175 patents
Utility
Novel Zinc Finger Protein Transcription Factors for Repressing Alpha-synuclein Expression
18 Jan 24
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.
Asa Hatami, Bryan Zeitler, Lei Zhang, David Emanuel Paschon
Filed: 1 Oct 21
Utility
Assays for Fabry Disease Treatment
4 Jan 24
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-α-Galactosidase A (GLA) neutralizing antibody in a biological sample of the subject, as measured by an anti-GLA neutralizing antibody assay.
Joshua Nelson HOLTER
Filed: 30 Sep 21
Utility
Method for the treatment of mucopolysaccharidosis type I
2 Jan 24
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.
Dale Ando, Cheryl Wong Po Foo, Sagar A. Vaidya, Shelley Q. Wang
Filed: 7 Aug 19
Utility
Methods and compositions for regulation of transgene expression
2 Jan 24
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.
Edward J. Rebar
Filed: 3 Mar 21
Utility
Methods and Compositions for Treating Huntington's Disease
28 Dec 23
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.
Jeffrey C. Miller, Edward J. Rebar, H. Steve Zhang
Filed: 29 Jun 23
Utility
Methods and Compositions for Treatment of a Genetic Condition
28 Dec 23
Methods and compositions for a genetic disease are provided.
Gregory J. Cost, Dmitry Guschin, Philip D. Gregory, Michael C. Holmes, Jeffrey C. Miller, David Paschon, Edward J. Rebar, Andreas Reik, Fyodor Urnov, Lei Zhang
Filed: 7 Nov 22
Utility
Regulation of gene expression using engineered nucleases
19 Dec 23
The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic cell.
Jeffrey C. Miller, Edward J. Rebar
Filed: 9 Jan 20
Utility
Generation of CD4+ Effector and Regulatory T Cells from Human Pluripotent Stem Cells
7 Dec 23
Provided herein are improved methods and compositions for generating CD4-positive effector T cells and regulatory T cells and methods of use thereof.
Helen Fong, Anthony Conway, Matthew C. Mendel
Filed: 28 Oct 21
Utility
Engineered target specific base editors
5 Dec 23
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.
Friedrich Fauser, Jeffrey C. Miller, Edward Rebar
Filed: 20 Aug 19
Utility
Methods for the Treatment of Beta-thalassemia
30 Nov 23
Described herein are methods and compositions for treating a beta-thalassemia.
Weston P. Miller, IV, John Tomaro, Sagar A. Vaidya, Mark Walters
Filed: 20 Mar 23
Utility
Methods and Compositions for Modification of a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Gene
30 Nov 23
Nucleases and methods of using these nucleases for alteration of a CFTR gene and generation of cells and animal models.
Anthony Conway
Filed: 12 Apr 23
Utility
Methods of Detecting Anti-aav Antibodies
30 Nov 23
The disclosure relates to total antibody assays, including screening assays and confirmatory assays, for the detection of anti-AAV (e.g. anti-AAV6) antibodies in a subject as it relates to pre- and post-treatment total antibody levels.
Jing Hu, Yanmei Lu
Filed: 1 Oct 21
Utility
Engineered target specific nucleases
28 Nov 23
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.
Jeffrey C. Miller, Edward J. Rebar
Filed: 14 Jul 20
Utility
Targeted Disruption of T Cell Receptor Genes Using Engineered Zinc Finger Protein Nucleases
2 Nov 23
Disclosed herein are methods and compositions for inactivating TCR 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.
Maria Chiara Bonini, Pietro Genovese, Philip D. Gregory, Michael C. Holmes, Luigi Naldini, David Paschon, Elena Provasi, Lei Zhang
Filed: 12 Sep 22
Utility
Methods and compositions for engineering immunity
24 Oct 23
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.
Michael C. Holmes, Brigit E. Riley
Filed: 30 Mar 20
Utility
Methods and Compositions for the Treatment of Neurologic Disease
19 Oct 23
Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic IDUA or IDS protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of MPS I or MPS II disease.
Russell DeKelver, R. Scott McIvor, Li Ou, Thomas Wechsler, Chester B. Whitley, Kanut Laoharawee
Filed: 24 Oct 22
Utility
Methods and Compositions for Expressing Phenylalanine Hydroxylase
12 Oct 23
The present disclosure provides expression constructs comprising a phenylalanine hydroxylase (PAH) transgene and methods of using the constructs for treating phenylketonuria (PKU).
Gustavo de Alencastro
Filed: 11 Jun 21
Utility
Targeted Disruption of T Cell And/or Hla Receptors
21 Sep 23
Disclosed herein are methods and compositions for inactivating TCR 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.
Anthony Conway, Sumiti Jain, Gary K. Lee, David Paschon, Edward J. Rebar, Lei Zhang
Filed: 21 Oct 22
Utility
Tau Modulators and Methods and Compositions for Delivery Thereof
31 Aug 23
The present disclosure is in the field of diagnostics and therapeutics for Alzheimer's Disease.
Annemarie Ledeboer, Bryan Zeitler, H. Steve Zhang, Sarah DeVos, Bradley T. Hyman, Susanne Wegmann
Filed: 13 Oct 22
Utility
Genetic Modification of Cytokine Inducible SH2-CONTAINING Protein (Cish) Gene
31 Aug 23
The present disclosure is in the field of genome engineering, particularly targeted genetic modification of a CISH gene.
Anthony Conway, Gary K. Lee
Filed: 22 Nov 22