SG10201908848RA - Pharmacologically induced transgene ablation system - Google Patents

Pharmacologically induced transgene ablation system

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Publication number
SG10201908848RA
SG10201908848RA SG10201908848RA SG10201908848RA SG 10201908848R A SG10201908848R A SG 10201908848RA SG 10201908848R A SG10201908848R A SG 10201908848RA SG 10201908848R A SG10201908848R A SG 10201908848RA
Authority
SG
Singapore
Prior art keywords
transgene
pharmacologically induced
ablating
replication
subject
Prior art date
Application number
Inventor
James M Wilson
Shu-Jen Chen
Anna P Tretiakova
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.)
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Publication date
Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of SG10201908848RA publication Critical patent/SG10201908848RA/en

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    • 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
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    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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    • 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
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    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
    • C07K2319/715—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16 containing a domain for ligand dependent transcriptional activation, e.g. containing a steroid receptor domain
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    • C07K—PEPTIDES
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    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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    • 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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
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    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • C12N2830/003—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor tet inducible
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    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/005—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination repressible enhancer/promoter combination, e.g. KRAB
    • C12N2830/006—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination repressible enhancer/promoter combination, e.g. KRAB tet repressible
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
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    • 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 RNAses, DNAses

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Abstract

OF THE INVENTION PHARMACOLOGICALLY INDUCED TRANSGENE ABLATION SYSTEM The present invention relates to gene therapy systems designed for the delivery of a therapeutic product to a subject using replication-defective virus composition(s) engineered with a built-in safety mechanism for ablating the therapeutic gene product, 10 either permanently or temporarily, in response to a pharmacological agent - preferably an oral formulation, e.g., a pill. The invention is based, in part, on the applicants' development of an integrated approach, referred to herein as "PITA" (Pharmacologically Induced Transgene Ablation), for ablating a transgene or negatively regulating transgene expression. In this approach, replication-deficient viruses are used to deliver a transgene encoding a 15 therapeutic product (an RNA or a protein) so that it is expressed in the subject, but can be reversibly or irreversibly turned offby administering the pharmacological agent; e.g., by administration of a small molecule that induces expression of an ablator specific for the transgene or its RNA transcript. 20 Figure for publication: None 126
SG10201908848R 2010-03-29 2011-03-28 Pharmacologically induced transgene ablation system SG10201908848RA (en)

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US31875210P 2010-03-29 2010-03-29

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SG10201908848R SG10201908848RA (en) 2010-03-29 2011-03-28 Pharmacologically induced transgene ablation system
SG10201502270TA SG10201502270TA (en) 2010-03-29 2011-03-28 Pharmacologically induced transgene ablation system
SG2012065868A SG183929A1 (en) 2010-03-29 2011-03-28 Pharmacologically induced transgene ablation system

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SG2012065868A SG183929A1 (en) 2010-03-29 2011-03-28 Pharmacologically induced transgene ablation system

Country Status (11)

Country Link
US (1) US20130023033A1 (en)
EP (1) EP2553106A2 (en)
JP (1) JP5922095B2 (en)
KR (1) KR20130040844A (en)
CN (1) CN102869779A (en)
AU (1) AU2011238708B2 (en)
BR (1) BR112012024934A2 (en)
CA (1) CA2793633A1 (en)
MX (1) MX342858B (en)
SG (3) SG10201908848RA (en)
WO (1) WO2011126808A2 (en)

