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公开(公告)号:US20240067987A1
公开(公告)日:2024-02-29
申请号:US18468142
申请日:2023-09-15
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 A.AV 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: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:US11357870B2
公开(公告)日:2022-06-14
申请号:US17463262
申请日:2021-08-31
Applicant: 4D MOLECULAR THERAPEUTICS INC.
Inventor: David H. Kirn , Melissa A. Kotterman , Peter Francis
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.
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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: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.
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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: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.
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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: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.
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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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