/WO2021257668A1/en unknown
- 2021-06-16 JP JP2022578663A patent/JP2023531451A/ja active Pending
- 2021-06-16 US US18/002,060 patent/US20230220069A1/en active Pending
- 2021-06-16 TW TW110121993A patent/TW202214695A/zh unknown
Also Published As
Publication number | Publication date |
---|---|
KR20230025000A (ko) | 2023-02-21 |
IL299167A (en) | 2023-02-01 |
EP4171738A1 (en) | 2023-05-03 |
CN115968302A (zh) | 2023-04-14 |
AU2021292200A1 (en) | 2023-02-02 |
US20230220069A1 (en) | 2023-07-13 |
JP2023531451A (ja) | 2023-07-24 |
TW202214695A (zh) | 2022-04-16 |
CA3183153A1 (en) | 2021-12-23 |
WO2021257668A1 (en) | 2021-12-23 |
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