Families Citing this family (123)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9315825B2 (en) * 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
IN2014DN08812A (en) 2012-04-18 2015-05-22 Philadelphia Children Hospital
TWI775096B (en) * 2012-05-15 2022-08-21 澳大利亞商艾佛蘭屈澳洲私營有限公司 Treatment of amd using aav sflt-1
WO2013188316A1 (en) * 2012-06-11 2013-12-19 Avalanche Biotechnologies, Inc. Optical regulation of gene expression in the retina
CN103088009B (en) * 2013-02-18 2014-09-03 中国科学院微生物研究所 Polypeptide and application thereof in regulating protein accumulation degree by micromolecules
US20140271550A1 (en) 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
KR102346455B1 (en) 2013-03-15 2022-01-04 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Compositions and methods for treating mpsi
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
CN103352053A (en) * 2013-07-11 2013-10-16 江苏省原子医学研究所 Exogenous-gene removable slow virus controlled expression carrier system and application
EP2843414B1 (en) * 2013-08-26 2018-09-19 Roche Diagniostics GmbH Marker for statin treatment stratification in heart failure
PT3116900T (en) 2014-03-09 2020-10-08 Univ Pennsylvania Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency
SG10201810150UA (en) 2014-03-17 2018-12-28 Adverum Biotechnologies Inc Compositions and methods for enhanced gene expression in cone cells
EP3919508A1 (en) 2014-04-25 2021-12-08 The Trustees of The University of Pennsylvania Ldlr variants and their use in compositions for reducing cholesterol levels
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
CA2966620A1 (en) 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
SG11201703419UA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Modulatory polynucleotides
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
WO2016098078A2 (en) * 2014-12-19 2016-06-23 Novartis Ag Dimerization switches and uses thereof
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
AU2016226289B2 (en) 2015-03-02 2021-04-29 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
WO2016176191A1 (en) 2015-04-27 2016-11-03 The Trustees Of The University Of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
US10662440B2 (en) 2015-06-19 2020-05-26 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
WO2017015637A1 (en) 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
JP6978407B2 (en) 2015-08-06 2021-12-08 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Its use in compositions for treating GLP-1 and metabolic disorders
MX2018002339A (en) 2015-08-25 2018-12-19 Univ Duke Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases.
AU2016315699B2 (en) 2015-08-31 2021-09-09 The Trustees Of The University Of Pennsylvania AAV-EPO for treating companion animals
WO2017053732A2 (en) 2015-09-24 2017-03-30 The Trustees Of The University Of Pennsylvania Composition and method for treating complement-mediated disease
WO2017062750A1 (en) 2015-10-09 2017-04-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating stargardt's disease and other ocular disorders
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
KR20180100304A (en) 2015-10-28 2018-09-10 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 Intra-spinal administration of adeno-associated-viral vectors for gene therapy
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
US10889832B2 (en) 2015-12-11 2021-01-12 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
CN109069668B (en) 2015-12-14 2023-04-18 宾夕法尼亚州大学信托人 Gene therapy for eye diseases
JP7061067B2 (en) 2015-12-14 2022-04-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Composition for the treatment of Crigler-Najer syndrome
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
MA43968A (en) 2016-02-03 2018-12-12 Univ Pennsylvania GENE THERAPY TO TREAT MUCOPOLYSACCHARIDOSIS TYPE I
MX2018012537A (en) 2016-04-15 2019-02-25 Univ Pennsylvania Gene therapy for treating hemophilia a.
CN109661470A (en) 2016-04-15 2019-04-19 宾夕法尼亚州大学信托人 New A AV8 mutant capsids and composition containing it
US11191847B2 (en) 2016-04-15 2021-12-07 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia B
