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公开(公告)号：US12070702B2
公开(公告)日：2024-08-27
申请号：US17251869
申请日：2019-06-13
申请人： REGENXBIO INC.
发明人： Claire G. Zhang , Jeffrey Levine , Sara Zafar , Franz M. Gerner
CPC分类号： B01D15/363 , B01D15/166 , C12N7/00 , C12N2750/14123 , C12N2750/14151
摘要： Provided herein are methods for the purification of recombinant Adeno-Associated Virus (rAAV) particles using anion exchange chromatography.
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62.发明授权公开(公告)号：US12070510B2
公开(公告)日：2024-08-27
申请号：US16075122
申请日：2017-01-25
申请人： Emory University , REGENXBIO Inc.
CPC分类号： A61K48/0075 , A61K9/0085 , A61K45/06 , A61P25/00 , A61P37/00 , C12N15/86 , C12N2750/14143
摘要： It is disclosed herein that ssAAV and scAAV vectors of the same serotype administered by injection into the cerebrospinal fluid (CSF) via the intracerebroventricular (ICV) or intrathecal (cisternal or lumbar) route exhibit different cellular tropisms in the central nervous system. Thus, a subject can be treated by injection into the CSF of ssAAV or scAAV vector encoding a therapeutic protein, such as an ssAAV9 or scAAV9 vector. The therapeutic protein can be targeted to specific cells using these vectors. In some embodiments, scAAV9 is utilized to achieve superior transduction in the hippocampus, cerebellum and cerebral cortex where both neurons, particularly Purkinje neurons, and glial cells (such as astrocytes) are transduced. In other embodiments, ssAAV9 is utilized to minimize transduction of astrocytes. In further embodiments, an immunosuppressive agent is also administered to the subject.
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公开(公告)号：US20240218397A1
公开(公告)日：2024-07-04
申请号：US18558495
申请日：2022-05-03
申请人： REGENXBIO Inc.
发明人： Ye LIU , Andrew MERCER , Chunping QIAO , April R. TEPE , Nicolas BUSS
IPC分类号： C12N15/86
CPC分类号： C12N15/86 , C12N2750/14143 , C12N2830/38
摘要： The present invention relates to nucleic acid expression cassettes that are engineered to include polynucleotide sequences useful as inert stuffer (or filler) sequences in expression cassettes, particularly useful for transgene expression delivered as viral vectors, incorporating the engineered expression cassettes described herein, including rAA Vs, for use in therapy.
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公开(公告)号：US20240216543A1
公开(公告)日：2024-07-04
申请号：US18557155
申请日：2022-04-26
申请人： REGENXBIO INC.
发明人： Olivier Danos , SunJung Kim , Nicholas Buss , Ye Liu , Chunping Qiao , Michele Fiscella , Hiren Patel
IPC分类号： A61K48/00 , A61K9/00 , A61K31/436 , A61K31/573 , A61K38/17 , A61K39/395 , A61P21/00 , C12N15/86
CPC分类号： A61K48/0058 , A61K9/0019 , A61K31/436 , A61K31/573 , A61K38/177 , A61K39/3955 , A61K48/0033 , A61P21/00 , C12N15/86 , C12N2750/14143 , C12N2830/008 , C12N2830/50
摘要： Provided are methods of treating or ameliorating the symptoms of dystrophinopathies, such as Duchenne muscular dystrophy and Becker muscular dystrophy by administration of therapeutically effective doses of recombinant adeno-associated viruses (rAAV) containing a transgene encoding a microdystrophin.
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65.发明授权公开(公告)号：US11986515B2
公开(公告)日：2024-05-21
申请号：US18356961
申请日：2023-07-21
申请人： REGENXBIO Inc.
CPC分类号： A61K38/465 , A61P25/28 , C12N15/86 , C12Y301/06013 , G01N33/5308 , C12N2750/14143 , C12N2750/14171 , G01N2400/00 , G01N2800/04
摘要： Compositions and methods are described for the delivery of recombinant human iduronate-2-sulfatase (IDS) produced by human neuronal or glial cells to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis II (MPS II).
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公开(公告)号：US20240124890A1
公开(公告)日：2024-04-18
申请号：US18034330
申请日：2021-10-28
申请人： REGENXBIO INC.
