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公开(公告)号：US12037362B2
公开(公告)日：2024-07-16
申请号：US16981086
申请日：2019-04-04
Applicant: Genethon , Institut National de la Sante et de la Recherche Medicale , Universite d'Evry Val d'Essonne , Sorbonne Universite , Association Institut de Myologie
Inventor： Isabelle Richard , Evelyne Gicquel , Federico Mingozzi
IPC: C07K14/005 , A61K48/00 , A61P25/28 , C07K14/075 , C12N7/00 , C12N9/22 , C12N15/11 , C12N15/86
CPC classification number: C07K14/005 , A61P25/28 , C07K14/075 , C12N7/00 , C12N9/22 , C12N15/111 , C12N15/86 , A61K48/00 , C12N2310/20 , C12N2750/14121 , C12N2750/14122 , C12N2750/14143 , C12N2750/14152 , C12N2750/14171
Abstract: The invention relates to a recombinant adeno-associated virus (AAV) capsid protein, which is a hybrid between AAV serotype 9 (AAV9) and AAV serotype 74 (AAVrh74) capsid proteins, wherein said recombinant hybrid AAV capsid protein has a reduced liver tropism compared to the parent AAV9 and AAVrh74 capsid proteins. The invention relates also to the derived hybrid AAV serotype vector particles packaging a gene of interest and their use in gene therapy, in particular for treating neuromuscular genetic diseases.
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公开(公告)号：US11099193B2
公开(公告)日：2021-08-24
申请号：US15110868
申请日：2015-01-09
Applicant: GENETHON
Inventor： Jeremy Rouillon , Fedor Svinartchouk , Jerome Poupiot , Isabelle Richard
Abstract: The invention relates to a method for prognosing, diagnosing, determining the risk, and monitoring the evolution of a muscular dystrophy. It also relates to a method for evaluating the efficacy of a treatment of a muscular dystrophy in a subject in need thereof.
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3.发明申请USE OF EPIGENOME-MODIFYING COMPOUNDS FOR THE TREATMENT OF GENETIC MUSCULAR DISEASES LINKED TO A PROTEIN-CONFORMATIONAL DISORDER 有权用于治疗与蛋白质一致性疾病相关的遗传性疾病的EPIGENOME-MODIFYING化合物的使用公开(公告)号：US20150141361A1
公开(公告)日：2015-05-21
申请号：US14407211
申请日：2013-07-16
Applicant: GENETHON
Inventor： Isabelle Richard
IPC: A61K31/706 , G01N33/50 , A61K31/167
CPC classification number: A61K31/706 , A61K31/167 , A61K31/7068 , G01N33/5061 , G01N2440/10 , G01N2440/12 , G01N2500/10 , G01N2800/2878
Abstract: A pharmaceutical composition including at least one epigenome-modifying compound, for a use thereof in the treatment of genetic muscular diseases linked to a conformational disorder of at least one protein, said disorder causing the cellular degradation of the protein.
Abstract translation: 一种药物组合物，其包含至少一种表观遗传调节蛋白修饰化合物，用于治疗与至少一种蛋白质的构象紊乱相关的遗传性肌肉疾病，所述疾病导致蛋白质的细胞降解。
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公开(公告)号：US11819478B2
公开(公告)日：2023-11-21
申请号：US15988728
申请日：2018-05-24
Applicant: GENETHON , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
Inventor： Ana Maria Buj Bello , Isabelle Richard
CPC classification number: A61K48/0066 , A61K38/465 , A61K38/4873 , A61K48/0058 , A61K48/0075 , C12N9/16 , C12N9/6472 , C12N15/86 , C07K14/4707 , C12N2750/14132 , C12N2750/14143 , C12N2830/008 , C12Y301/03048 , C12Y304/22054
Abstract: The present invention relates to an expression system for systemic administration comprising a sequence encoding a protein, said expression system allowing:
the expression at a therapeutically acceptable level of the protein in the target tissues including skeletal muscles; and
the expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart.-
公开(公告)号：US09981049B2
公开(公告)日：2018-05-29
申请号：US14782396
申请日：2014-04-10
Applicant: GENETHON , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
Inventor： Ana Maria Buj Bello , Isabelle Richard
CPC classification number: A61K48/0066 , A61K38/465 , A61K38/4873 , A61K48/0058 , A61K48/0075 , C07K14/4707 , C12N9/16 , C12N9/6472 , C12N15/86 , C12N2750/14132 , C12N2750/14143 , C12N2830/008 , C12Y301/03048 , C12Y304/22054
Abstract: The present invention relates to an expression system for systemic administration comprising a sequence encoding a protein, said expression system allowing: the expression at a therapeutically acceptable level of the protein in the target tissues including skeletal muscles and/or the peripheral nervous tissue; and the expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart.
