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Tissue-Specific Chimeric Constructs for Treating Duchenne Muscular Dystrophy
Delivers Micro-dystrophin and Utrophin Chimeric Constructs to the Skeletal and Cardiac Muscles, Optimizing the Gene Therapy to Each Tissue These micro-dystrophin and utrophin chimeric constructs optimize the treatment of Duchenne Muscular Dystrophy (DMD). DMD is a rare, X-chromosome-linked disorder characterized by progressive muscle degeneration and...
Published: 9/4/2024   |   Inventor(s): Hugh Sweeney
Keywords(s):  
Category(s): Technology Classifications > Human Health Care > Therapeutics, Technology Classifications > Human Health Care > Gene Therapy
Recombinant AAV9 Vectors with Modified Capsids for Escaping Neutralizing Human Antibodies
Amino Acid Substitutions on the Capsid Surface to Evade Neutralizing Antibodies and Improve Gene Therapy Efficacy and Safety Adeno-associated virus (AAV)-based vectors are the leading platform for the delivery of gene therapy in vivo, but have a limitation in that they need to avoid neutralizing and potentially dangerous immune response to the AAV...
Published: 9/4/2024   |   Inventor(s): Robert McKenna, Mario Mietzsch, Jane Hsi, Austin Nelson
Keywords(s):  
Category(s): Technology Classifications > Human Health Care > Gene Therapy, Technology Classifications > Human Health Care > Therapeutics
Exosome Gene Therapy for Treating Inner Ear Disease
Extracellular Vesicles Directly Target Inner Ear Cells to Correct Malfunctioning Genes In Vivo This in vivo gene therapy strategy combines selective gene editors with the cell targeting capability using extracellular vesicles for safe and targeted delivery., Hearing loss affects at least 1 in 500 newborns, and over 50% are hereditary in nature. One...
Published: 9/3/2024   |   Inventor(s): Mei He, Xiaoshu Pan
Keywords(s):  
Category(s): Technology Classifications > Human Health Care > Gene Therapy, Technology Classifications > Human Health Care > Drug Delivery
Modified Arrestin-1 Variants for Enhancing Photoreceptor Survival in Retinal Disease
Binds to Enolase-1 without Inhibiting its Catalytic Activity, Slowing Retinal Degradation and Alleviating Vision Loss These arrestin-1 variants slow retinal degradation by reducing the inhibitory effects of enolase-1 activity, enhancing photoreceptor survival in retinal disease. Inherited retinal diseases (IRDs) affect over two million people worldwide,...
Published: 1/30/2023   |   Inventor(s): Wesley Smith, Astra Dinculescu, Vince Chiodo
Keywords(s):  
Category(s): Technology Classifications > Human Health Care > Gene Therapy
Variant AAV Particles for Improved Gene Therapy
Capsid Proteins Optimize Delivery of Therapeutic Genes to Targeted Tissues These variant recombinant adeno-associated virus (rAAV) particles feature capsid surface proteins that focus delivery of therapeutic genes toward specific tissues, decreasing uptake in non-target tissues. Demand for the AAV gene therapy delivery platform is rapidly increasing...
Published: 8/9/2022   |   Inventor(s): Hugh Sweeney
Keywords(s):  
Category(s): Technology Classifications > Human Health Care > Gene Therapy, Technology Classifications > Human Health Care > Therapeutics
AAV Gene Therapy Capsid Variants That Evade Neutralizing Antibodies
Enable Patients With Anti-Capsid Antibodies for AAV8 or AAVrh.10 to Participate in Clinical Trials to Undergo Gene Therapy with AAV These adeno-associate vector capsid variants offer antibody-escaping abilities that will enable patients with antibodies against an AAV serotype to receive therapy or participate in clinical trials anyway. Due to the high...
Published: 9/15/2021   |   Inventor(s): Mavis Agbandje-McKenna, Mario Mietzsch
Keywords(s):  
Category(s): Technology Classifications > Human Health Care > Gene Therapy, Technology Classifications > Human Health Care > Therapeutics
Immunotherapy That Converts Glioblastoma Cells Into Immune Cells to Attack Cancerous Tumors
Converts Cancer Cells to Immune Cells that Recruit Body’s Own Immune System to Target Brain Tumors This immunotherapy directly reprograms cancer cells into immune cells, specifically dendritic cells, making the reprogrammed cancer cells a therapy for the rest of the tumor. Glioblastoma (GBM) is the most common and lethal brain cancer in adults....
Published: 6/27/2021   |   Inventor(s): David Tran, Son Le
Keywords(s):  
Category(s): Technology Classifications > Human Health Care > Therapeutics, Technology Classifications > Human Health Care > Gene Therapy
rAAV Gene Therapy to Treat Hyperthyroidism in One Administration
Blocks Hormone Receptors to Stop Overproduction of Thyroid Stimulating Hormone This vector-based gene therapy encodes antibodies that block the stimulation of thyroid-stimulating hormone receptors (TSHR), which when activated can lead to hyperthyroidism. Graves’ disease is the most common form of hyperthyroidism, and is caused by stimulation...
Published: 6/27/2021   |   Inventor(s): Scott Rivkees, Arun Srivastava
Keywords(s):  
Category(s): Technology Classifications > Human Health Care > Gene Therapy, Technology Classifications > Human Health Care > Therapeutics
A Drug-Regulated, Broad-Spectrum Gene Therapy for Retinal Degeneration
Encodes a Protective Protein Using AAV Vector and Allows Control of Protein Expression This gene therapy delivers a transgene encoding the Retinal Protective Factor-2 (RPF-2) protein that is easy to regulate and provides broad-spectrum protection against retinal degeneration. Retinal degenerations are diseases characterized by the irreversible loss...
Published: 6/27/2021   |   Inventor(s): John Ash, Clayton Santiago
Keywords(s):  
Category(s): Technology Classifications > Human Health Care > Gene Therapy
Embryonated Avian Eggs as Host Vehicles to Produce Recombinant AAV
Expands AAV Manufacturing Capacity to Support the Growing Clinical Applications of Gene TherapyThis large-scale production of rAAV employs embryonated avian eggs to stably package and produce adeno associated viral (AAV) vectors. A powerful research and clinical tool, AAVs deliver genetic material to specific cells and provide in vivo long-term gene...
Published: 6/27/2021   |   Inventor(s): Pedro Cruz, Todd Golde, Carolina Diaz, Xuefei Liu, Daniel Ryu
Keywords(s):  
Category(s): Technology Classifications > Human Health Care > Gene Therapy
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