IL305449A (en) 2016-04-15 2023-10-01 Univ Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
KR102392236B1 (en) 2016-05-18 2022-05-03 보이저 테라퓨틱스, 인크. Regulatory Polynucleotides
SG11201809643UA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
KR20230062878A (en) 2016-07-08 2023-05-09 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Methods and compositions for treatment of disorders and diseases involving rdh12
WO2018022511A1 (en) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
IL305149A (en) 2016-07-26 2023-10-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
US11298041B2 (en) 2016-08-30 2022-04-12 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
WO2018050783A1 (en) * 2016-09-14 2018-03-22 Ruprecht-Karls-Universität Adeno-associated virus (aav) based regulatory system
TW201819398A (en) 2016-09-28 2018-06-01 美商寇峇有限公司 Therapeutic peptides
JP7065852B2 (en) 2016-12-01 2022-05-12 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル Pharmaceutical composition for the treatment of retinal degenerative diseases
BR112019013576A2 (en) 2016-12-30 2020-02-04 Univ Pennsylvania gene therapy for the treatment of phenylketonuria
US11473106B2 (en) 2016-12-30 2022-10-18 The Trustees Of The University Of Pennsylvania Gene therapy for treating Wilson's disease
WO2018144709A2 (en) 2017-02-01 2018-08-09 The Trustees Of The University Of Pennsylvania Gene therapy for treating citrullenemia
WO2018152485A1 (en) 2017-02-20 2018-08-23 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
WO2018156892A1 (en) 2017-02-23 2018-08-30 Adrx, Inc. Peptide inhibitors of transcription factor aggregation
SG11201907714UA (en) 2017-02-28 2019-09-27 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
JOP20190200A1 (en) 2017-02-28 2019-08-27 Univ Pennsylvania Compositions useful in treatment of spinal muscular atrophy
CA3052487A1 (en) 2017-02-28 2018-09-07 The Trustees Of The University Of Pennsylvania Influenza vaccines based on aav vectors
US11564996B2 (en) 2017-03-01 2023-01-31 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
US11879133B2 (en) 2017-04-24 2024-01-23 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
WO2018204803A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
CN110913866A (en) 2017-05-05 2020-03-24 沃雅戈治疗公司 Compositions and methods for treating Amyotrophic Lateral Sclerosis (ALS)
WO2018209205A1 (en) 2017-05-11 2018-11-15 The Trustees Of The University Of Pennsylvania Gene therapy for neuronal ceroid lipofuscinoses
EP3630986A4 (en) 2017-05-31 2021-08-11 The Trustees of The University of Pennsylvania Gene therapy for treating peroxisomal disorders
EP3634978A1 (en) 2017-06-07 2020-04-15 Adrx, Inc. Tau aggregation inhibitors
US11827898B2 (en) 2017-06-14 2023-11-28 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CA3068328A1 (en) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
TWI835747B (en) 2017-09-22 2024-03-21 賓州大學委員會 Gene therapy for treating mucopolysaccharidosis type ii
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
JP7502991B2 (en) 2017-10-16 2024-06-19 ボイジャー セラピューティクス インコーポレイテッド Treatment of amyotrophic lateral sclerosis (ALS)
WO2019079496A2 (en) * 2017-10-18 2019-04-25 Regenxbio, Inc. Fully-human post-translationally modified antibody therapeutics
KR20200104307A (en) 2017-11-30 2020-09-03 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 Gene therapy for mucopolysaccharide type IIIA
EP3717652A4 (en) 2017-11-30 2021-11-24 The Trustees of the University of Pennsylvania Gene therapy for mucopolysaccharidosis iiib
US11629198B2 (en) 2017-12-05 2023-04-18 The Trustees Of The University Of Pennsylvania Fusion proteins and antibodies targeting human red blood cell antigens
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
WO2019183634A1 (en) * 2018-03-23 2019-09-26 Inscopix, Inc. Reagent coated lenses
CN113164762A (en) 2018-05-09 2021-07-23 生物马林药物股份有限公司 Method for treating phenylketonuria
TW202005978A (en) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 Novel liver targeting adeno-associated viral vectors
CN108841868A (en) * 2018-05-31 2018-11-20 康霖生物科技（杭州）有限公司 A kind of gene order construct for Treatment of Central Nervous System Diseases
