MX2023011035A - Composición de terapia génica y tratamiento de la miocardiopatía arritmogénica del ventriculo derecho. - Google Patents

Composición de terapia génica y tratamiento de la miocardiopatía arritmogénica del ventriculo derecho.

Info

Publication number
MX2023011035A
MX2023011035A MX2023011035A MX2023011035A MX2023011035A MX 2023011035 A MX2023011035 A MX 2023011035A MX 2023011035 A MX2023011035 A MX 2023011035A MX 2023011035 A MX2023011035 A MX 2023011035A MX 2023011035 A MX2023011035 A MX 2023011035A
Authority
MX
Mexico
Prior art keywords
gene therapy
treatment
right ventricular
therapy composition
arrhythmogenic cardiomyopathy
Prior art date
Application number
MX2023011035A
Other languages
English (en)
Inventor
Thomas Voit
Julie Dumonceaux
Perry Elliott
Virginie Mariot
Original Assignee
Ucl Business Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ucl Business Ltd filed Critical Ucl Business Ltd
Publication of MX2023011035A publication Critical patent/MX2023011035A/es

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
    • 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
    • A61K48/0008—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
    • A61K48/0041—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 the non-active part being polymeric
    • 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
    • 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
    • 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
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • 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
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • 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
    • 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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

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)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Cardiology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Se describen una composición y método para tratar o prevenir la miocardiopatía en un sujeto humano. En algunas modalidades, el método comprende administrar una dosis terapéutica de un vector de terapia génica a los cardiomiocitos del sujeto humano, en donde el vector de terapia génica comprende una secuencia de ácidos nucleicos que codifica para PKP2.
MX2023011035A 2021-03-19 2022-03-18 Composición de terapia génica y tratamiento de la miocardiopatía arritmogénica del ventriculo derecho. MX2023011035A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163163393P 2021-03-19 2021-03-19
PCT/EP2022/057145 WO2022195074A2 (en) 2021-03-19 2022-03-18 Gene therapy composition and treatment of right ventricular arrhythmogenic cardiomyopathy

Publications (1)

Publication Number Publication Date
MX2023011035A true MX2023011035A (es) 2024-02-09

Family

ID=81384690

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023011035A MX2023011035A (es) 2021-03-19 2022-03-18 Composición de terapia génica y tratamiento de la miocardiopatía arritmogénica del ventriculo derecho.

Country Status (12)

Country Link
US (1) US20240042059A1 (es)
EP (1) EP4308172A2 (es)
JP (1) JP2024512483A (es)
KR (1) KR20230159471A (es)
CN (1) CN117715660A (es)
AU (1) AU2022239846A1 (es)
BR (1) BR112023018944A2 (es)
CA (1) CA3212043A1 (es)
IL (1) IL305959A (es)
MX (1) MX2023011035A (es)
TW (1) TW202300180A (es)
WO (1) WO2022195074A2 (es)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220154217A1 (en) 2019-04-01 2022-05-19 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
EP4192487A4 (en) 2020-08-07 2024-10-02 Spacecraft Seven Llc GENE THERAPY FOR PLAKOPHILIN-2 (PKP2) INVOLVING AAV VECTOR
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
IL301675A (en) * 2020-10-09 2023-05-01 Tenaya Therapeutics Inc Methods and preparations for placophilin-2 gene therapy
CN115851751A (zh) * 2022-12-21 2023-03-28 百世诺（北京）医疗科技有限公司 肥厚型心肌病变异基因tnnt2及其应用