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Patent Agency Ranking
公开(公告)号：US20240209030A1
公开(公告)日：2024-06-27
申请号：US18024103
申请日：2021-09-10
Applicant: Genethon , Universite d'Evry Val d'Essonne , Institut National de la Santé et de la Recherche Médicale
Inventor： Isabelle Richard , Natalia Dominguez
IPC: C07K14/005 , C12N15/86
CPC classification number: C07K14/005 , C12N15/86 , C12N2750/14122 , C12N2750/14143
Abstract: The invention relates to a peptide-modified AAV capsid with improved tropism, in particular increased muscle tropism and/or reduced liver tropism. The invention relates also to the derived recombinant AAV vector particle packaging a gene of interest, and its use in gene therapy, in particular for treating muscle diseases.
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公开(公告)号：US20230250450A1
公开(公告)日：2023-08-10
申请号：US18009025
申请日：2021-06-09
Applicant: Genethon , Universite d'Evry Val d'Essonne , INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE
Inventor： Isabelle Richard
CPC classification number: C12N15/86 , A61P9/10 , A61K38/1709 , C12N2750/14143 , C12N2750/14171 , A61K48/00
Abstract: The present invention relates to the treatment of dilated cardiomyopathies, in particular to the use of an activator of NRF2.
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公开(公告)号：US11298348B2
公开(公告)日：2022-04-12
申请号：US16960503
申请日：2019-01-10
Applicant: INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , UNIVERSITE D'EVRY-VAL-D'ESSONNE , GENETHON , UNIVERSITA 'DEGLI STUDI DI PADOVA
Inventor： Isabelle Richard , Dorianna Sandona
IPC: A61K31/47 , A61P21/00 , A61K31/427
Abstract: Sarcoglycanopathies are autosomal recessive diseases caused by mutations in the one of the genes coding for any sarcoglycans (SG). The inventors previously showed that the application of small molecules developed to rescue ΔF508-CFTR trafficking, and known as CFTR correctors, improved the maturation of several α-sarcoglycan mutants that were consequently rescued at the plasma membrane (WO 014086687). Now, the inventors show that some specific CFTR correctors provide additive and even synergic effect when administered in combination.
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9.发明授权Use of epigenome-modifying compounds for the treatment of genetic muscular diseases linked to a protein-conformational disorder 有权表观遗传改变化合物用于治疗与蛋白质构象紊乱相关的遗传性肌肉疾病的用途公开(公告)号：US09526742B2
公开(公告)日：2016-12-27
申请号：US14407211
申请日：2013-07-16
Applicant: GENETHON
Inventor： Isabelle Richard
IPC: A61K31/706 , A61K31/167 , G01N33/50 , A61K31/7068
CPC classification number: A61K31/706 , A61K31/167 , A61K31/7068 , G01N33/5061 , G01N2440/10 , G01N2440/12 , G01N2500/10 , G01N2800/2878
Abstract: A pharmaceutical composition including at least one epigenome-modifying compound, for a use thereof in the treatment of genetic muscular diseases linked to a conformational disorder of at least one protein, said disorder causing the cellular degradation of the protein.
Abstract translation: 一种包含至少一种表观遗传调节蛋白修饰化合物的药物组合物，用于治疗与至少一种蛋白质的构象紊乱相关的遗传性肌肉疾病，所述疾病导致蛋白质的细胞降解。
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公开(公告)号：US20160058890A1
公开(公告)日：2016-03-03
申请号：US14782396
申请日：2014-04-10
Applicant: GENETHON , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
Inventor： Ana Maria Buj Bello , Isabelle Richard
CPC classification number: A61K48/0066 , A61K38/465 , A61K38/4873 , A61K48/0058 , A61K48/0075 , C07K14/4707 , C12N9/16 , C12N9/6472 , C12N15/86 , C12N2750/14132 , C12N2750/14143 , C12N2830/008 , C12Y301/03048 , C12Y304/22054
Abstract: The present invention relates to an expression system for systemic administration comprising a sequence encoding a protein, said expression system allowing: the expression at a therapeutically acceptable level of the protein in the target tissues including skeletal muscles and/or the peripheral nervous tissue; and the expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart.
Abstract translation: 本发明涉及全身给药的表达系统，其包含编码蛋白质的序列，所述表达系统允许：包括骨骼肌和/或周围神经组织在内的靶组织中蛋白质的治疗可接受水平的表达; 以及在靶组织以外的组织中，特别是心脏中的蛋白质在毒性可接受水平的表达。
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