WO2022221320A1 - Gene therapy for arrhythmogenic right ventricular cardiomyopathy - Google Patents

Gene therapy for arrhythmogenic right ventricular cardiomyopathy Download PDF

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Publication number
WO2022221320A1
WO2022221320A1 PCT/US2022/024478 US2022024478W WO2022221320A1 WO 2022221320 A1 WO2022221320 A1 WO 2022221320A1 US 2022024478 W US2022024478 W US 2022024478W WO 2022221320 A1 WO2022221320 A1 WO 2022221320A1
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WO
WIPO (PCT)
Prior art keywords
promoter
seq
nucleic acid
pkp2
aspects
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.)
Ceased
Application number
PCT/US2022/024478
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English (en)
French (fr)
Inventor
Farah Sheikh
William Bradford
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.)
University of California Berkeley
University of California San Diego UCSD
Original Assignee
University of California Berkeley
University of California San Diego UCSD
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
Priority to KR1020237035949A priority Critical patent/KR20230170686A/ko
Priority to CA3213231A priority patent/CA3213231A1/en
Priority to MX2023011875A priority patent/MX2023011875A/es
Priority to US18/554,771 priority patent/US20240216541A1/en
Priority to IL307506A priority patent/IL307506A/en
Priority to CN202280029149.5A priority patent/CN117178057A/zh
Priority to JP2023561167A priority patent/JP2024514110A/ja
Priority to BR112023021037A priority patent/BR112023021037A2/pt
Application filed by University of California Berkeley, University of California San Diego UCSD filed Critical University of California Berkeley
Priority to AU2022258329A priority patent/AU2022258329A1/en
Priority to EP22788797.3A priority patent/EP4323534A4/en
Publication of WO2022221320A1 publication Critical patent/WO2022221320A1/en
Anticipated expiration legal-status Critical
Priority to CONC2023/0014900A priority patent/CO2023014900A2/es
Ceased legal-status Critical Current

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    • 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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • 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
    • 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
    • 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
    • A61K48/0083—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 administration regime
    • 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
    • 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
    • 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
    • A61P9/06—Antiarrhythmics
    • 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
    • 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
    • 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
    • C12N2830/48—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

