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Capsid Mutation System for Producing Recombinant AAV2 Vectors With Lower Affinity to Herapin Sulfate
Produces Recombinant AAV2 vectors with Higher Target SpecificityThis system for generating AAV2, or recombinant Adeno Associated Virus stereotype 2, lowers the presence of herapin sulfate receptor sites by mutating viral capsid proteins. Recent developments in gene therapy have shown great potential of recombinant AAV2 as a vector, due to its low immunogenicity...
Published: 6/27/2021
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Inventor(s): Shaun Opie, Kenneth Warrington, Nicholas Muzyczka
Keywords(s): Gene Therapy, Genetic Therapy
Category(s): Technology Classifications > Human Health Care > Gene Therapy, Technology Classifications > Human Health Care > Others
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Improved Large Scale Production of Adeno-Associated Viruses
Enables a More Efficient and More Potent Production of AAV VectorsThis production of highly concentrated and potent Adeno-Associated Virus (AAV) vectors has significant advantage over available protocol and allows for large-scale manufacturing of the virus. More than 6 percent of all gene therapy clinical trials worldwide use AAV vectors due to their...
Published: 6/27/2021
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Inventor(s): Barry Byrne, Nathalie Clement, Laura Adamson
Keywords(s): AAV, Gene Therapy, HEK293, HSV
Category(s): Technology Classifications > Human Health Care > Gene Therapy
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Purification of AAV Serotypes Using Off-the-Shelf Reagents
Purification Protocol Is Easily Adaptable to Large-Scale Manufacturing of Versatile, Successful Gene Therapy Delivery VehiclesThis purification of recombinant adeno-associated virus serotype three (rAAV3), eight (rAAV8) and nine (rAAV9) uses inexpensive, efficient reagents and is easily adaptable to large-scale manufacturing. An AAV is a small nonenveloped...
Published: 6/27/2021
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Inventor(s): Barry Byrne, Mark Potter
Keywords(s): Gene Therapy, Purification, rAAV, Serotype nine
Category(s): Technology Classifications > Human Health Care > Gene Therapy, Technology Classifications > Research Tools > Therapeutics
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rAAV Vector Gene Therapy for Medullary Thyroid Carcinoma
Targets Cancerous Cells and Uses Tissue-Specific Promoters to Promote Gene ExpressionThis gene therapy utilizes rAAV vectors to target metastatic medullary thyroid carcinoma cells, providing a treatment for the highly aggressive form of thyroid cancer. With available treatments, medullary thyroid carcinoma is incurable once metastasized; for example,...
Published: 6/27/2021
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Inventor(s): Arun Srivastava, Jacqueline Hobbs, Scott Rivkees, Laura Adamson
Keywords(s): Adeno-Associated Virus, calcitonin, Gene Therapy, medullary thyroid carcinoma
Category(s): Technology Classifications > Human Health Care > Gene Therapy, Technology Classifications > Human Health Care > Therapeutics
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Receptor-Ligand Combination for Specific Drug Delivery to the Brain
Molecule Crosses the Blood-Cerebrospinal Fluid Barrier to Deliver Therapy into Targeted CellsThis engineered receptor-ligand combination targets and crosses the brain barriers of mammals to deliver drugs and therapeutics to the brain. About 12 percent of all deaths worldwide are caused by neurological disorders such as Alzheimer’s, Parkinson’s...
Published: 12/20/2021
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Inventor(s): Hector Mendez-Gomez, Nicholas Muzyczka
Keywords(s): Blood-brain barrier, brain delivery, brain-specific drug delivery, Gene Therapy, receptor-mediated transcytosis
Category(s): Technology Classifications > Human Health Care > Drug Delivery, Technology Classifications > Human Health Care > Gene Therapy, Technology Classifications > Research Tools > Therapeutics
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High Complexity AAV3 Capsid Library for Targeted Human Gene Therapy
Allows Efficient Development of Specialized Vectors for Targeted Gene DeliveryThis adeno-associated virus (AAV) vector library combines tens of millions of actual capsid variants in an AAV3 backbone in order to allow the isolation of specialized variants with increased transduction efficiency and high specificity for particular organs or cell populations,...
Published: 6/27/2021
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Inventor(s): Sergei Zolotukhin, Damien Marsic
Keywords(s): AAV Vectors, Gene Therapy, Liver
Category(s): Technology Classifications > Human Health Care, Technology Classifications > Human Health Care > Gene Therapy
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AAV Gene Therapy for Complications with Diabetes and Vascular Diseases
AAV Vectors Rely on Tissue-Specific Promoters to Target Vascular Endothelial Cells This AAV gene therapy transduces vascular endothelial cells and targets complications associated with diabetes and other vascular diseases. Available gene therapy vectors do not efficiently or selectively transduce vascular endothelial cells, which are associated with...
Published: 6/27/2021
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Inventor(s): William Hauswirth, Sanford Boye, Michael Boulton, Shannon Boye, Daniel Lipinski
Keywords(s): AAV, Diabetes, Gene Therapy, hypercholesterolemia, Hypertension, retinal disease, vascular endothelial junctions, Vascular Repair
Category(s): Technology Classifications > Human Health Care > Gene Therapy, Technology Classifications > Human Health Care > Therapeutics
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AAV Vector that Promotes Antioxidant Production for Treatment of Age-Related Macular Degeneration
Gene Therapy Requires Just One Intraocular Injection to Treat Eye Ailment Caused by Oxidative StressThis AAV gene therapy approach provides a one-time treatment to oxidative stress. Sustained oxidative stress can lead to a variety of pathophysiological diseases including age-related macular degeneration, diabetic retinopathy, and amyotrophic lateral...
Published: 12/22/2022
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Inventor(s): Qiuhong Li, Christhian Ildefonso, Alfred Lewin
Keywords(s): Age Related Macular Degeneration, Alzheimer's Disease, Diabetic Retinopathy, Gene Therapy, Macular Degeneration, Retinal Degeneration, uveitis
Category(s): Technology Classifications > Human Health Care > Gene Therapy, Technology Classifications > Human Health Care > Others
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Synthetic AAV Capsid Library for Targeted Gene Therapy with Improved Treatment Efficacy
Capsid Library Allows for Gene Therapy Vectors to Target Specific Organs and Tissues
This adeno-associated virus (AAV) capsid library allows for targeted gene therapy to specific organs and tissues. Gene therapy is a burgeoning market within the pharmaceutical industry, with a market value projected to reach $500 million by 2020. AAV derived vectors...
Published: 6/27/2021
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Inventor(s): Sergei Zolotukhin, Mavis Agbandje-McKenna, Damien Marsic
Keywords(s): AAV Vectors, capsid library, combinatorial library, Gene Therapy, Targeted delivery
Category(s): Technology Classifications > Human Health Care > Drug Delivery, Technology Classifications > Human Health Care > Gene Therapy
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