JP2022512589A (en) 2018-10-01 2022-02-07 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Composition useful for the treatment of GM1 gangliosidosis
AU2020216135A1 (en) 2019-01-28 2021-08-05 Cohbar, Inc. Therapeutic peptides
KR20210131370A (en) 2019-02-22 2021-11-02 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Recombinant adeno-associated virus for the treatment of GRN-associated adult-onset neurodegeneration
KR20210132095A (en) 2019-02-26 2021-11-03 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Compositions useful for the treatment of Krabe's disease
CN110423281B (en) * 2019-07-31 2021-04-30 成都金唯科生物科技有限公司 Fusion protein, viral vector and drug for treating age-related macular degeneration
TW202140791A (en) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 Methods of treating phenylketonuria
EP4096721A1 (en) 2020-02-02 2022-12-07 The Trustees of The University of Pennsylvania Compositions useful for treating gm1 gangliosidosis
US20230089312A1 (en) * 2020-02-25 2023-03-23 University Of Massachusetts Inducible single aav system and uses thereof
US20230304034A1 (en) 2020-05-12 2023-09-28 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
AU2021271635A1 (en) 2020-05-12 2023-01-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
JP2023531451A (en) 2020-06-17 2023-07-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Compositions and methods for treatment of gene therapy patients
EP4179097A1 (en) 2020-07-13 2023-05-17 The Trustees of The University of Pennsylvania Compositions useful for treatment of charcot-marie-tooth disease
AU2021325954A1 (en) 2020-08-14 2023-03-02 The Trustees Of The University Of Pennsylvania Novel AAV capsids and compositions containing same
MX2023002293A (en) 2020-08-24 2023-05-19 Univ Pennsylvania Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases.
BR112023002904A2 (en) 2020-08-26 2023-04-25 Univ Pennsylvania RECOMBINANT ADENO-ASSOCIATED VIRUS TO TREAT ADULT-ONCEIVE NEURODEGENERATION ASSOCIATED WITH GRN
IL301643A (en) 2020-10-07 2023-05-01 Regenxbio Inc Gene therapy for ocular manifestations of cln2 disease
US20230365955A1 (en) 2020-10-09 2023-11-16 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
IL302127A (en) 2020-10-18 2023-06-01 Univ Pennsylvania Improved adeno-associated virus (aav) vector and uses therefor
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
TW202233844A (en) 2020-10-29 2022-09-01 賓州大學委員會 Aav capsids and compositions containing same
US20240024507A1 (en) 2020-12-01 2024-01-25 The Trustees Of The University Of Pennsylvania Novel compositions with tissue-specific targeting motifs and compositions containing same
JP2023551911A (en) 2020-12-01 2023-12-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Compositions and uses thereof for the treatment of Angelman syndrome
CA3209779A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
EP4323520A1 (en) 2021-04-12 2024-02-21 The Trustees of The University of Pennsylvania Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
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
AR125467A1 (en) 2021-04-27 2023-07-19 Univ Pennsylvania PIG DERIVED ADENO ASSOCIATED VIRUS CAPSIDS AND THEIR USES
TW202325845A (en) 2021-10-02 2023-07-01 賓州大學委員會 Novel aav capsids and compositions containing same
AU2022373653A1 (en) * 2021-10-18 2024-05-02 Regeneron Pharmaceuticals, Inc. Eukaryotic cells comprising adenovirus-associated virus polynucleotides
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
TW202338086A (en) 2022-01-10 2023-10-01 賓州大學委員會 Compositions useful in treatment of metachromatic leukodystrophy
TW202340467A (en) 2022-01-10 2023-10-16 賓州大學委員會 Compositions and methods useful for treatment of c9orf72-mediated disorders
WO2023147304A1 (en) 2022-01-25 2023-08-03 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
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
WO2023201308A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Gene therapy for treating an ocular disease
WO2023205610A2 (en) 2022-04-18 2023-10-26 Regenxbio Inc. Hybrid aav capsids
WO2024015966A2 (en) 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same

