38 patents
Utility
Codon Optimized Gla Genes and Uses Thereof
18 Jan 24
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.
David H. KIRN, Melissa KOTTERMAN, Peter FRANCIS, David SCHAFFER, Paul SZYMANSKI, Kevin WHITTLESEY
Filed: 8 Sep 23
Utility
Adeno-associated virus variant capsids and methods of use thereof
12 Dec 23
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.
David H. Kirn, Melissa Kotterman, David Schaffer
Filed: 21 Dec 22
Utility
Adeno-associated variants, formulations and methods for pulmonary delivery
7 Nov 23
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.
Melissa Kotterman, Peter Francis, Melissa Calton, Johnny Gonzales, Roxanne Croze, Christopher Schmitt
Filed: 6 Oct 22
Utility
Codon optimized GLA genes and uses thereof
31 Oct 23
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.
David H. Kirn, Melissa Kotterman, Peter Francis, David Schaffer, Paul Szymanski, Kevin Whittlesey
Filed: 11 Oct 22
Utility
Codon Optimized RPGRORF15 Genes and Uses Thereof
26 Oct 23
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.
David H. KIRN, Melissa A. KOTTERMAN, David SCHAFFER, Peter FRANCIS
Filed: 30 Aug 21
Utility
Treatments for retinal disease
26 Sep 23
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.
David H. Kirn, Melissa Kotterman, David Schaffer
Filed: 21 Dec 22
Utility
Adeno-associated virus variant capsids and use for inhibiting angiogenesis
26 Sep 23
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.
David H. Kirn, Melissa Kotterman, David Schaffer, Paul Szymanski, Peter Francis
Filed: 26 Nov 18
Utility
Adeno-associated Virus Variant Capsids
and Methods of Use Thereof
7 Sep 23
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.
David H. Kirn, Melissa Kotterman, David Schaffer
Filed: 21 Dec 22
Utility
Adeno-associated Variants, Formulations and Methods for Pulmonary Delivery
17 Aug 23
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.
Melissa Kotterman, Peter Francis, Melissa Calton, Johnny Gonzales, Roxanne Croze, Christopher Schmitt
Filed: 6 Oct 22
Utility
Treatments for Retinal Disease
6 Jul 23
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.
David H. Kirn, Melissa Kotterman, David Schaffer
Filed: 21 Dec 22
Utility
Codon Optimized REP1 Genes and Uses Thereof
25 May 23
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.
David H. Kirn, Melissa A. Kotterman, Peter Francis
Filed: 4 Nov 22
Utility
Adeno-associated virus variant capsids and methods of use thereof
28 Mar 23
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.
David H. Kirn, Melissa Kotterman, David Schaffer
Filed: 12 Apr 22
Utility
Treatments for retinal disease
14 Feb 23
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.
David H. Kirn, Melissa Kotterman, David Schaffer
Filed: 17 May 22
Utility
Treatments for Retinal Disease
5 Jan 23
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.
David H. KIRN, Melissa KOTTERMAN, David SCHAFFER
Filed: 17 May 22
Utility
Codon optimized REP1 genes and uses thereof
13 Dec 22
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.
David H. Kirn, Melissa A. Kotterman, Peter Francis
Filed: 9 May 22
Utility
Adeno-associated variants, formulations and methods for pulmonary delivery
15 Nov 22
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.
Melissa Kotterman, Peter Francis, Melissa Calton, Johnny Gonzales, Roxanne Croze, Christopher Schmitt
Filed: 26 Apr 21
Utility
Codon Optimized RPGRORF15 Genes and Uses Thereof
15 Sep 22
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.
David H. Kirn, Melissa A. Kotterman, David Schaffer, Peter Francis
Filed: 26 May 22
Utility
Codon Optimized REP1 Genes and Uses Thereof
25 Aug 22
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.
David H. KIRN, Melissa A. KOTTERMAN, Peter FRANCIS
Filed: 9 May 22
Utility
Adeno-associated virus variant capsids and methods of use thereof
23 Aug 22
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.
David H. Kirn, Melissa Kotterman, David Schaffer
Filed: 29 Jan 21
Utility
Adeno-associated Virus Variant Capsids and Methods of Use Thereof
11 Aug 22
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.
David H. KIRN, Melissa KOTTERMAN, David SCHAFFER
Filed: 12 Apr 22