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公开(公告)号：US20230279435A1
公开(公告)日：2023-09-07
申请号：US18069701
申请日：2022-12-21
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. Kirn , Melissa Kotterman , David Schaffer
IPC: C12N15/86 , C07K14/005 , C12N7/00 , C12N15/864 , A61K38/17 , A61K38/44 , A61P21/00 , C12N9/10 , A61K38/47 , A61K9/00 , A61K48/00
CPC classification number: C12N15/86 , C07K14/005 , C12N7/00 , C12N15/8645 , A61K38/1719 , C12Y302/01022 , C12Y204/01001 , C12Y116/03001 , C12Y302/0102 , A61K38/44 , A61P21/00 , C12N9/1051 , A61K38/47 , C12N2750/14121 , A61K9/0019 , C12N2750/14171 , C12N2750/14145 , C12N2750/14133 , A61K48/00 , C12N2750/14122 , C12N2750/14143
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：US11419949B2
公开(公告)日：2022-08-23
申请号：US17163093
申请日：2021-01-29
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 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：US11840704B2
公开(公告)日：2023-12-12
申请号：US18069701
申请日：2022-12-21
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. Kirn , Melissa Kotterman , David Schaffer
IPC: C12N15/86 , C07K14/005 , C12N15/864 , A61P21/00 , A61K38/47 , C12N7/00 , A61K38/17 , A61K38/44 , C12N9/10 , A61K9/00 , A61K48/00
CPC classification number: C12N15/86 , A61K38/1719 , A61K38/44 , A61K38/47 , A61P21/00 , C07K14/005 , C12N7/00 , C12N9/1051 , C12N15/8645 , C12Y116/03001 , C12Y204/01001 , C12Y302/0102 , C12Y302/01022 , A61K9/0019 , A61K48/00 , C12N2750/14121 , C12N2750/14122 , C12N2750/14133 , C12N2750/14143 , C12N2750/14145 , C12N2750/14171
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：US11807868B2
公开(公告)日：2023-11-07
申请号：US17938641
申请日：2022-10-06
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： Melissa Kotterman , Peter Francis , Melissa Calton , Johnny Gonzales , Roxanne Croze , Christopher Schmitt
CPC classification number: C12N15/86 , A61K9/0078 , A61K9/12 , A61P11/00 , C07K14/005 , C07K14/4712 , C12N2750/14121 , C12N2750/14122 , C12N2750/14141 , C12N2750/14142 , C12N2750/14143 , C12N2750/14151
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：US11364308B2
公开(公告)日：2022-06-21
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 , A61P27/02 , A61K9/00 , C07K14/005
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 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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Patent Agency Ranking
公开(公告)号：US20230211013A1
公开(公告)日：2023-07-06
申请号：US18069347
申请日：2022-12-21
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. Kirn , Melissa Kotterman , David Schaffer
IPC: C12N15/113 , C12N15/86 , A61P27/02
CPC classification number: C12N15/113 , C12N15/86 , A61P27/02 , C12N2750/14143
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 cells of the retina for the treatment of retinal disorders and diseases.
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公开(公告)号：US20230190964A1
公开(公告)日：2023-06-22
申请号：US18045650
申请日：2022-10-11
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. Kirn , Melissa Kotterman , Peter Francis , David Schaffer , Paul Szymanski , Kevin Whittlesey
CPC classification number: A61K48/0066 , A61P3/00 , A61K48/0083 , A61K48/0075
Abstract: The present disclosure provides codon optimized nucleotide sequences encoding human 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：US11576983B2
公开(公告)日：2023-02-14
申请号：US17746443
申请日：2022-05-17
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. Kirn , Melissa Kotterman , David Schaffer
IPC: C12N15/864 , 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 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：US11499166B2
公开(公告)日：2022-11-15
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：US12221616B2
公开(公告)日：2025-02-11
申请号：US17400041
申请日：2021-08-11
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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