Family Cites Families (111)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US318755A (en) 1885-05-19 Attachment foe sewing machines
US622349A (en) 1899-04-04 Wiliielm halstenbach
GB1590524A (en) 1976-11-24 1981-06-03 Nat Res Dev Assay of immune complexes
US4210622A (en) 1977-09-07 1980-07-01 National Research Development Corporation Kit for assay of immune complexes
US4331649A (en) 1978-10-10 1982-05-25 Burroughs Wellcome Co. Immune complex assay
EP0091760B1 (en) 1982-04-09 1986-07-02 FUJIREBIO KABUSHIKI KAISHA also trading as FUJIREBIO INC. Anti immune complex antibody and preparation thereof
US4886876A (en) 1983-03-31 1989-12-12 Scripps Clinic And Research Foundation Factor VIII coagulant polypeptides
US4757006A (en) 1983-10-28 1988-07-12 Genetics Institute, Inc. Human factor VIII:C gene and recombinant methods for production
WO1985001961A1 (en) 1983-10-28 1985-05-09 Genetics Institute Production of factor viii and related products
US5045455A (en) 1984-01-12 1991-09-03 Chiron Corporation Factor VIII:C cDNA cloning and expression
US4965199A (en) 1984-04-20 1990-10-23 Genentech, Inc. Preparation of functional human factor VIII in mammalian cells using methotrexate based selection
IL74909A (en) 1984-04-20 1992-01-15 Genentech Inc Preparation of functional human factor viii and dna sequences,expression vectors,transformed microorganisms and cell lines used therein
EP0182448A3 (en) 1984-08-24 1987-10-28 Genetics Institute, Inc. Production of factor viii and related products
US4753893A (en) 1985-05-31 1988-06-28 Biostar Medical Products, Inc. Method and article for detection of immune complexes
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
FI98829C (en) 1986-01-27 1997-08-25 Chiron Corp Process for Preparation of Recombinant Protein Complex with Human Factor VIII: C Activity
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US5451521A (en) 1986-05-29 1995-09-19 Genetics Institute, Inc. Procoagulant proteins
US5422260A (en) 1986-05-29 1995-06-06 Genetics Institute, Inc. -Legal Affairs Human factor VIII:c muteins
US5149637A (en) 1987-04-06 1992-09-22 Scripps Clinic & Research Foundation Recombinant Factor VIIIC fragments
IE69026B1 (en) 1987-06-12 1996-08-07 Immuno Ag Novel proteins with factor VIII activity process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them
FR2619314B1 (en) 1987-08-11 1990-06-15 Transgene Sa FACTOR VIII ANALOG, PREPARATION METHOD AND PHARMACEUTICAL COMPOSITION CONTAINING THE SAME
US5004803A (en) 1988-11-14 1991-04-02 Genetics Institute, Inc. Production of procoagulant proteins
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
EP0500734B1 (en) 1989-11-17 1998-02-11 Novo Nordisk A/S Protein complexes having factor viii:c activity and production thereof
SE465222C5 (en) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab A recombinant human factor VIII derivative and process for its preparation
US5661008A (en) 1991-03-15 1997-08-26 Kabi Pharmacia Ab Recombinant human factor VIII derivatives
SE468050C (en) 1991-03-15 1998-04-27 Pharmacia & Upjohn Ab Recombinant human factor VIII derivative
CA2078721A1 (en) 1991-09-24 1993-03-25 Hiroshi Yonemura Process for preparing human coagulation factor viii protein complex
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5563045A (en) 1992-11-13 1996-10-08 Genetics Institute, Inc. Chimeric procoagulant proteins
AU5602394A (en) 1992-11-13 1994-06-08 Duke University Chimeric blood coagulation proteins
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
NZ261277A (en) 1993-02-05 1997-04-24 Laporte Group Australia Ltd Slag defoaming composition comprising a carbon source; an exothermic material (eg aluminium) and a dense inert granular material (eg calcium silicate)
US5830462A (en) 1993-02-12 1998-11-03 President & Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
US6972193B1 (en) 1993-02-12 2005-12-06 Board Of Trustees Of Leland Stanford Junior University Regulated transcription of targeted genes and other biological events
US6140120A (en) 1993-02-12 2000-10-31 Board Of Trustees Of Leland Stanford Jr. University Regulated transcription of targeted genes and other biological events
US5834266A (en) 1993-02-12 1998-11-10 President & Fellows Of Harvard College Regulated apoptosis
US5869337A (en) 1993-02-12 1999-02-09 President And Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
