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Search Results - gene+editing
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Human Artificial Chromosome Generation Technology
Technology allowing efficient formation, delivery, isolation, and enrichment of single-copy Human Artificial Chromosome (HAC) for use in research, plant production, gene and cell therapy and more. Problem: Existing gene-editing techniques have limitations due to their reliance on viruses for delivery, restricting their packaging capacities. A more potent...
Published: 9/18/2025
|
Inventor(s):
Ben Black
,
Craig Gambogi
,
John Glass
,
David Brown
,
Gabriel Birchak
Keywords(s):
Bioengineering
,
Gene Editing
,
Gene Therapy
,
Genetic Disorders
,
Platform Technology
Category(s):
Technology Classifications > Research Tools & Reagents
,
Technology Classifications > Therapeutics
LNP + Squeeze: Enhancing Chimeric Antigen Receptor T-Cell Generation via Mechanoporation and Lipid Nanoparticles
Synergistic combination of mechanoporation and lipid nanoparticle (LNP) based transfection for an efficient, non-viral method of gene transfer to generate CAR-T cell therapies. Problem: Chimeric antigen receptor (CAR)-T cell therapy has transformed cancer treatment by engineering patients' T cells to precisely target and eradicate cancer cells....
Published: 8/28/2025
|
Inventor(s):
Jina Ko
,
Jianhua Lim
Keywords(s):
Bioengineering
,
Cell Therapy
,
Gene Editing
,
Immunology
,
Immunoncology
,
Microfluidics
,
Nanotechnology
Category(s):
Technology Classifications > Research Tools & Reagents
,
Technology Classifications > Therapeutics
,
Technology Classifications > Chemical Processes and Synthesis
Targeted In Utero Therapeutics Delivery System for Precise
Gene Editing
Targeted lipid nanoparticles (LNPs) that can deliver mRNA-based
gene editing
therapeutics to hematopoietic stem cells (HSCs) in utero Problem: Monogenic blood diseases, including sickle cell disease and / thalassemia, are among the most prevalent genetic conditions worldwide, causing significant pediatric morbidity with complications like painful...
Published: 1/2/2025
|
Inventor(s):
Rohan Palanki
,
Michael Mitchell
,
William Peranteau
Keywords(s):
Bioengineering
,
Gene Editing
,
Gene Therapy
,
Platform Technology
Category(s):
Technology Classifications > Therapeutics
The First Curative Treatment of Phenylketonuria Using Genetic Engineering Approaches
The base editing of phenylalanine hydroxylase enzyme in the liver to restore phenylalanine metabolism and prevent neurotoxic effects. Problem: Phenylketonuria (PKU) is a severe disorder affecting approximately 1 in 10,000 U.S. newborns. PKU is marked by a genetic mutation in the phenylalanine hydroxylase enzyme (PAH), resulting in phenylalanine (Phe)...
Published: 8/21/2025
|
Inventor(s):
Kiran Musunuru
,
Xiao Wang
,
Dominique Brooks
Keywords(s):
Gene Editing
,
Gene Therapy
,
Genetic Disorders
,
Neurodegenerative Diseases
,
Neurology
Category(s):
Technology Classifications > Therapeutics
LNP Delivery of Gene-Editing Machinery to Cure Hereditary Tyrosinemia Type 1g
Problem: Hereditary tyrosinemia type 1 (HT1) is a genetic metabolic disease caused by the inability to break down tyrosine, an amino acid present in many meats, dairy products, fruits, and nuts. This disease can be fatal within the first few months of life and increases the risk of liver cancer. The current treatment involves a strict drug regimen and...
Published: 8/21/2025
|
Inventor(s):
Kiran Musunuru
,
Madelynn Whittaker
,
Xiao Wang
,
William Peranteau
,
Cara Berkowitz
,
Ana Dumitru
Keywords(s):
Gene Editing
,
Genetic Disorders
,
Obesity Metabolic Disease and Diabetes
Category(s):
Technology Classifications > Therapeutics
Strategies for Mitigating Acute Side Effects of Lung-Targeted Nanocarriers
Approaches and Design Techniques to Alleviate Coagulation-Related Acute Side Effects of Lung-Targeted Nanocarriers Problem: Lipid nanoparticles (LNPs) play a crucial role in drug delivery, enabling targeted drug administration to specific tissues. LNPs tailored for lung-specific delivery often incorporate cationic (positively charged) lipids. Unfortunately,...
