Patents Assigned to 4D MOLECULAR THERAPEUTICS INC.
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Publication number: 20220251145Abstract: 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.Type: ApplicationFiled: April 12, 2022Publication date: August 11, 2022Applicant: 4D MOLECULAR THERAPEUTICS INC.Inventors: David H. KIRN, Melissa KOTTERMAN, David SCHAFFER
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Patent number: 11364308Abstract: 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.Type: GrantFiled: May 12, 2017Date of Patent: June 21, 2022Assignee: 4D Molecular Therapeutics Inc.Inventors: David H. Kirn, Melissa Kotterman, David Schaffer
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Patent number: 11357870Abstract: 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: GrantFiled: August 31, 2021Date of Patent: June 14, 2022Assignee: 4D Molecular Therapeutics Inc.Inventors: David H. Kirn, Melissa A. Kotterman, Peter Francis
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Patent number: 11345930Abstract: 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: GrantFiled: August 30, 2021Date of Patent: May 31, 2022Assignee: 4D MOLECULAR THERAPEUTICS INC.Inventors: David H. Kirn, Melissa A. Kotterman, David Schaffer, Peter Francis
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Publication number: 20220064673Abstract: 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: ApplicationFiled: August 30, 2021Publication date: March 3, 2022Applicant: 4D MOLECULAR THERAPEUTICS INC.Inventors: David H. KIRN, Melissa A. KOTTERMAN, David SCHAFFER, Peter FRANCIS
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Publication number: 20220062438Abstract: 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: ApplicationFiled: August 31, 2021Publication date: March 3, 2022Applicant: 4D MOLECULAR THERAPEUTICS INC.Inventors: David H. KIRN, Melissa A. KOTTERMAN, Peter FRANCIS
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Publication number: 20210395772Abstract: 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: ApplicationFiled: April 26, 2021Publication date: December 23, 2021Applicant: 4D MOLECULAR THERAPEUTICS INC.Inventors: Melissa KOTTERMAN, Peter FRANCIS, Melissa CALTON, Johnny GONZALES, Roxanne CROZE, Christopher SCHMITT
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Patent number: 11198858Abstract: 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: GrantFiled: April 26, 2021Date of Patent: December 14, 2021Assignee: 4D Molecular Therapeutics Inc.Inventors: David H. Kirn, Melissa Kotterman, Peter Francis, David Schaffer, Paul Szymanski, Kevin Whittlesey
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Publication number: 20210380643Abstract: 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.Type: ApplicationFiled: August 11, 2021Publication date: December 9, 2021Applicant: 4D MOLECULAR THERAPEUTICS INC.Inventors: David H. KIRN, Melissa KOTTERMAN, David SCHAFFER
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Patent number: 11179477Abstract: 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.Type: GrantFiled: November 18, 2020Date of Patent: November 23, 2021Assignee: 4D MOLECULAR THERAPEUTICS INC.Inventors: David H. Kirn, Melissa Kotterman, David Schaffer
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Patent number: 11167041Abstract: 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.Type: GrantFiled: November 18, 2020Date of Patent: November 9, 2021Assignee: 4D MOLECULAR THERAPEUTICS INC.Inventors: David H. Kirn, Melissa Kotterman, David Schaffer
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Publication number: 20210332341Abstract: 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: ApplicationFiled: April 26, 2021Publication date: October 28, 2021Applicant: 4D MOLECULAR THERAPEUTICS INC.Inventors: David H. KIRN, Melissa KOTTERMAN, Peter FRANCIS, David SCHAFFER, Paul SZYMANSKI, Kevin WHITTLESEY
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Patent number: 11118192Abstract: 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.Type: GrantFiled: September 19, 2018Date of Patent: September 14, 2021Assignee: 4D MOLECULAR THERAPEUTICS INC.Inventors: David H. Kirn, Melissa Kotterman, David Schaffer
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Publication number: 20210177990Abstract: 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.Type: ApplicationFiled: January 29, 2021Publication date: June 17, 2021Applicant: 4D MOLECULAR THERAPEUTICS INC.Inventors: David H. KIRN, Melissa KOTTERMAN, David SCHAFFER
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Publication number: 20210162071Abstract: 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.Type: ApplicationFiled: January 29, 2021Publication date: June 3, 2021Applicant: 4D MOLECULAR THERAPEUTICS INC.Inventors: David H. KIRN, Melissa KOTTERMAN, David SCHAFFER
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Publication number: 20210069349Abstract: 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.Type: ApplicationFiled: November 18, 2020Publication date: March 11, 2021Applicant: 4D MOLECULAR THERAPEUTICS INC.Inventors: David H. KIRN, Melissa KOTTERMAN, David SCHAFFER
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Publication number: 20210069348Abstract: 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.Type: ApplicationFiled: November 18, 2020Publication date: March 11, 2021Applicant: 4D MOLECULAR THERAPEUTICS INC.Inventors: David H. KIRN, Melissa KOTTERMAN, David SCHAFFER
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Publication number: 20200282077Abstract: 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.Type: ApplicationFiled: November 26, 2018Publication date: September 10, 2020Applicant: 4D MOLECULAR THERAPEUTICS INC.Inventors: David H. KIRN, Melissa KOTTERMAN, David SCHAFFER, Paul SZYMANSKI, Peter FRANCIS
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Publication number: 20200248205Abstract: 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.Type: ApplicationFiled: September 19, 2018Publication date: August 6, 2020Applicant: 4D MOLECULAR THERAPEUTICS INC.Inventors: David H. KIRN, Melissa KOTTERMAN, David SCHAFFER
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Publication number: 20190255192Abstract: 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 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.Type: ApplicationFiled: May 12, 2017Publication date: August 22, 2019Applicant: 4D MOLECULAR THERAPEUTICS INC.Inventors: David H. KIRN, Melissa KOTTERMAN, David SCHAFFER