JP3824633B2 (en) 1993-02-12 2006-09-20 ザ・ボード・オブ・トラスティーズ・オブ・ザ・リランド・スタンフォード・ジュニアー・ユニバーシティ Regulatory transcription of target genes and other biological consequences
US20020173474A1 (en) 1993-02-12 2002-11-21 President And Fellows Of Harvard College Methods & materials involving dimerization-mediated regulation of biological events
ATE272122T1 (en) 1993-06-10 2004-08-15 Bayer Ag VECTORS AND CELL LINES OF MAMMALS WITH INCREASED PRODUCTIVITY
US6150137A (en) 1994-05-27 2000-11-21 Ariad Pharmaceuticals, Inc. Immunosuppressant target proteins
US6476200B1 (en) 1994-06-27 2002-11-05 The Johns Hopkins University Mammalian proteins that bind to FKBP12 in a rapamycin-dependent fashion
US6492106B1 (en) 1994-06-27 2002-12-10 The Johns Hopkins University Mammalian proteins that bind to FKBP12 in a rapamycin-dependent fashion
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6133456A (en) 1994-08-18 2000-10-17 Ariad Gene Therapeutics, Inc. Synthetic multimerizing agents
US6150527A (en) 1994-08-18 2000-11-21 Ariad Pharmaceuticals, Inc. Synthetic multimerizing agents
ATE299145T1 (en) 1994-08-18 2005-07-15 Ariad Gene Therapeutics Inc NEW MULTIMERIZING REAGENT
JPH08178926A (en) 1994-10-25 1996-07-12 Sumitomo Pharmaceut Co Ltd Immunoassay plate and use thereof
EP0805819B1 (en) 1994-12-29 2012-02-08 Massachusetts Institute Of Technology Chimeric dna-binding proteins
US6326166B1 (en) 1995-12-29 2001-12-04 Massachusetts Institute Of Technology Chimeric DNA-binding proteins
US5681746A (en) 1994-12-30 1997-10-28 Chiron Viagene, Inc. Retroviral delivery of full length factor VIII
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
CA2219080A1 (en) 1995-06-07 1996-12-27 Ariad Gene Therapeutics, Inc. Rapamycin-based regulation of biological events
US6187757B1 (en) 1995-06-07 2001-02-13 Ariad Pharmaceuticals, Inc. Regulation of biological events using novel compounds
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
AU6486196A (en) 1995-07-11 1997-02-10 Chiron Corporation Novel factor viii:c polypeptide analogs with altered protease sites
CA2244363C (en) 1996-02-28 2006-11-14 Ariad Gene Therapeutics, Inc. Synthetic derivatives of rapamycin as multimerizing agents for chimeric proteins with immunophilin-derived domains
AU735648B2 (en) 1996-07-12 2001-07-12 Ariad Pharmaceuticals, Inc. Materials and method for treating or preventing pathogenic fungal infection
JP4135120B2 (en) 1997-04-14 2008-08-20 セル ジェネシス，インコーポレーテッド Method for increasing the efficiency of recombinant AAV production
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6015709A (en) 1997-08-26 2000-01-18 Ariad Pharmaceuticals, Inc. Transcriptional activators, and compositions and uses related thereto
JP2003524368A (en) 1997-08-26 2003-08-19 アリアド ジーン セラピューティクス インコーポレイテッド Fusion protein comprising a dimerization domain, a trimerization domain or a tetramerization domain and a complementary heterologous transcriptional activation domain, a transcription repression domain, a DNA binding domain or a ligand binding domain
US6479653B1 (en) 1997-08-26 2002-11-12 Ariad Gene Therapeutics, Inc. Compositions and method for regulation of transcription
AU752129B2 (en) 1997-08-27 2002-09-05 Ariad Gene Therapeutics, Inc. Chimeric transcriptional activators and compositions and uses related thereto
WO1999036553A2 (en) 1998-01-15 1999-07-22 Ariad Gene Therapeutics, Inc. Regulation of biological events using multimeric chimeric proteins
US6984635B1 (en) 1998-02-13 2006-01-10 Board Of Trustees Of The Leland Stanford Jr. University Dimerizing agents, their production and use
AU766513B2 (en) 1998-02-13 2003-10-16 Board Of Trustees Of The Leland Stanford Junior University Novel dimerizing agents, their production and use
EP1080218A1 (en) 1998-05-27 2001-03-07 University of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US7109317B1 (en) 1998-11-06 2006-09-19 President And Fellows Of Harvard College FK506-based regulation of biological events
WO2000028004A1 (en) 1998-11-10 2000-05-18 The University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
GB9917512D0 (en) 1999-07-26 1999-09-29 Univ Southampton Data and/or video communications
JP4827353B2 (en) 1999-08-09 2011-11-30 ターゲティッド ジェネティクス コーポレイション Increased expression of single-stranded heterologous nucleotide sequences from recombinant viral vectors by designing sequences to form intrastrand base pairs
