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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：US11802278B2
公开(公告)日：2023-10-31
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: C12N9/2465 , A61K9/0019 , A61K48/0066 , A61K48/0075 , A61K48/0083 , A61P3/00 , C12N15/86 , C12Y302/01022 , A61K48/00 , C12N2840/00
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：US11198858B2
公开(公告)日：2021-12-14
申请号：US17240948
申请日：2021-04-26
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor： David H. Kirn , Melissa Kotterman , Peter Francis , David Schaffer , Paul Szymanski , Kevin Whittlesey
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：US11766489B2
公开(公告)日：2023-09-26
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: C07K14/005 , C07K16/22 , A61K48/00 , A61K9/00 , C12N7/00
CPC classification number: A61K48/005 , A61K9/0048 , A61K48/0008 , A61K48/0075 , C07K14/005 , C07K16/22 , C12N7/00 , 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 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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