GB2634434A - ABCA4 trans-splicing molecules - Google Patents

ABCA4 trans-splicing molecules Download PDF

Info

Publication number
GB2634434A
GB2634434A GB2418240.4A GB202418240A GB2634434A GB 2634434 A GB2634434 A GB 2634434A GB 202418240 A GB202418240 A GB 202418240A GB 2634434 A GB2634434 A GB 2634434A
Authority
GB
United Kingdom
Prior art keywords
seq
nucleic acid
identity
acid trans
domain
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
GB2418240.4A
Other languages
English (en)
Other versions
GB202418240D0 (en
Inventor
Krumbach Rebekka
Dooley Scott
Doi Akiko
Burkhart Kirk
Gray Jesse
Peng Lingtao
Wu Dennis
Noma Akiko
Gosik Kirk
Slomovic Shimyn
Clemens Adam
Bell Robert
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ascidian Therapeutics Inc
Original Assignee
Ascidian Therapeutics Inc
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 Ascidian Therapeutics Inc filed Critical Ascidian Therapeutics Inc
Publication of GB202418240D0 publication Critical patent/GB202418240D0/en
Publication of GB2634434A publication Critical patent/GB2634434A/en
Pending legal-status Critical Current

Links

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
    • 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
    • A61K48/0066—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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C12N15/67—General methods for enhancing the expression
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect
    • 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
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • C12N2840/445—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
GB2418240.4A 2022-05-13 2023-05-12 ABCA4 trans-splicing molecules Pending GB2634434A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263341665P 2022-05-13 2022-05-13
US202363478472P 2023-01-04 2023-01-04
PCT/US2023/066969 WO2023220742A2 (en) 2022-05-13 2023-05-12 Abca4 trans-splicing molecules

Publications (2)

Publication Number Publication Date
GB202418240D0 GB202418240D0 (en) 2025-01-29
GB2634434A true GB2634434A (en) 2025-04-09

Family

ID=88731152

Family Applications (1)

Application Number Title Priority Date Filing Date
GB2418240.4A Pending GB2634434A (en) 2022-05-13 2023-05-12 ABCA4 trans-splicing molecules

Country Status (11)

Country Link
US (3) US20240042057A1 (enExample)
EP (1) EP4522758A4 (enExample)
JP (1) JP2025521102A (enExample)
KR (1) KR20250011939A (enExample)
CN (1) CN119731328A (enExample)
AU (1) AU2023269865A1 (enExample)
CA (1) CA3252043A1 (enExample)
GB (1) GB2634434A (enExample)
IL (1) IL316944A (enExample)
MX (1) MX2024014029A (enExample)
WO (1) WO2023220742A2 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2020010959A (es) 2018-04-17 2021-01-15 Univ Pennsylvania Moleculas de empalme trans.
IL316944A (en) 2022-05-13 2025-01-01 Ascidian Therapeutics Inc ABCA4 cross-linking molecules
WO2025217163A2 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Novel aav capsids binding to human cd59
WO2025217174A1 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with carbonic anhydrase iv

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016005524A1 (en) * 2014-07-09 2016-01-14 Lexogen Gmbh Methods and products for quantifying rna transcript variants
WO2019204514A1 (en) * 2018-04-17 2019-10-24 Limelight Bio, Inc. Trans-splicing molecules
WO2020214990A1 (en) * 2019-04-17 2020-10-22 Ascidian Therapeutics, Inc. Abca4 cell line and methods of use
WO2020223180A1 (en) * 2019-05-01 2020-11-05 The Procter & Gamble Company Probiotic bacterial strains that produce short chain fatty acids and compositions comprising same

