Abstract: Disclosed herein are compositions for linking DNA binding domains and cleavage domains (or cleavage half-domains) to form non-naturally occurring nucleases with alternative configurations. Also described are methods of making and using compositions comprising these linkers.
Type:
Grant
Filed:
June 19, 2020
Date of Patent:
March 5, 2024
Assignee:
Sangamo Therapeutics, Inc.
Inventors:
Jeffrey C. Miller, David Paschon, Edward J. Rebar
Abstract: The present invention provides compositions comprising a recombinant AAV and one or more pharmaceutically acceptable excipients. The compositions have improved stability and shelf life as compared to other AAV compositions.
Type:
Application
Filed:
December 17, 2021
Publication date:
February 29, 2024
Applicants:
Sangamo Therapeutics, Inc., Pfizer Inc.
Inventors:
Jessica Eileen Conner, Lindsey Anne Crawford, Robert Damitz, Brendan Michael Davis, Cody Michael Hodge, Michael Leland Kimmel II, Tihami Qureshi Willard, Phillip Ramsey, Daniel Joseph Thorne, Anthony Lee Young
Abstract: Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of a lysosomal storage disease.
Abstract: Provided herein are base editor systems comprising fusion proteins that comprise zinc finger protein and cytidine deaminase domains, as well as methods of using the base editor systems. The systems can be used to specifically alter a single base pair in a target DNA sequence.
Type:
Application
Filed:
September 24, 2021
Publication date:
February 8, 2024
Applicant:
Sangamo Therapeutics, Inc.
Inventors:
Friedrich A. Fauser, Jeffrey C. Miller, Sebastian Arangundy
Abstract: The present disclosure provides zinc finger fusion proteins that inhibit expression of alpha-synuclein in the nervous system, and methods of using the proteins to treat Parkinsons disease, Lewy body dementia, multiple system atrophy, Alzheimers disease, and other neurodegenerative diseases.
Type:
Application
Filed:
October 1, 2021
Publication date:
January 18, 2024
Applicant:
Sangamo Therapeutics, Inc.
Inventors:
Asa Hatami, Bryan Zeitler, Lei Zhang, David Emanuel Paschon
Abstract: The disclosure provides the enzyme and non-enzyme replacement therapies for treating Fabry disease in a human subject, determined based on measuring the presence or absence of an anti-?-Galactosidase A (GLA) neutralizing antibody in a biological sample of the subject, as measured by an anti-GLA neutralizing antibody assay.
Abstract: Described herein are methods and compositions for treating MPS I (Hurler/Hurler-Scheie/Scheie Syndrome) by administering to the subject a composition comprising a iduronidase (IDUA) polynucleotide. In one aspect, provided is a method of reducing, delaying and/or eliminating one or more of the need for additional treatment procedures, the onset, progression and/or severity of symptoms in a subject with MPS I, by administering to the subject a composition comprising a iduronidase (IDUA) polynucleotide.
Type:
Grant
Filed:
August 7, 2019
Date of Patent:
January 2, 2024
Assignee:
Sangamo Therapeutics, Inc.
Inventors:
Dale Ando, Cheryl Wong Po Foo, Sagar A. Vaidya, Shelley Q. Wang
Abstract: Nucleases and methods of using these nucleases for expressing a transgene from a safe harbor locus in a secretory tissue, and clones and animals derived therefrom.
Abstract: Provided herein are improved methods and compositions for generating CD4-positive effector T cells and regulatory T cells and methods of use thereof.
Type:
Application
Filed:
October 28, 2021
Publication date:
December 7, 2023
Applicant:
Sangamo Therapeutics, Inc.
Inventors:
Helen Fong, Anthony Conway, Matthew C. Mendel
Abstract: Described herein are DNA-editing complexes, particularly DNA-editing complexes that specifically alter a single base pair in target DNA sequence as well as methods of making and using these DNA-editing complexes.
Type:
Grant
Filed:
August 20, 2019
Date of Patent:
December 5, 2023
Assignee:
Sangamo Therapeutics, Inc.
Inventors:
Friedrich Fauser, Jeffrey C. Miller, Edward Rebar
Abstract: Described herein are engineered nucleases comprising mutations in the cleavage domain (e.g., FokI or homologue thereof) and/or DNA binding domain (zinc finger protein, TALE, single guide RNA) such that on-target specificity is increased.
Abstract: Nucleases and methods of using these nucleases for expressing an antibody from a safe harbor locus in a secretory tissue, and clones and animals derived therefrom.
Abstract: The present disclosure provides expression constructs comprising a phenylalanine hydroxylase (PAH) transgene and methods of using the constructs for treating phenylketonuria (PKU).
Abstract: Disclosed herein are compositions comprising non-naturally occurring zinc finger domains, fusion proteins comprising these zinc finger domains, polynucleotides encoding these proteins, cells expressing these proteins and pharmaceutical compositions comprising these proteins or polynucleotides as well as methods of modifying an Htt gene using these compositions for treating or preventing Huntington's Disease.
Type:
Grant
Filed:
November 16, 2020
Date of Patent:
August 15, 2023
Assignee:
Sangamo Therapeutics, Inc.
Inventors:
Jeffrey C. Miller, Edward J. Rebar, H. Steve Zhang
Abstract: The present disclosure provides methods for the separation and quantification of empty and full viral capsids (e.g., AAV capsids) within a viral preparation, such as a viral pharmaceutical composition or drug product.
Abstract: Disclosed herein are engineered cleavage half-domains; fusion polypeptides comprising these engineered cleavage half-domains; polynucleotides encoding the engineered cleavage half-domains and fusion proteins; and cells comprising said polynucleotides and/or fusion proteins. Also described are methods of using these polypeptides and polynucleotides, for example for targeted cleavage of a genomic sequence.
Abstract: Provided herein are nucleic acid constructs, host insect cells, and methods for producing recombinant AAV capsids with high potency at high yield.
Type:
Grant
Filed:
February 14, 2020
Date of Patent:
August 8, 2023
Assignee:
Sangamo Therapeutics, Inc.
Inventors:
Andrea J. Kang, Richard T. Surosky, Alex Michael Ward