Search Results - therapeutics+%3e+gene+therapy+and+editing

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Engineered Gamma Delta T-Cells and Methods of Making and Using Thereof (UCLA Case No. 2020-913)
UCLA researchers in the Department of Molecular Immunology & Molecular Genetics have developed gamma delta (gd) T cell receptor gene-modified immune cells derived from stem and progenitor cells that enable scalable, off-the-shelf allogeneic cell therapies with broad therapeutic potential. BACKGROUND: Cell-based immunotherapies have demonstrated...
Published: 3/4/2026   |   Updated: 3/4/2026   |   Inventor(s): Lili Yang, Derek Lee, Tyler Halladay
Keywords(s):  
Category(s): Diagnostic Markers > Immunology, Diagnostic Markers > Cancer, Life Science Research Tools > Cell Lines, Therapeutics > Oncology, Therapeutics > CNS and Neurology, Therapeutics > Hematology, Therapeutics > Autoimmune, Therapeutics > Gene Therapy And Editing, Therapeutics > Stem Cells And Regenerative Medicine, Therapeutics > Infectious Diseases
Regulated Gene Expression During Differentiation of Mature T Cells (UCLA Case No. 2022-147)
UCLA researchers from the Department of Pathology & Laboratory Medicine have developed a process to genetically modify stem cells to express transgenes in a regulated, stage-specific manner, avoiding the negative consequences of transgene expression during T cell development. Contact UCLA Technology Development Group for more information.
Published: 1/27/2026   |   Updated: 1/27/2026   |   Inventor(s): Gay Crooks, Donald Kohn
Keywords(s):  
Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Stem Cells And Regenerative Medicine, Therapeutics > Oncology, Therapeutics > Immunology And Immunotherapy, Therapeutics > Inflammation And Inflammatory Diseases
In Cellulo Synthesis of mRNA-Specific Stoichiometrically-Precise Virus-Like Particles (UCLA Case No. 2024-056)
UCLA researchers from the Department of Chemistry and Biochemistry have developed a novel virus-like particle (VLP) platform for the encapsulation of mRNA for efficient therapeutic delivery. BACKGROUND: Messenger RNA (mRNA) technologies have emerged as powerful therapeutics in vaccines and gene therapies. While traditional methods of mRNA therapeutics...
Published: 12/8/2025   |   Updated: 12/8/2025   |   Inventor(s): William Gelbart
Keywords(s):  
Category(s): Therapeutics > Vaccines, Platforms > Drug Delivery, Therapeutics > Gene Therapy And Editing, Materials > Nanotechnology
Nanoparticle & dsDNA Toolkit for Decreased Toxicity and Gene Editing of Epithelial Stem Cells via Epithelial Disruption (UCLA Case No. 2023-205/209) -- for Cystic Fibrosis and other genetic diseases
UCLA researchers in the Department of Pediatrics, Hematology/ Oncology have constructed a nanoparticle toolkit for increased integration and reduced toxicity for gene therapies that treat debilitating diseases such as Cystic Fibrosis and other critical genetic diseases. BACKGROUND: Gene therapies are the forefront of therapeutic advancements as a one-off...
Published: 12/1/2025   |   Updated: 12/1/2025   |   Inventor(s): Steven Jonas, Brigitte Gomperts, Donald Kohn
Keywords(s):  
Category(s): Therapeutics > Gene Therapy And Editing, Platforms > Drug Delivery
Bioinformatically-Identified Control Elements for Regulated Expression from a Lentiviral Vector to Treat X-Linked Lymphoproliferative Disease (UCLA Case No. 2022-065)
UCLA researchers from the Department of Microbiology, Immunology, and Molecular Genetics and Pediatrics have used a bioinformatics-guided approach to design regulated lentiviral vectors for the treatment of X-linked lymphoproliferative disease type 1. BACKGROUND: X-linked lymphoproliferative disease type 1 (XLP1) is a rare primary immunodeficiency...
Published: 10/17/2025   |   Updated: 10/17/2025   |   Inventor(s): Donald Kohn, Paul Ayoub
Keywords(s):  
Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Stem Cells And Regenerative Medicine
Lipid Nanoparticle-Mediated Delivery of Gene Editing Reagents for Targeted Disruption of Oncogenic Drivers in C-Myc Overexpressing Malignancies (UCLA Case No. 2025-9A9)
Contact UCLA Technology Development Group to learn more.
Published: 9/19/2025   |   Updated: 9/19/2025   |   Inventor(s): Steven Jonas, Tanya Stoyanova
Keywords(s):  
Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Oncology
Vectors Combining Anti-Sickling Beta-AS3-Globin with Anti BCL11A ShRNAmir to Treat Beta-Hemoglobinopathies (UCLA Case No. 2021-190)
UCLA researchers from the Department of Microbiology, Immunology and Molecular Genetics and Pediatrics have developed a potent bi-functional lentiviral expression vector to treat sickle cell disease and beta-thalassemia. BACKGROUND: Beta-hemoglobinopathies, such as sickle cell disease and beta-thalassemia, are genetic disorders characterized by dysfunctional...
Published: 9/11/2025   |   Updated: 9/11/2025   |   Inventor(s): Donald Kohn
Keywords(s):  
Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Hematology, Therapeutics > Stem Cells And Regenerative Medicine
Novel AAV Serotypes Derived From a Library Screen (UCLA Case No. 2020-789)
UCLA researchers in the Department of Neurology have produced novel AAV serotypes for a wide range of tissue and cell specific gene delivery. BACKGROUND: Gene therapies are the forefront of therapeutic advancements for debilitating diseases. However, delivery and insertion of the therapeutic transgene are major hurdles that must be solved to effectively...
Published: 11/12/2025   |   Updated: 7/8/2025   |   Inventor(s): Melissa Spencer
Keywords(s):  
Category(s): Therapeutics > Gene Therapy And Editing
UCLA CRISPR Licensing Program (Multiple Technology Case Numbers)
UCLA CRISPR Licensing Program A major challenge in CRISPR-based gene editing is efficient delivery of the editing system to target cells. UCLA has filed patents on plant viral delivery vectors expressing highly-miniaturized CRISPR systems that may overcome these challenges. The technology is based on recent work from the Jacobsen (UCLA) and Doudna...
Published: 9/12/2025   |   Updated: 6/12/2025   |   Inventor(s): Steven Jacobsen, Jennifer Doudna
Keywords(s):  
Category(s): Therapeutics > Gene Therapy And Editing
Gene Modifications to Enhance the Function of PSC-Derived T Cells and NK Cells (UCLA Case No. 2023-129)
UCLA researchers in the Department of Medicine have generated novel pluripotent stem cell derived T cells that exhibit improved survival, expansion, and anti-tumor efficacy for use in cell therapy treatments. BACKGROUND: The Food and Drug Administration has approved several chimeric antigen receptor-T (CAR-T) cell therapies since 2017 to combat lymphoma...
Published: 7/31/2025   |   Updated: 4/22/2025   |   Inventor(s): Christopher Seet
Keywords(s): CAR-T cell therapy, Immunotherapy, pluripotent stem cell (PSC), T-cell engineering
Category(s): Therapeutics > Immunology And Immunotherapy, Therapeutics > Gene Therapy And Editing
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