AU783158B2 (en) 1999-08-24 2005-09-29 Ariad Pharmaceuticals, Inc. 28-epirapalogs
US7067526B1 (en) 1999-08-24 2006-06-27 Ariad Gene Therapeutics, Inc. 28-epirapalogs
WO2001083692A2 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
US20030013189A1 (en) 2000-04-28 2003-01-16 Wilson James M. Compositions and methods useful for non-invasive delivery of therapeutic molecules to the bloodstream
DE10023887A1 (en) * 2000-05-17 2001-11-29 Axel Haverich Nucleic acid construct for transient cell transfection, useful ,e.g., in gene therapy, contains recombinase gene controlled by inducible promoter and flanking recombinase sites
JP2004501113A (en) 2000-06-01 2004-01-15 ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル Methods and formulations for controlled release of recombinant parvovirus vectors
DE10110449A1 (en) * 2001-03-05 2002-09-19 Lisa Wiesmueller Test system for the determination of genotoxicity
NZ545697A (en) * 2001-05-30 2008-06-30 Glaxo Group Ltd A lambda-intR mutein comprising a glutamic acid to arginine change at position 174 of Wt lambda-intR
JP2003033179A (en) * 2001-07-05 2003-02-04 Asahi Kasei Corp Vector for reversible transduction of gene
JP2004538005A (en) 2001-08-08 2004-12-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Method for purifying a viral vector having a protein binding to sialic acid
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
AU2002359284A1 (en) 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
AU2002360291A1 (en) 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
DE60310297T2 (en) 2002-04-29 2007-06-06 Trustees Of The University Of Pennsylvania Method for direct recovery and amplification of integrated viruses from cellular tissue DNA
US7247328B2 (en) 2002-05-31 2007-07-24 Zinpro Corporation Chromium (III) alpha amino acid complexes
WO2003104413A2 (en) 2002-06-05 2003-12-18 University Of Florida Production of pseudotyped recombinant aav virions
AU2003265855A1 (en) 2002-08-28 2004-03-19 University Of Florida Modified aav
WO2004048583A2 (en) * 2002-11-22 2004-06-10 Institut Clayton De La Recherche Compositions and systems for the regulation of genes
WO2004108922A2 (en) 2003-04-25 2004-12-16 The Trustees Of The University Of Pennsylvania Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver
JP5054975B2 (en) 2003-09-30 2012-10-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Syngeneic strains of adeno-associated virus (AAV), sequences, vectors containing them and uses thereof
US7273266B2 (en) 2004-04-14 2007-09-25 Lexmark International, Inc. Micro-fluid ejection assemblies
US7638119B2 (en) * 2004-12-02 2009-12-29 Wisconsin Alumni Research Foundation Method of diminishing the symptoms of neurodegenerative disease
US20080274093A1 (en) * 2004-12-02 2008-11-06 Johnson Jeffrey A Method of diminishing the symptoms of neurodegenerative disease
JP5702519B2 (en) 2005-04-07 2015-04-15 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア How to increase the function of an AAV vector
CN100513623C (en) 2005-04-21 2009-07-15 中国科学院物理研究所 Cerium-based non-crystalline metal plastics
JP4495210B2 (en) 2005-06-09 2010-06-30 パナソニック株式会社 Amplitude error compensator and orthogonality error compensator
US20090317417A1 (en) 2006-04-28 2009-12-24 The Trustees Of The University Of Pennsylvania Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof
ES2400235T3 (en) 2006-04-28 2013-04-08 The Trustees Of The University Of Pennsylvania AAV scalable production method
US9388425B2 (en) * 2006-10-20 2016-07-12 Trustees Of Boston University Tunable genetic switch for regulating gene expression
JP2009171880A (en) * 2008-01-23 2009-08-06 Yokohama City Univ Development of next-generation gene therapy and immunotherapy in alzheimer's disease
JP2009171890A (en) * 2008-01-24 2009-08-06 Tsukishima Foods Industry Co Ltd Antioxidant bread and confectionery
WO2009146179A1 (en) * 2008-04-15 2009-12-03 University Of Iowa Research Foundation Zinc finger nuclease for the cftr gene and methods of use thereof
US9408008B2 (en) 2014-02-28 2016-08-02 Sonos, Inc. Playback zone representations
US20160120174A1 (en) 2014-10-29 2016-05-05 Ronald Steven Cok Imprinted multi-layer biocidal particle structure

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