-
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.
-
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.
-
公开(公告)号：US20240358859A1
公开(公告)日：2024-10-31
申请号：US18766963
申请日：2024-07-09
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. Kirn , Melissa A. Kotterman , Peter Francis
CPC classification number: A61K48/0058 , A61P27/02 , C12N9/1085 , C12N15/86 , C12Y205/01059 , C12N2750/14143
Abstract: The present disclosure provides codon optimized nucleotide sequences encoding human REP1, vectors, and host cells comprising codon optimized REP1 sequences, and methods of treating retinal disorders such as choroideremia comprising administering to the subject a codon optimized sequence encoding human REP1.
-
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.
-
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.
-
Patent Agency Ranking
公开(公告)号：US11345930B2
公开(公告)日：2022-05-31
申请号：US17461716
申请日：2021-08-30
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. Kirn , Melissa A. Kotterman , David Schaffer , Peter Francis
Abstract: The present disclosure provides codon optimized RPGRorf15 sequences, vectors, and host cells comprising codon optimized RPGRorf15 sequences, and methods of treating retinal disorders such as XLRP comprising administering to the subject a codon optimized RPGRorf15 sequence.
-
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.
-
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.
-
公开(公告)号：US20230158172A1
公开(公告)日：2023-05-25
申请号：US18052735
申请日：2022-11-04
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. Kirn , Melissa A. Kotterman , Peter Francis
CPC classification number: A61K48/0058 , C12N9/1085 , C12N15/86 , A61P27/02 , C12Y205/01059 , C12N2750/14143
Abstract: The present disclosure provides codon optimized nucleotide sequences encoding human REP1, vectors, and host cells comprising codon optimized REP1 sequences, and methods of treating retinal disorders such as choroideremia comprising administering to the subject a codon optimized sequence encoding human REP1.
-
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.
-
-
-
-
-
-
-
-
-
Search Results
25
records
(took 0.016 seconds)