Definitions

  • an rAAV vector can comprise a first AAV ITR sequence, a promoter sequence, a transgene nucleic acid molecule, a post-transcriptional regulatory element, a polyA sequence, and a second AAV ITR sequence.
  • an rAAV vector can comprise, in the 5’ to 3’ direction, a first AAV ITR sequence, a promoter sequence, a transgene nucleic acid molecule, a post-transcriptional regulatory element, a polyA sequence, and a second AAV ITR sequence.
  • an rAAV vector can comprise more than one promoter sequence.
  • an rAAV vector can comprise at least two promoter sequences, such that the rAAV vector comprises a first promoter sequence and an at least second promoter sequence.
  • the first and the at least second promoter sequences can comprise the same sequence.
  • the first and the at least second promoter sequences can comprise different sequences.
  • the first and the at least second promoter sequences can be adjacent to each other.
  • bacterial plasmids of the present disclosure can comprise a prokaryotic promoter.
  • a PKP2 polypeptide comprises, consists essentially of, or consists of an amino acid sequence at least 65%, 70%, 75%, 80%, 85%, 90%, 95%, 96%, 97%, 98%, 99% or 100% (or any percentage in between) identical to the amino acid sequence put forth in SEQ ID NO: 13, or a fragment thereof.
  • a PKP2 polypeptide comprises, consists essentially of, or consists of an amino acid sequence at least 65%, 70%, 75%, 80%, 85%, 90%, 95%, 96%, 97%, 98%, 99% or 100% (or any percentage in between) identical to at least one portion of the amino acid sequence put forth in SEQ ID NO: 13, or a fragment thereof.
  • MeCP2 polyA sequence a retinol dehydrogenase 1 (RDHl) polyA sequence, a bovine growth hormone (BGH) polyA sequence, an SV40 polyA sequence, a SPA49 polyA sequence, a sNRP-TK65 polyA sequence, a sNRP polyA sequence, or a TK65 polyA sequence.
  • RHl retinol dehydrogenase 1
  • BGH bovine growth hormone
  • a "viral vector” is defined as a recombinantly produced virus or viral particle that contains a polynucleotide to be delivered into a host cell, either in vivo, ex vivo or in vitro.
  • viral vectors include retroviral vectors, AAV vectors, lentiviral vectors, adenovirus vectors, alphavirus vectors and the like.
  • Alphavirus vectors such as Semliki Forest virus-based vectors and Sindbis virus-based vectors, have also been developed for use in gene therapy and immunotherapy. See, e.g., Schlesinger and Dubensky (1999) Curr. Opin. Biotechnol. 5:434-439 and Ying, et al. (1999) Nat. Med. 5(7):823-827.
  • An AAV capsid protein can be any AAV capsid protein known in the art.
  • compositions and Pharmaceutical Compositions
  • the term “pharmaceutically acceptable” means approved by a regulatory agency of the Federal or a state government or listed in the U. S. Pharmacopoeia, other generally recognized pharmacopoeia in addition to other formulations that are safe for use in animals, and more particularly in humans and/or non-human mammals.
  • a pharmaceutical composition can comprise tris, wherein the tris is present at a concentration of about 10 mM to about 100 mM, or about 10 mM to about 50 mM, or about 15 mM to about 25 mM. In some aspects, the tris can be present at a concentration of about 20 mM.
  • administer intends to mean delivery of a substance to a subject such as an animal or human. Administration can be effected in one dose, continuously or intermittently throughout the course of treatment. Methods of determining the most effective means and dosage of administration are known to those of skill in the art and will vary with the composition used for therapy, the purpose of the therapy, as well as the age, health or gender of the subject being treated. Single or multiple administrations can be carried out with the dose level and pahem being selected by the treating physician or in the case of pets and other animals, treating veterinarian.
  • the amounts of viral particles in a composition, pharmaceutical composition, or the amount of viral particles administered to a patient can calculated based on the percentage of viral particles that are predicted to contain viral genomes.
  • kits of the present disclosure include any one of the isolated polynucleotides, rAAV vectors, rAAV viral vectors, compositions, pharmaceutical compositions, host cells, isolated tissues, as described herein.
  • Values or ranges may be also be expressed herein as “about,” from “about” one particular value, and/or to “about” another particular value. When such values or ranges are expressed, other embodiments disclosed include the specific value recited, from the one particular value, and/or to the other particular value. Similarly, when values are expressed as approximations, by use of the antecedent “about,” it will be understood that the particular value forms another embodiment. It will be further understood that there are a number of values disclosed therein, and that each value is also herein disclosed as “about” that particular value in addition to the value itself. In embodiments, “about” can be used to mean, for example, within 10% of the recited value, within 5% of the recited value, or within 2% of the recited value.
  • nucleic acids containing one or more carbocyclic sugars are also included within the definition of nucleic acids (see Jenkins et al. (1995) Chem. Soc. Rev. pp 169-176, which is incorporated by reference).
  • nucleic acid analogs are also described in, e.g., Rawls, C & E News Jun. 2, 1997 page 35, which is incorporated by reference. These modifications of the ribose- phosphate backbone may be done to facilitate the addition of additional moieties such as labels, or to alter the stability and half-life of such molecules in physiological environments.
  • modified bases and nucleotides are also described in, e.g., U.S.
  • expression refers to the two-step process by which polynucleotides are transcribed into mRNA and/or the process by which the transcribed mRNA is subsequently translated into peptides, polypeptides, or proteins. If the polynucleotide is derived from genomic DNA, expression may include splicing of the mRNA in a eukaryotic cell.
  • FIG. 3 At 4 weeks post-injection heart lysates were analyzed via western blot and found that AAV9 PKP2 administration could improve levels of cell-cell junction proteins (PKP2, DSP, DSG2, JUP, CX43) (FIG. 3B). Early AAV9 PKP2 injection could also prevent ARVC disease development at 4 weeks of age (FIG. 3C), as there was preservation of cardiac mechanical and electrical function, significantly less fibrosis in myocardium, and prolonged survival (Fig. 4; Fig. 5; Fig. 6A). Cell-cell junction protein levels, cardiac mechanical function, and cardiac electrical function were still preserved 6 months post-AAV9 PKP2 injection (Fig.

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  • General Health & Medical Sciences (AREA)
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  • Microbiology (AREA)
  • Cardiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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PCT/US2022/024478 2021-04-12 2022-04-12 Gene therapy for arrhythmogenic right ventricular cardiomyopathy Ceased WO2022221320A1 (en)

Priority Applications (11)