Published: 8/6/2025
|
Inventor(s):
Jacob Brenner
,
Vladimir Muzykantov
,
Jacob Myerson
,
Oscar Marcos Contreras
,
Marco Zamora
,
Serena Omo-Lamai
Keywords(s):
Bioengineering
,
Drug Delivery
,
Gene Editing
,
Gene Therapy
,
Nanotechnology
,
Platform Technology
,
Pulmonary
Category(s):
Technology Classifications > Therapeutics
Cholesterol Analogues Incorporated Into LNPs For Enhanced mRNA Delivery
Hydroxycholesterols are used to formulate lipid nanoparticles (LNPs) for enhanced mRNA delivery into T cells. Problem: In recent times, the delivery of mRNA has become a major focus for therapeutics. Intracellular delivery is the major challenge. Intracellular delivery requires ionizable lipids for mRNA delivery. In addition to this, these constituents...
Published: 2/6/2024
|
Inventor(s):
Michael Mitchell
,
Margaret Billingsley
,
Savan Patel
,
Xuexiang Han
,
Hanwen Zhang
Keywords(s):
Bioengineering
,
Cell Therapy
,
Drug Delivery
,
Gene Editing
,
Gene Therapy
,
Genetic Disorders
,
Immunology
,
Immunoncology
,
Immunotherapy
,
Inflammation
,
Nanotechnology
,
Platform Technology
Category(s):
Technology Classifications > Therapeutics
Amniotic Fluid Stabilized Lipid Nanoparticles (LNPs) for In Utero Intra-Amniotic mRNA Delivery
Nanoparticles for potent encapsulation and delivery of therapeutic molecules for prenatal fetal therapy in the amniotic sac Problem: Many prenatal genetic diseases suffer from ineffective postnatal treatments. For this reason, therapies that are delivered to the fetus before birth hold promise to treat diseases prior to or in the early stages of...
Published: 2/7/2024
|
Inventor(s):
Michael Mitchell
,
Kelsey Swingle
,
Margaret Billingsley
,
William Peranteau
Keywords(s):
Bioengineering
,
Cell Therapy
,
Gene Editing
,
Gene Therapy
,
Genetic Disorders
,
Immunology
,
Nanotechnology
,
Orphan Disease
,
Platform Technology
Category(s):
Technology Classifications > Therapeutics
Ligand-Tethered Lipid Nanoparticles for Targeted RNA Delivery to Treat Liver Fibrosis
A new class of anisimide-functionalized lipids that enables targeted delivery of lipid nanoparticles (LNPs) to activated fibroblasts in the liver. Problem: Lipid nanoparticles (LNPs) have been successfully used in FDA-approved therapies to deliver messenger RNA (mRNA) and small interfering RNA (siRNA) . One of the biggest challenge in developing...
Published: 2/7/2024
|
Inventor(s):
Michael Mitchell
,
Xuexiang Han
Keywords(s):
Bioengineering
,
Drug Delivery
,
Gene Editing
,
Gene Therapy
,
Genetic Disorders
,
Immunology
,
Immunoncology
,
Platform Technology
,
Regenerative Medicine
Category(s):
Technology Classifications > Therapeutics
,
Technology Classifications > Vaccines
ADENO-ASSOCIATED VIRUS (AAV) DIRECTED ANTIOXIDATIVE GENE THERAPY FOR THE PREVENTION AND TREATMENT OF NOISE-INDUCED HEARING LOSS
SOD1 gene therapy (blue) shows a persistent neuroprotective effect compared to the severe hearing loss observed in the control animals (purple) Invention Summary: Hearing loss is the third most common chronic health problem in the United States, with noise-induced hearing loss effecting nearly 1 out of every 4 adults (over 60M). Inner ear hearing...
Published: 8/12/2025
|
Inventor(s):
P. Ashley Wackym
,
Todd Mowery
Keywords(s):
Gene editing
Category(s):
Technology Classifications > Healthcare & Life Sciences
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