发明人： Jared Smith , Bradley Hollidge , Joseph Bruder , Ye Liu , Randolph Qian , Chunping Qiao
CPC分类号： C12N15/86 , A61K48/0058 , A61P25/06 , C07K16/18 , C07K16/28 , A61K2039/54 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145
摘要： Compositions and methods are described for the delivery of a fully human post-translationally modified therapeutic monoclonal antibody that binds to CGRP or CGRP receptor to a human subject for the treatment or prevention of migraines and cluster headaches. The antibodies may be delivered by gene therapy vectors, particularly rAAV vectors. Also provided are dual transgene constructs for the delivery of anti-CGRP and anti-CGRP receptor antibodies or antigen binding fragments thereof.
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67.发明公开公开(公告)号：US20240117328A1
公开(公告)日：2024-04-11
申请号：US18276356
申请日：2022-01-31
申请人： REGENXBIO Inc.
CPC分类号： C12N9/16 , A61K38/465 , A61K45/06 , A61K48/005 , A61K48/0075 , A61P3/00 , C12N15/86 , G01N33/5008 , C12N2750/14143 , C12Y301/06013
摘要： Compositions and methods are described for the delivery of recombinant human iduronate-2-sulfatase (IDS) produced by human neuronal or glial cells to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis II (MPS II).
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68.发明公开公开(公告)号：US20240058426A1
公开(公告)日：2024-02-22
申请号：US18356961
申请日：2023-07-21
申请人： REGENXBIO Inc.
CPC分类号： A61K38/465 , A61P25/28 , C12N15/86 , C12Y301/06013 , G01N33/5308 , C12N2750/14143 , C12N2750/14171 , G01N2400/00 , G01N2800/04
摘要： Compositions and methods are described for the delivery of recombinant human iduronate-2-sulfatase (IDS) produced by human neuronal or glial cells to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis II (MPS II).
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69.发明公开公开(公告)号：US20240050537A1
公开(公告)日：2024-02-15
申请号：US18480172
申请日：2023-10-03
申请人： REGENXBIO Inc.
IPC分类号： A61K38/47 , A61P25/28 , A61K31/365 , A61K31/436 , A61K31/573 , A61K48/00
CPC分类号： A61K38/47 , A61P25/28 , A61K31/365 , A61K31/436 , A61K31/573 , A61K48/005 , A61K48/0083 , C12Y302/01076
摘要： Compositions and methods are described for the delivery of a fully human-glycosylated (HuGly) α-L-iduronidase (IDUA) to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis I (MPS I).
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公开(公告)号：US20230323366A1
公开(公告)日：2023-10-12
申请号：US18014977
申请日：2021-07-09
申请人： INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MEDICALE) , UNIVERSITÉ D’AIX-MARSEILLE , UNIVERSITÉ DE BORDEAUS , REGENXBIO INC. , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
发明人： Céline BOILEAU , Valérie CREPEL , Séverine DEFORGES , Julie MASANTE , Christophe NULLE , Angélique PERET , Olivier DANOS , Andrew MERCER
IPC分类号： C12N15/113 , A61P25/08 , C12N15/86
CPC分类号： C12N15/1138 , A61P25/08 , C12N15/86 , C12N2310/11 , C12N2310/531 , C12N2750/14143
摘要： The present disclosure relates to gene therapy targeting GluK2 subunit that can be used to inhibit epileptiform discharges. Short interfering RNA sequences against the human Grik2 gene sequence are described which are efficient in decreasing the expression of GluK2-containing KARs in neurons engineered to express the equivalent shRNA or miRNA. Using a tissue culture model of TLE, the examples remarkably demonstrate that viral expression of shRNA or miRNA inhibits the frequency of epileptiform discharges. Therefore, RNA therapeutics aimed at decreasing the expression of GluK2-containing KARs in neurons can remarkably prevent spontaneous epileptiform discharges in TLE. In particular, the present disclosure relates to a recombinant antisense oligonucleotide that targets a Grik2 mRNA. The present disclosure also relates to a method for treating epilepsy in a subject in need thereof, wherein the method comprises: administering an effective amount of a vector comprising an oligonucleotide encoding an inhibitory RNA that binds (e.g., hybridizes) specifically to Grik2 mRNA and inhibits expression of Grik2 in the subject.
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