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公开(公告)号：US11883506B2
公开(公告)日：2024-01-30
申请号：US17670389
申请日：2022-02-11
IPC分类号： A61K48/00 , C12N15/864 , C12N15/113 , C12N15/02 , A61P9/00 , C07K14/005 , C12N15/86 , A61K38/00
CPC分类号： A61K48/005 , C07K14/005 , C12N15/113 , C12N15/86 , A61K38/00 , C12N2750/14143
摘要： Provided herein is a gene therapy for PKP2 (Plakophilin-2), e.g. using an adeno-associated virus (AAV) vector. The promoter of the vector may be a MHCK7 promoter or a cardiac troponin T (HTNNT2) promoter. The capsid may be an AAV9 or AAVrh74 capsid or a functional variant thereof. Other promoters or capsids may be used. Further provided are methods of treatment, such as by intravenous, intracoronary, intracarotid or intracardiac administration of the rAAV vector, and other compositions and methods.
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公开(公告)号：US20220143215A1
公开(公告)日：2022-05-12
申请号：US17430107
申请日：2020-02-12
发明人： Annahita KERAVALA , Raj PRABHAKAR , Gaurav SHAH , Roderick WONG , Naveen YALAMANCHI , Piratip PRATUMSUWAN
IPC分类号： A61K48/00 , A61P21/00 , C07K14/005 , C07K14/705 , C12N15/86
摘要： The present disclosure provides gene therapy vectors comprising a polynucleotide sequence encoding a LAMP-2 polypeptide, methods of use thereof, pharmaceutical compositions, and more. In particular, the disclosure provides recombinant AAV vectors having AAVrh74 serotype expressing LAMP-2A, LAMP-2B, or LAMP-2C for use as a therapeutic in, for example, Danon Disease.
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公开(公告)号：US20210381003A1
公开(公告)日：2021-12-09
申请号：US17144933
申请日：2021-01-08
发明人： Annahita KERAVALA , Simon MOORE , David RICKS
摘要： The disclosure relates to optimized polynucleotide sequences for LAMP-2B, expression cassettes, vectors, and methods of use thereof in treating disease, e.g. Danon disease.
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公开(公告)号：US20240335566A1
公开(公告)日：2024-10-10
申请号：US18718023
申请日：2022-12-09
CPC分类号： A61K48/0058 , A61K38/1709 , C07K14/47 , C12N15/86 , C12N2750/14143 , C12N2830/008 , C12N2830/48 , C12N2830/50
摘要： Provided herein is a gene therapy for TNNC1 (Troponin C)-related cardiomyopathy, e.g. using an adeno-associated virus (AAV) vector. The promoter of the vector may be a MHCK7 promoter or a cardiac troponin T (hTNNT2) promoter. The capsid may be an AAV9 or AAVrh.74 capsid or a functional variant thereof. Other promoters or capsids may be used. Further provided are methods of treatment, such as by intravenous, intracoronary, intracarotid or intracardiac administration of the rAAV vector, and other compositions and methods.
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公开(公告)号：US20220168447A1
公开(公告)日：2022-06-02
申请号：US17670390
申请日：2022-02-11
IPC分类号： A61K48/00 , C12N15/113 , C12N15/86
摘要： Provided herein is a gene therapy for PKP2 (Plakophilin-2), e.g. using an adeno-associated virus (AAV) vector. The promoter of the vector may be a MHCK7 promoter or a cardiac troponin T (HTNNT2) promoter. The capsid may be an AAV9 or AAVrh74 capsid or a functional variant thereof. Other promoters or capsids may be used. Further provided are methods of treatment, such as by intravenous, intracoronary, intracarotid or intracardiac administration of the rAAV vector, and other compositions and methods.
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公开(公告)号：US20210198695A1
公开(公告)日：2021-07-01
申请号：US17268409
申请日：2019-08-16
发明人： David RICKS , Brian BEARD , Kenneth LAW , Raj PRABHAKAR , Kinnari PATEL
摘要： The present disclosure provides methods for manufacturing a recombinant lentiviral vectors in an adherent bioreactor, for example, by calcium-phosphate transfection of cells grown in adherent mode on low-compaction macrocarriers in an iCELLis® bioreactor system.
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公开(公告)号：US20240335565A1
公开(公告)日：2024-10-10
申请号：US18718021
申请日：2022-12-07
CPC分类号： A61K48/0058 , A61K38/1709 , A61K48/0083 , A61P9/10 , C12N15/86 , C12N2750/14143 , C12N2830/48 , C12N2830/50
摘要： Provided herein is a gene therapy for JPH2 (Junctophilin-2), e.g., using an adeno-associated virus (AAV) vector. The promoter of the vector may be a MHCK7 promoter or a cardiac troponin T (HTNNT2) promoter. The capsid may be an AAV9 or AAVrh74 capsid or a functional variant thereof. Other promoters or capsids may be used. Further provided are methods of treatment, such as by intravenous, intracoronary, intracarotid or intracardiac administration of the rAAV vector, and other compositions and methods.
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公开(公告)号：US20230272422A1
公开(公告)日：2023-08-31
申请号：US18019393
申请日：2021-08-03
IPC分类号： C12N15/86 , C07K14/705 , A61P25/00
CPC分类号： C12N15/86 , C07K14/705 , A61P25/00 , C12N2750/14143 , C12N2750/14171
摘要： Provided herein is a gene therapy for GLUT1 Deficiency Syndrome and related disorders using a recombinant adeno-associated virus (rAAV) virion as a vector to express an GLUT1 protein or functional variant thereof. The rAAV virion may use an endothelial-specific promoter, e.g., a FLT-1 or Tie-1 promoter. The capsid may be an AAV6, AAV8, AAV9, AAVrh.74, or AAVrh.10 capsid or a functional variant thereof. Other promoters or capsids may be used. Further provided are methods of treatment, such as by intracerebrally and/or intravenously of the rAAV virion, and other compositions and methods.
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公开(公告)号：US20230174994A1
公开(公告)日：2023-06-08
申请号：US17926024
申请日：2021-05-20
IPC分类号： C12N15/62 , C12N15/86 , C07K14/005
CPC分类号： C12N15/62 , C07K14/005 , C12N15/86 , C07K2319/03 , C07K2319/07 , C12N2750/14143
摘要： Parkin protein variants having activating mutations and/or fused to a mitochondrial targeting sequence are provided. The engineered Parkin may be a fusion protein including a mitochondrial targeting sequence (MTS); a transmembrane domain; and a Parkin protein or functional variant or fragment thereof, such as a Parkin having an N-terminal deletion. The MTS may be the MTS of PINK1 or a functional variant thereof. Alternatively or in addition, the engineered Parkin may have one or more activating mutations, such as single amino-acid substitutions. The engineered Parkin may be delivered in a vector, such as an adeno-associated virus (AAV) vector, and may be used to treat a disease or disorder, such as Parkinson’s disease or any of various neurodegenerative diseases.
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公开(公告)号：US20220218844A1
公开(公告)日：2022-07-14
申请号：US17612134
申请日：2020-05-22
发明人： Brian BEARD , David RICKS , Raj PRABHAKAR
摘要： The present disclosure provides improved gene therapy vectors comprising a polynucleotide sequence encoding a TCIRG1 polypeptide or functional variant thereof, methods of use thereof, pharmaceutical compositions, and more. In particular, the disclosure provides lentiviral vectors for treatment of infantile malignant osteopetrosis (IMO).
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