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111107880A (zh) * 2017-09-20 2020-05-05 加利福尼亚大学董事会 用于恢复致心律失常性右室心肌病中的电性和心脏功能以及心脏结构的基因疗法策略
US12098385B2 (en) * 2017-09-20 2024-09-24 The Regents Of The University Of California Gene therapy strategy to restore cardiac electrical and structural function in arrhythmogenic right ventricular cardiomyopathy
CN109004092A (zh) 2018-06-29 2018-12-14 云谷（固安）科技有限公司 有机电致发光器件和有机电致发光装置
TW202126674A (zh) * 2019-09-20 2021-07-16 英商Ｕｃｌ商業有限責任公司 右心室心律失常性心肌病之基因療法組合物及治療
EP4192487A4 (en) * 2020-08-07 2024-10-02 Spacecraft Seven Llc GENE THERAPY FOR PLAKOPHILIN-2 (PKP2) INVOLVING AAV VECTOR
US11781156B2 (en) * 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
IL301675A (en) 2020-10-09 2023-05-01 Tenaya Therapeutics Inc Methods and preparations for placophilin-2 gene therapy

Also Published As

Publication number Publication date
KR20230159471A (ko) 2023-11-21
AU2022239846A9 (en) 2023-11-09
IL305959A (en) 2023-11-01
BR112023018944A2 (pt) 2023-12-26
JP2024512483A (ja) 2024-03-19
AU2022239846A1 (en) 2023-10-26
WO2022195074A2 (en) 2022-09-22
CA3212043A1 (en) 2022-09-22
CN117715660A (zh) 2024-03-15
US20240042059A1 (en) 2024-02-08
WO2022195074A3 (en) 2022-11-10
EP4308172A2 (en) 2024-01-24
TW202300180A (zh) 2023-01-01

Similar Documents

Publication Publication Date Title
MX2023011035A (es) Composición de terapia génica y tratamiento de la miocardiopatía arritmogénica del ventriculo derecho.
MX2024000120A (es) Uso de un vector viral aadc para el tratamiento de la deficiencia de la aadc.
AU2018372763A1 (en) Materials and methods for treatment of autosomal dominant Retinitis Pigmentosa
MX2019011215A (es) Ácidos nucleicos que codifican proteínas asociadas a crispr y usos de los mismos.
MX2023001741A (es) Inhibidores de cetonas no hidratadas de nav1.7 para el tratamiento de dolor.
MX336710B (es) Oligoribonucleotidos y metodos de uso de los mismos para tratamiento de la alopecia, insuficiencia renal aguda y otras enfermedades.
GB0223380D0 (en) Combination therapy
MX2020010994A (es) Terapia de gen para enfermedades causadas por combinaciones desequilibradas de nucleótidos que incluyen síndromes de agotamiento de adn mitocondrial.
PH12020500239A1 (en) Factor viii (fviii) gene therapy methods
EP1455840A4 (en) TREATMENT OF HUMAN CANCER WITH ADENOVIRAL VECTOR TNF-ALPHA
MX2023003984A (es) Metodos y composiciones para terapia genica con placofilina-2.
EP4289475A3 (en) Methods of treating fabry disease in patients having a mutation in the gla gene
WO2006014159A3 (en) Methods of using regenerative cells in the treatment of musculoskeletal disorders
MX2018010223A (es) Terapia combinada para tratamiento de cancer de ovario.
ZA202304965B (en) Combination therapy for treating cancer
MX2023003993A (es) Tratamiento de trastornos bipolares y psicosis con clorhidrato de dexmedetomidina.
MX2021002349A (es) Inhibidores de calicreína en plasmá y usos de los mismos para tratar ataque de angioedema hereditario.
MX2023007609A (es) Métodos para mejorar la terapia génica no viral.
WO2002083080A3 (en) Method of treating arthritis using lentiviral vectors in gene therapy
MX2021009696A (es) Vectores de genoterapia para el tratamiento de la enfermedad de danon.
WO2022197720A3 (en) Compositions and methods for treating coronavirus infection
WO2022076556A3 (en) Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21
MX2015007402A (es) Fusion de hormona de crecimiento humano y albumina, formulacion y usos de los mismos.
MX2022005670A (es) Composiciones y metodos para recombinacion de alta eficiencia de moleculas de arn.
WO2020079489A3 (en) Treatment of neuropathy with igf-1-encoding dna constructs and hgf-encoding dna constructs