Family Cites Families (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE69636937T3 (de) 1995-12-15 2011-01-05 Virxsys Corp. Durch trans-spaltung erhaltene therapeutische molekule
US6280978B1 (en) 1995-12-15 2001-08-28 Intronn Holdings, Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
ES2401121T3 (es) 1996-10-04 2013-04-17 Veri-Q Inc. Dispositivos de recolección de muestras y métodos que utilizan marcadores y el uso de dichos marcadores como controles en la validación de una muestra, evaluación y/o certificación de laboratorio
US6929907B2 (en) 1999-12-31 2005-08-16 North Carolina State University Methods and compositions for determining the purity of chemically synthesized nucleic acids
JP4500540B2 (ja) 2001-06-29 2010-07-14 ヴェリ‐キュー，インコーポレイテッド 化学的に合成された核酸の純度を決定し、かつ化学的に合成された核酸を精製するための方法および組成物
JP2006505242A (ja) 2002-02-12 2006-02-16 イントロン，インコーポレーテッド スプライセオソームにより媒介されるｒｎａトランス−スプライシングにおいて使用するための方法および組成物
AU2003213270A1 (en) 2002-02-25 2003-09-09 Intronn, Inc. Trans-splicing mediated imaging of gene expression
US7399753B2 (en) 2002-02-25 2008-07-15 Virxsys Corporation Trans-splicing mediated photodynamic therapy
EP1549755A4 (en) 2002-06-05 2006-09-20 Intronn Inc SPREAD OSOM-MEDIATED TRANS-RNA SPLEISSEN IN STEM CELLS
JP2005528911A (ja) 2002-06-05 2005-09-29 イントロン，インコーポレーテッド スプライセオソーム媒介型ｒｎａトランススプライシング、及びスプライセオソーム媒介型ｒｎａトランススプライシングを利用した第ｖｉｉｉ因子の遺伝的欠陥の修正
US20040018622A1 (en) 2002-07-17 2004-01-29 Mitchell Lloyd G. Spliceosome mediated RNA trans-splicing for correction of skin disorders
EP1579004B1 (en) 2002-10-23 2010-06-16 VIRxSYS Corporation Screening methods for identification of efficient pre-trans-splicing molecules
AU2005207053A1 (en) 2004-01-23 2005-08-04 Virxsys Corporation Expression of ApoA-1 and variants thereof using spliceosome mediated RNA trans-splicing
WO2005070948A1 (en) 2004-01-23 2005-08-04 Intronn, Inc. Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated rna trans splicing
US7968334B2 (en) 2004-01-23 2011-06-28 Virxsys Corporation Expression of apoAI and variants thereof using spliceosome mediated RNA trans-splicing
WO2006026611A2 (en) 2004-08-31 2006-03-09 Mitchell Lloyd G Reverse transcriptase mediated rna gene expression
US7943374B2 (en) 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
US20090214478A1 (en) 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
US8236557B2 (en) 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
EP2151248A1 (en) 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
EP2486136B1 (en) 2009-10-08 2016-03-30 Association Institut de Myologie Nucleic acid molecules and methods for exchanging exon(s) by transsplicing
US8648053B2 (en) 2010-10-20 2014-02-11 Rosalind Franklin University Of Medicine And Science Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for Usher syndrome
WO2012158757A1 (en) 2011-05-16 2012-11-22 The Trustees Of The University Of Pennsylvania Proviral plasmids for production of recombinant adeno-associated virus
EA033653B1 (ru) 2011-09-05 2019-11-13 Stichting Katholieke Univ Антисмысловые олигонуклеотиды для лечения врожденного амавроза лебера
EP2872183B1 (en) 2012-07-11 2018-09-26 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for rpgr x-linked retinal degeneration
CN104955951A (zh) 2012-10-16 2015-09-30 麻省理工学院 稳定的非多腺苷酸化rna的产生
EP2986635B1 (en) 2013-04-18 2018-10-03 Fondazione Telethon Effective delivery of large genes by dual aav vectors
CA2948728A1 (en) 2014-03-10 2015-09-17 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
EP3201339A4 (en) 2014-10-03 2018-09-19 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
AU2016355343C1 (en) * 2015-11-19 2024-10-03 Lloyd G. Mitchell Compositions and methods for correction of heritable ocular disease
WO2017106370A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of eye diseases
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
EP3436590A4 (en) 2016-04-01 2019-12-04 National University of Singapore TRANSSPLEISSEN OF RNA (TSRNA)
WO2019027869A1 (en) 2017-07-31 2019-02-07 Massachusetts Institute Of Technology RNA-CLUSTER INDUCED TRANSCRIPT STABILIZER AND USES THEREOF
SI3673080T1 (sl) 2017-08-25 2024-03-29 Stoke Therapeutics, Inc. Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni
EP3765023A4 (en) * 2018-03-15 2022-01-19 Intergalactic Therapeutics, Inc. SYNTHETIC DNA VECTORS AND METHODS OF USE
CN114040974B (zh) * 2019-04-17 2025-11-04 宾夕法尼亚州大学信托人 用于高效rna反式剪接的三螺旋终止子
AU2020333667A1 (en) 2019-08-16 2022-02-24 Massachusetts Institute Of Technology Targeted trans-splicing using CRISPR/Cas13
EP4045665A4 (en) 2019-10-15 2023-11-15 University of Massachusetts RNA EDITOR ENHANCED RNA TRANS-SPLICING
AU2021225696B2 (en) 2020-02-28 2024-11-14 Rznomics Inc. Trans-splicing ribozyme specific to APOE4 RNA and use thereof
EP4217010A4 (en) 2020-09-28 2025-01-08 Tacit Therapeutics, Inc. TRANS-SPLICING SYSTEM FOR TISSUE-SPECIFIC REPLACEMENT OF RNA SEQUENCES
AU2022259416A1 (en) 2021-04-15 2023-11-16 Tacit Therapeutics, Inc. High efficiency trans-splicing for replacement of targeted rna sequences in human cells
IL311101A (en) 2021-09-03 2024-04-01 Tacit Therapeutics Inc RNA editing through recruitment of SPLICEOSOME components
WO2023205694A2 (en) 2022-04-20 2023-10-26 Tacit Therapeutics, Inc. Stabilization of therapeutic trans-splicing rna molecules in human cells
US20250290067A1 (en) 2022-05-03 2025-09-18 Tacit Therapeutics, Inc. Localization of trans-splicing nucleic acid molecules to and within the cellular nucleus
IL316944A (en) 2022-05-13 2025-01-01 Ascidian Therapeutics Inc ABCA4 cross-linking molecules

