MX2024007903A - Terapia genica para deficiencias relacionadas con lamina a. - Google Patents

Terapia genica para deficiencias relacionadas con lamina a.

Info

Publication number
MX2024007903A
MX2024007903A MX2024007903A MX2024007903A MX2024007903A MX 2024007903 A MX2024007903 A MX 2024007903A MX 2024007903 A MX2024007903 A MX 2024007903A MX 2024007903 A MX2024007903 A MX 2024007903A MX 2024007903 A MX2024007903 A MX 2024007903A
Authority
MX
Mexico
Prior art keywords
aav
lamin
hlamina
raav
itr
Prior art date
Application number
MX2024007903A
Other languages
English (en)
Spanish (es)
Inventor
Christian Hinderer
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of MX2024007903A publication Critical patent/MX2024007903A/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
    • A61K48/0008—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
    • A61K48/0033—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 non-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
    • 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
    • A61K48/005—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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • 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
    • 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
    • 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
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric 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
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
MX2024007903A 2021-12-24 2022-12-24 Terapia genica para deficiencias relacionadas con lamina a. MX2024007903A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163293680P 2021-12-24 2021-12-24
PCT/US2022/082383 WO2023122803A1 (en) 2021-12-24 2022-12-24 Gene therapy for lamin a - associated deficiencies

Publications (1)

Publication Number Publication Date
MX2024007903A true MX2024007903A (es) 2024-07-10

Family

ID=86903820

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2024007903A MX2024007903A (es) 2021-12-24 2022-12-24 Terapia genica para deficiencias relacionadas con lamina a.

Country Status (11)

Country Link
US (1) US20250049949A1 (https=)
EP (1) EP4453226A4 (https=)
JP (1) JP2025501768A (https=)
KR (1) KR20240118821A (https=)
CN (1) CN118434877A (https=)
AU (1) AU2022419657A1 (https=)
CA (1) CA3241202A1 (https=)
CO (1) CO2024008188A2 (https=)
IL (1) IL313734A (https=)
MX (1) MX2024007903A (https=)
WO (1) WO2023122803A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
EP4634394A2 (en) 2022-12-17 2025-10-22 The Trustees of The University of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
TW202516019A (zh) 2023-06-29 2025-04-16 賓州大學委員會 具中樞神經系統靶向模體的突變aav及含有其之組成物
WO2025075551A1 (en) * 2023-10-06 2025-04-10 National University Of Singapore Method of treating lmna dilated cardiomyopathy
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 (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7291699B2 (en) * 2003-03-18 2007-11-06 The Regents Of The University Of Colorado Product and methods for diagnosis and therapy for cardiac and skeletal muscle disorders
WO2013071047A1 (en) * 2011-11-11 2013-05-16 Children's Medical Center Corporation Compositions and methods for in vitro transcription of rna
CN111886029B (zh) * 2018-01-19 2024-04-12 新加坡科技研究局 破坏linc复合物以治疗核纤层蛋白病
SG11202109362XA (en) * 2019-02-28 2021-09-29 Encoded Therapeutics Inc Compositions and methods for treating laminopathies
EP4198053A1 (en) * 2021-12-15 2023-06-21 Universitätsklinikum Hamburg-Eppendorf Lmna gene expression for treatment of laminopathies

Also Published As

Publication number Publication date
EP4453226A4 (en) 2026-03-18
US20250049949A1 (en) 2025-02-13
WO2023122803A1 (en) 2023-06-29
CN118434877A (zh) 2024-08-02
AU2022419657A1 (en) 2024-06-13
EP4453226A1 (en) 2024-10-30
KR20240118821A (ko) 2024-08-05
CA3241202A1 (en) 2023-06-29
CO2024008188A2 (es) 2024-08-08
IL313734A (en) 2024-08-01
JP2025501768A (ja) 2025-01-23

Similar Documents

Publication Publication Date Title
MX2024007903A (es) Terapia genica para deficiencias relacionadas con lamina a.
PH12021551341A1 (en) Compositions for drg-specific reduction of transgene expression
ES2555132T3 (es) Tratamiento de PCVD subclínica
ES2726777T3 (es) Vacuna porcina anti-PCV2 en dosis única
US7892809B2 (en) Chimeric vectors
MX2022009883A (es) Vectores de genoterapia para tratar enfermedades cardíacas.
MX2024000175A (es) Casetes de expresión optimizada para genoterapia.
WO2021163357A3 (en) Gene therapy vectors for treating heart disease
US9801936B2 (en) Method of attenuating porcine pseudorabies virus, attenuated strains of porcine pseudorabies virus, vaccine composition and use thereof
WO2019169004A8 (en) Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
EA202091352A1 (ru) Генотерапия мукополисахаридоза iiib
MY199445A (en) Adeno-associated virus variant capsids and methods of use thereof
PH12021552638A1 (en) Compositions useful for treatment of pompe disease
UA110632C2 (uk) Цирковірус свиней 2 типу (цвс2), імуногенна композиція, що містить його, набір для аналізу та їх застосування
RU2014121256A (ru) Векторы, кодирующие фактор жизнеспособности колбочек, полученный из палочек
US20230212609A1 (en) Codon-optimized nucleic acid encoding smn1 protein
MX2025003411A (es) Vectores de aav recombinantes para tratamiento de distrofia muscular
Rghei et al. Adeno-associated virus mediated expression of monoclonal antibody MR191 protects mice against Marburg virus and provides long-term expression in sheep
Mnyandu et al. Advances in designing Adeno-associated viral vectors for development of anti-HBV gene therapeutics
WO2023086928A3 (en) Gene therapy for treatment of mucopolysaccharidosis iiia
US12514916B2 (en) Porcine circovirus type 2 VLP vaccine
KR20190014263A (ko) 돼지 써코바이러스 2형(pcv2)의 orf2 재조합 유전자 및 이를 이용한 백신 조성물
EA202191744A1 (ru) Композиции для drg-специфического снижения экспрессии трансгена
WO2025010334A3 (en) Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept
Mnyandu et al. Advances in designing Adeno-associated viral vectors for development of anti-HBV gene