Abstract: The present disclosure provides compositions and methods for the treatment of ocular diseases associated with angiogenesis, particularly wet age-related macular degeneration.
Type:
Application
Filed:
October 5, 2023
Publication date:
April 25, 2024
Applicant:
4D MOLECULAR THERAPEUTICS INC.
Inventors:
Christian BURNS, Melissa CALTON, Meredith LEONG, Paul SZYMANSKI
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.
Type:
Application
Filed:
November 4, 2022
Publication date:
May 25, 2023
Applicant:
4D MOLECULAR THERAPEUTICS INC.
Inventors:
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.
Type:
Application
Filed:
May 9, 2022
Publication date:
August 25, 2022
Applicant:
4D MOLECULAR THERAPEUTICS INC.
Inventors:
David H. KIRN, Melissa A. KOTTERMAN, 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.
Type:
Application
Filed:
August 30, 2021
Publication date:
March 3, 2022
Applicant:
4D MOLECULAR THERAPEUTICS INC.
Inventors:
David H. KIRN, Melissa A. KOTTERMAN, David SCHAFFER, 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.
Type:
Application
Filed:
August 31, 2021
Publication date:
March 3, 2022
Applicant:
4D MOLECULAR THERAPEUTICS INC.
Inventors:
David H. KIRN, Melissa A. KOTTERMAN, 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.
Type:
Application
Filed:
August 30, 2021
Publication date:
October 26, 2023
Applicant:
4D MOLECULAR THERAPEUTICS INC.
Inventors:
David H. KIRN, Melissa A. KOTTERMAN, David SCHAFFER, Peter FRANCIS
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.
Type:
Application
Filed:
April 26, 2021
Publication date:
December 23, 2021
Applicant:
4D MOLECULAR THERAPEUTICS INC.
Inventors:
Melissa KOTTERMAN, Peter FRANCIS, Melissa CALTON, Johnny GONZALES, Roxanne CROZE, Christopher SCHMITT
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.
Type:
Grant
Filed:
August 31, 2021
Date of Patent:
June 14, 2022
Assignee:
4D Molecular Therapeutics Inc.
Inventors:
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.
Type:
Grant
Filed:
May 9, 2022
Date of Patent:
December 13, 2022
Assignee:
4D Molecular Therapeutics Inc.
Inventors:
David H. Kirn, Melissa A. Kotterman, 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.
Type:
Grant
Filed:
August 30, 2021
Date of Patent:
May 31, 2022
Assignee:
4D MOLECULAR THERAPEUTICS INC.
Inventors:
David H. Kirn, Melissa A. Kotterman, David Schaffer, Peter Francis
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.
Type:
Application
Filed:
April 26, 2021
Publication date:
October 28, 2021
Applicant:
4D MOLECULAR THERAPEUTICS INC.
Inventors:
David H. KIRN, Melissa KOTTERMAN, Peter FRANCIS, David SCHAFFER, Paul SZYMANSKI, Kevin WHITTLESEY
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.
Type:
Application
Filed:
September 15, 2023
Publication date:
February 29, 2024
Applicant:
4D MOLECULAR THERAPEUTICS INC.
Inventors:
Melissa Kotterman, Peter Francis, Melissa Calton, Johnny Gonzales, Roxanne Croze, Christopher Schmitt
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.
Type:
Application
Filed:
October 6, 2022
Publication date:
August 17, 2023
Applicant:
4D Molecular Therapeutics Inc.
Inventors:
Melissa Kotterman, Peter Francis, Melissa Calton, Johnny Gonzales, Roxanne Croze, Christopher Schmitt
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.
Type:
Application
Filed:
October 11, 2022
Publication date:
June 22, 2023
Applicant:
4D MOLECULAR THERAPEUTICS INC.
Inventors:
David H. Kirn, Melissa Kotterman, Peter Francis, David Schaffer, Paul Szymanski, Kevin Whittlesey
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.
Type:
Application
Filed:
September 8, 2023
Publication date:
January 18, 2024
Applicant:
4D MOLECULAR THERAPEUTICS INC.
Inventors:
David H. KIRN, Melissa KOTTERMAN, Peter FRANCIS, David SCHAFFER, Paul SZYMANSKI, Kevin WHITTLESEY
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.
Type:
Grant
Filed:
April 26, 2021
Date of Patent:
November 15, 2022
Assignee:
4D Molecular Therapeutics Inc.
Inventors:
Melissa Kotterman, Peter Francis, Melissa Calton, Johnny Gonzales, Roxanne Croze, Christopher Schmitt
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.
Type:
Grant
Filed:
April 26, 2021
Date of Patent:
December 14, 2021
Assignee:
4D Molecular Therapeutics Inc.
Inventors:
David H. Kirn, Melissa Kotterman, Peter Francis, David Schaffer, Paul Szymanski, Kevin Whittlesey
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.
Type:
Grant
Filed:
October 6, 2022
Date of Patent:
November 7, 2023
Assignee:
4D Molecular Therapeutics Inc.
Inventors:
Melissa Kotterman, Peter Francis, Melissa Calton, Johnny Gonzales, Roxanne Croze, Christopher Schmitt
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.
Type:
Application
Filed:
May 17, 2022
Publication date:
January 5, 2023
Applicant:
4D MOLECULAR THERAPEUTICS INC.
Inventors:
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 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.
Type:
Application
Filed:
December 21, 2022
Publication date:
July 6, 2023
Applicant:
4D MOLECULAR THERAPEUTICS INC.
Inventors:
David H. Kirn, Melissa Kotterman, David Schaffer