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016005524A1 (en) * 2014-07-09 2016-01-14 Lexogen Gmbh Methods and products for quantifying rna transcript variants
WO2019204514A1 (en) * 2018-04-17 2019-10-24 Limelight Bio, Inc. Trans-splicing molecules
WO2020214990A1 (en) * 2019-04-17 2020-10-22 Ascidian Therapeutics, Inc. Abca4 cell line and methods of use
WO2020223180A1 (en) * 2019-05-01 2020-11-05 The Procter & Gamble Company Probiotic bacterial strains that produce short chain fatty acids and compositions comprising same

Also Published As

Publication number Publication date
AU2023269865A1 (en) 2024-11-21
MX2024014029A (es) 2025-02-10
EP4522758A4 (en) 2026-05-06
US20240042057A1 (en) 2024-02-08
US20240091381A1 (en) 2024-03-21
GB202418240D0 (en) 2025-01-29
WO2023220742A2 (en) 2023-11-16
WO2023220742A3 (en) 2024-03-28
KR20250011939A (ko) 2025-01-22
IL316944A (en) 2025-01-01
US12410440B2 (en) 2025-09-09
CA3252043A1 (en) 2023-11-16
EP4522758A2 (en) 2025-03-19
JP2025521102A (ja) 2025-07-08
CN119731328A (zh) 2025-03-28
US20260049332A1 (en) 2026-02-19

Similar Documents

Publication Publication Date Title
US12104175B2 (en) High activity regulatory elements
EP3851537B1 (en) Treatment of hyperbilirubinemia
JP2020537544A (ja) ライソゾーム病の遺伝子治療
CA2386546A1 (en) Adeno-associated viruses and uses thereof
AU2016355343A1 (en) Compositions and methods for correction of heritable ocular disease
CN113710693B (zh) Dna结合结构域反式激活因子及其用途
CA3097004A1 (en) Trans-splicing molecules
JP2025521102A5 (enExample)
EP3628011A1 (en) Gene therapy for ciliopathies
EP4225782A1 (en) Nucleic acid constructs, viral vectors and viral particles
TW202221119A (zh) Dna結合域轉活化子及其用途
JP7609436B2 (ja) 顕性型変異遺伝子に由来する疾患の治療剤
KR20250061713A (ko) 유전자의 망막 세포 내 발현을 위한 프로모터 및 이를 포함하는 벡터 시스템
KR20230112672A (ko) 신경변성 질환을 위한 유전자 요법
WO2021138286A1 (en) Self-complementary aav delivery system for crispr/cas9
CN119823985A (zh) 视网膜神经节细胞特异性启动子以及其用途
AU784420B2 (en) Adeno-associated viruses and uses thereof
WO2022187671A1 (en) Compositions and methods for the treatment of hemophilia
US20260098279A1 (en) Reconstitution of large genes via cre-lox dna recombination in adeno-associated virus vectors
US20230078498A1 (en) Targeted Translation of RNA with CRISPR-Cas13 to Enhance Protein Synthesis
WO2025017168A1 (en) Novel optimized utrophin micro-genes
WO2026032020A1 (zh) Aav介导的rpgr x连锁视网膜变性的基因编辑治疗方法
WO2025019820A1 (en) All in one vectors for the treatment of facioscapulohumeral muscular dystrophy
EP4594505A1 (en) Therapy by trans-splicing of opa1 pre-messenger rnas for the treatment of diseases associated with opa1 gene mutations
WO2023156530A1 (en) Gene therapy for neurodegenerative diseases