Search Results - gene+editing

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Durable cure for Pseudoxanthoma Elasticum: High-Efficiency, Precision-Targeted Gene Therapy
Lipid nanoparticles containing messenger RNA (mRNA-LNPs) encoding Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) base-editors that correct pseudoxanthoma elasticum-causing mutations. Problem: Pseudoxanthoma elasticum (PXE) is a genetic disorder affecting approximately 1 in 25,000 Americans. It is caused by loss-of-function mutations...
Published: 6/17/2026   |   Updated: 6/17/2026   |   Inventor(s): Xiao Wang, Hanbing Cao
Keywords(s): Bioengineering, Cardiovascular, Gene Editing, Gene Therapy, Genetic Disorders, Orphan Disease
Category(s): Technology Classifications > Research Tools & Reagents, Technology Classifications > Therapeutics
A Fast And Cost-Effective Method For Early Cancer Detection
A quick and inexpensive method to detect cancer early by exponentially amplifying rare and scarce cancer mutations. Problem: Early cancer detection is challenging due to the low levels of cancer-indicating mutant DNA compared to healthy, wild-type DNA. The background noise from the substantial amounts of healthy DNA overwhelms mutant signals. Some...
Published: 6/12/2026   |   Updated: 6/12/2026   |   Inventor(s): Haim Bau, Jinzhao Song, Junman Chen
Keywords(s): Bioengineering, Gene Editing, Gene Therapy, Oncology
Category(s): Technology Classifications > Diagnostics, Technology Classifications > Research Tools & Reagents
Multipronged Composite Protocol for High-Efficiency Deep Tissue RNA Delivery and Expression
An RNA transfection and protein expression approach comprising lipid nanoparticles, hyaluronic acid, and photostimulation Problem: As the field of cell modification has expanded, researchers have sought more effective methods for nucleic acid (NA) delivery into cells. RNA transfection is easier to facilitate and control than DNA transfection, making...
Published: 5/13/2026   |   Updated: 5/13/2026   |   Inventor(s): James Eberwine, Jai-Yoon Sul, Hyunbum Kim
Keywords(s): Cell Therapy, Gene Editing, Gene Therapy, Platform Technology, Protein/Peptide (Non-Antibody)
Category(s): Technology Classifications > Medical Devices, Technology Classifications > Research Tools & Reagents, Technology Classifications > Therapeutics
An All-In-One “Swiss Army Knife” Tool To Edit Genes And Regulate Their Expression Without Off-Target Effects And Toxicity
A flexible toolkit for human gene editing, activation, and repression that can be applied in orthogonally and in various disease models. Problem: There is increasing demand for technologies that can simultaneously edit the genome and regulate the transcriptome without relying on toxic double-stranded breaks. Currently, treating complex diseases such...
Published: 5/5/2026   |   Updated: 5/5/2026   |   Inventor(s): Xue Sherry Gao, Tyler Daniel, Hongzhi Zeng, Qichen Yuan
Keywords(s): Bioengineering, Cell Therapy, Gene Editing, Genetic Disorders, Neurodegenerative Diseases, Obesity Metabolic Disease and Diabetes, Oncology, Orphan Disease, Pulmonary
Category(s): Technology Classifications > Research Tools & Reagents, Technology Classifications > Therapeutics
Device For Rapid, Tunable, And Scalable Generation Of Lipid Nanoparticle Formulations
Microfluidic device for generation of large, precisely-defined, diverse lipid nanoparticle libraries for screening LNP formulations. Problem: Lipid nanoparticles (LNPs) have broad applications such as delivery agents for gene editing cargo, immunotherapies, and many clinical applications. The structures of the lipid components of an LNP, their relative...
Published: 4/17/2026   |   Updated: 4/17/2026   |   Inventor(s): Michael Mitchell, David Issadore, Andrew Hanna
Keywords(s): Bioengineering, Drug Delivery, Gene Editing, Gene Therapy, Immunology, Immunotherapy, Microfluidics, Nanotechnology
Category(s): Technology Classifications > Chemical Processes and Synthesis, Technology Classifications > Hardware & Components, Technology Classifications > Robotics
An Improved In Vivo Gene-Editing System Using Plug-and-Play Lipid Nanoparticles (LNPs) to Support CRISPR, Small Interfering RNA (siRNA), and Adenine Base Gene Editing Platforms
Simple, plug-and-play biodegradable lipids that form highly potent lipid nanoparticles (LNPs) for CRISPR mRNA delivery, outperforming benchmark gene-editing systems. Problem: CRISPR therapies can permanently correct disease-causing genes, but today's CRISPR delivery systems are either too risky or not potent enough. Viral vectors raise red flags...
Published: 1/14/2026   |   Updated: 1/14/2026   |   Inventor(s): Michael Mitchell, Xuexiang Han, Kelsey Swingle, Alex Hamilton
Keywords(s): Bioengineering, Drug Delivery, Gene Editing, Gene Therapy, Nanotechnology, Platform Technology, Protein/Peptide (Non-Antibody), Small Molecule
Category(s): Technology Classifications > Therapeutics, Technology Classifications > Vaccines, Technology Classifications > Chemical Processes and Synthesis, Technology Classifications > Materials, Technology Classifications > Research Tools & Reagents
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: 6/10/2026   |   Updated: 9/17/2025   |   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   |   Updated: 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
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: 2/5/2026   |   Updated: 10/28/2024   |   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   |   Updated: 7/10/2024   |   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
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