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公开(公告)号：US20250163469A1
公开(公告)日：2025-05-22
申请号：US18978736
申请日：2024-12-12
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. Kirn , Melissa KOTTERMAN , David SCHAFFER
IPC: C12N15/86 , A61K9/00 , A61K38/17 , A61K38/44 , A61K38/47 , A61K48/00 , A61P21/00 , C07K14/005 , C12N7/00 , C12N9/10 , C12N15/864
Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of muscle cells as compared to the infectivity of the muscle cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more muscle cells for the treatment of muscle disorders and diseases.
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公开(公告)号：US20240018498A1
公开(公告)日：2024-01-18
申请号：US18463979
申请日：2023-09-08
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. KIRN , Melissa KOTTERMAN , Peter FRANCIS , David SCHAFFER , Paul SZYMANSKI , Kevin WHITTLESEY
CPC classification number: C12N9/2465 , C12Y302/01022 , A61K9/0019 , A61P3/00 , C12N15/86 , A61K48/0066 , A61K48/0075 , A61K48/0083 , C12N2840/00 , A61K48/00
Abstract: The present disclosure provides codon optimized nucleotide sequences encoding hum n alpha-galactosidase A, vectors, and host cells comprising codon optimized alpha-galactosidase A sequences, and methods of treating disorders such as Fabry disease comprising administering to the subject a codon optimized sequence encoding human alpha-galactosidase A.
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公开(公告)号：US20190255192A1
公开(公告)日：2019-08-22
申请号：US16300446
申请日：2017-05-12
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. KIRN , Melissa KOTTERMAN , David SCHAFFER
IPC: A61K48/00 , C12N15/86 , C07K14/005 , A61P27/02 , A61K9/00
Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AA V virion comprising the unmodified parental capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina for the treatment of retinal disorders and diseases.
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公开(公告)号：US20210380643A1
公开(公告)日：2021-12-09
申请号：US17400041
申请日：2021-08-11
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. KIRN , Melissa KOTTERMAN , David SCHAFFER
IPC: C07K14/005 , C12N7/00 , A61P21/00 , A61K9/00 , A61K38/47
Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of muscle cells as compared to the infectivity of the muscle cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more muscle cells for the treatment of muscle disorders and diseases.
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公开(公告)号：US20210069348A1
公开(公告)日：2021-03-11
申请号：US16951984
申请日：2020-11-18
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. KIRN , Melissa KOTTERMAN , David SCHAFFER
IPC: A61K48/00 , A61P27/02 , A61K9/00 , C07K14/005 , C12N15/86
Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina for the treatment of retinal disorders and diseases.
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公开(公告)号：US20200282077A1
公开(公告)日：2020-09-10
申请号：US16765758
申请日：2018-11-26
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. KIRN , Melissa KOTTERMAN , David SCHAFFER , Paul SZYMANSKI , Peter FRANCIS
IPC: A61K48/00 , C12N7/00 , C07K14/005 , A61K9/00 , C07K16/22
Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina for the treatment of retinal disorders and diseases.
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公开(公告)号：US20230001018A1
公开(公告)日：2023-01-05
申请号：US17746443
申请日：2022-05-17
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. KIRN , Melissa KOTTERMAN , David SCHAFFER
IPC: A61K48/00 , A61K9/00 , A61P27/02 , C07K14/005 , C12N15/86
Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more ceils of the retina for the treatment of retinal disorders and diseases.
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公开(公告)号：US20220251145A1
公开(公告)日：2022-08-11
申请号：US17718870
申请日：2022-04-12
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. KIRN , Melissa KOTTERMAN , David SCHAFFER
Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of muscle cells as compared to the infectivity of the muscle cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more muscle cells for the treatment of muscle disorders and diseases.
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公开(公告)号：US20210395772A1
公开(公告)日：2021-12-23
申请号：US17241032
申请日：2021-04-26
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： Melissa KOTTERMAN , Peter FRANCIS , Melissa CALTON , Johnny GONZALES , Roxanne CROZE , Christopher SCHMITT
IPC: C12N15/86
Abstract: The present disclosure provides a variant AAV capsid protein that confers tropism to lung cells and recombinant adeno-associated viruses comprising the variant AAV and pharmaceutical compositions comprising same and their use in the delivery of heterologous nucleic acids to lung cells for the treatment of pulmonary disorders.
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公开(公告)号：US20210162071A1
公开(公告)日：2021-06-03
申请号：US17163093
申请日：2021-01-29
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. KIRN , Melissa KOTTERMAN , David SCHAFFER
IPC: A61K48/00 , A61P27/02 , C07K14/005 , A61K9/00 , C12N15/86
Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina for the treatment of retinal disorders and diseases.
