Sangamo Therapeutics, Inc.

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.

Disclosed herein are methods and compositions for inactivating MHC genes, using zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain in conditions able to preserve cell viability. Polynucleotides encoding ZFNs, vectors comprising polynucleotides encoding ZFNs and cells comprising polynucleotides encoding ZFNs and/or cells comprising ZFNs are also provided.

The present disclosure provides expression constructs comprising a GLA transgene encoding the at least one α-Gal A protein for use in expressing α-Gal A proteins and preventing, inhibiting or treating Fabry disease or one or more symptoms associated with Fabry disease.

The disclosure provides the methods of detecting an anti-lysosomal enzyme antibody in a human subject (e.g., in the presence of a circulating lysosomal enzyme) and the methods of improving an efficacy of an anti-lysosomal enzyme antibody assay (e.g., improving the assay sensitivity and the circulating lysosomal enzyme tolerance), comprising pre-treating the biological sample of the subject with a base at pH of about 11 or greater and measuring the presence of the anti-lysosomal enzyme antibody in the biological sample of the subject.

Disclosed herein are Htt repressors and methods and compositions for use of these Htt repressors.

IL23R overexpression has been described as a common feature of pathogenic inflammatory cells involved in the onset and maintenance of autoimmune diseases and chronic inflammation. The present disclosure relates to a novel antigen binder directed to IL-23R, to compositions that comprise said antigen binder, and to the use of said antigen binder. The present disclosure further relates to fusion proteins comprising said antigen binder, such as, for example, chimeric antigen receptors.

Disclosed herein are methods and compositions for engineering cells to express a CAR where the cells also have inactivated TCR, HPRT, PD1, CISH and/or HLA genes, using engineered nucleases comprising at least one DNA binding domain and a cleavage domain or cleavage half-domain in conditions able to preserve cell viability. Polynucleotides encoding nucleases, vectors comprising polynucleotides encoding nucleases and cells comprising polynucleotides encoding nucleases and/or cells comprising nucleases are also provided.

The present disclosure provides mammalian cells such as regulatory T cells containing a transgene in the FOXP3 genomic locus. Also provided are methods of generating the cells and methods of using the cells to treat patients in need of immunosuppression.

Described herein are constructs used for liver-specific expression of a transgene.

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.