Application Number Priority Date Filing Date Title
JP2023561167A JP2024514110A (ja) 2021-04-12 2022-04-12 不整脈原性右心室心筋症に対する遺伝子療法
MX2023011875A MX2023011875A (es) 2021-04-12 2022-04-12 Tratamiento genico para miocardiopatía arritmogénica del ventriculo derecho.
US18/554,771 US20240216541A1 (en) 2021-04-12 2022-04-12 Gene therapy for arrhythmogenic right ventricular cardiomyopathy
IL307506A IL307506A (en) 2021-04-12 2022-04-12 Gene therapy for arrhythmogenic right ventricular cardiomyopathy
CN202280029149.5A CN117178057A (zh) 2021-04-12 2022-04-12 用于致心律失常性右心室心肌病的基因治疗
KR1020237035949A KR20230170686A (ko) 2021-04-12 2022-04-12 부정맥유발 우심실 심근병증에 대한 유전자 치료법
EP22788797.3A EP4323534A4 (en) 2021-04-12 2022-04-12 Gene therapy for arrhythmogenic right ventricular cardiomyopathy
BR112023021037A BR112023021037A2 (pt) 2021-04-12 2022-04-12 Terapia gênica para cardiomiopatia ventricular direita arritmogênica
AU2022258329A AU2022258329A1 (en) 2021-04-12 2022-04-12 Gene therapy for arrhythmogenic right ventricular cardiomyopathy
CA3213231A CA3213231A1 (en) 2021-04-12 2022-04-12 Gene therapy for arrhythmogenic right ventricular cardiomyopathy
CONC2023/0014900A CO2023014900A2 (es) 2021-04-12 2023-10-31 Tratamiento génico para miocardiopatía arritmogénica del ventrículo derecho

Applications Claiming Priority (2)

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US202163173527P 2021-04-12 2021-04-12
US63/173,527 2021-04-12

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WO2022221320A1 true WO2022221320A1 (en) 2022-10-20

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US (1) US20240216541A1 (https=)
EP (1) EP4323534A4 (https=)
JP (1) JP2024514110A (https=)
KR (1) KR20230170686A (https=)
CN (1) CN117178057A (https=)
AU (1) AU2022258329A1 (https=)
BR (1) BR112023021037A2 (https=)
CA (1) CA3213231A1 (https=)
CO (1) CO2023014900A2 (https=)
IL (1) IL307506A (https=)
MX (1) MX2023011875A (https=)
WO (1) WO2022221320A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11883506B2 (en) 2020-08-07 2024-01-30 Spacecraft Seven, Llc Plakophilin-2 (PKP2) gene therapy using AAV vector

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070072175A1 (en) * 2005-05-13 2007-03-29 Biogen Idec Ma Inc. Nucleotide array containing polynucleotide probes complementary to, or fragments of, cynomolgus monkey genes and the use thereof
US20070083334A1 (en) * 2001-09-14 2007-04-12 Compugen Ltd. Methods and systems for annotating biomolecular sequences
US20110296544A1 (en) * 2004-04-30 2011-12-01 Celera Corporation Colon cancer targets and uses thereof
US20140010861A1 (en) * 2012-04-02 2014-01-09 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
US20190203212A1 (en) * 2012-07-25 2019-07-04 Massachusetts Institute Of Technology Inducible dna binding proteins and genome perturbation tools and applications thereof
WO2021053222A1 (en) * 2019-09-20 2021-03-25 Ucl Business Ltd Gene therapy composition and treatment of right ventricular arrythmogenic cardiomyopathy

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230043869A (ko) * 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070083334A1 (en) * 2001-09-14 2007-04-12 Compugen Ltd. Methods and systems for annotating biomolecular sequences
US20110296544A1 (en) * 2004-04-30 2011-12-01 Celera Corporation Colon cancer targets and uses thereof
US20070072175A1 (en) * 2005-05-13 2007-03-29 Biogen Idec Ma Inc. Nucleotide array containing polynucleotide probes complementary to, or fragments of, cynomolgus monkey genes and the use thereof
US20140010861A1 (en) * 2012-04-02 2014-01-09 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
US20140206755A1 (en) * 2012-04-02 2014-07-24 Moderna Therapeutics, Inc. Modified polynucleotides for treating arylsulfatase a protein deficiency
US20190203212A1 (en) * 2012-07-25 2019-07-04 Massachusetts Institute Of Technology Inducible dna binding proteins and genome perturbation tools and applications thereof
WO2021053222A1 (en) * 2019-09-20 2021-03-25 Ucl Business Ltd Gene therapy composition and treatment of right ventricular arrythmogenic cardiomyopathy

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
See also references of EP4323534A4 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11883506B2 (en) 2020-08-07 2024-01-30 Spacecraft Seven, Llc Plakophilin-2 (PKP2) gene therapy using AAV vector

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MX2023011875A (es) 2024-01-05
US20240216541A1 (en) 2024-07-04
IL307506A (en) 2023-12-01
KR20230170686A (ko) 2023-12-19
JP2024514110A (ja) 2024-03-28
EP4323534A1 (en) 2024-02-21
EP4323534A4 (en) 2025-06-18
AU2022258329A1 (en) 2023-10-12
CA3213231A1 (en) 2022-10-20
CO2023014900A2 (es) 2023-11-20
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CN117178057A (zh) 2023-12-05
BR112023021037A2 (pt) 2023-12-19

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