Search Results - genetic+disorders

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A Method for Promoting Structural and Functional Rescue of Photoreceptors in the Eye to Treat Advanced Retinal Degeneration.
Discovery and use of retina-specific promoters to rescue photoreceptor function specifically at clinically relevant, advanced stages of disease. Problem: Despite the fact that retinal gene therapy studies have shown promising results in animal models, treatment of advanced retinal degeneration with currently available gene therapies have not been successful....
Published: 1/24/2025   |   Inventor(s): Raghavi Sudharsan, William Beltran
Keywords(s): Bioengineering, Gene Therapy, Genetic Disorders, Ophthalmology, Orphan Disease, Platform Technology, Regenerative Medicine
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: 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: 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
Silencing of PRLΔE1 expression: a gene-agnostic treatment for retinal degenerations
Viral vector-mediated gene silencing of a novel short retinal isoform of Prolactin promotes photoreceptor survival. Problem: Age-related macular degeneration (AMD) and inherited retinal degeneration (IRDs) have extreme mutational heterogeneity, and many IRDs are orphan diseases. Developing individual gene therapies for each causative gene is impractical...
Published: 7/2/2024   |   Inventor(s): William Beltran, Raghavi Sudharsan
Keywords(s): Drug Target, Gene Therapy, Genetic Disorders, Neurodegenerative Diseases, Ophthalmology, Orphan Disease
Category(s): Technology Classifications > Therapeutics
Methylene Blue and Methylene Violet Analogs as Mitochondrial Therapeutic Agents
Mitochondrial dysfunction has been implicated in a number of diseases, including Alzheimer’s disease (AD), movement disorders, diseases of the cardiovascular system, cancer, diabetes, blindness and deafness. Mitochondrial defects are damaging, particularly to tissues with high energy demands such as neural and muscle tissues. Membrane-penetrating...
Published: 2/13/2025   |   Inventor(s): Sidney Hecht, Omar Khdour, Sandipan Roy Chowdhury, Nishant Visavadiya
Keywords(s): Alzheimer's Disease, Diabetes, Genetic Disorders, Metabolic Disorders, Mitochondrial medicine
Category(s): Life Science (All LS Techs), Non-Cancer Therapeutics, Pharmaceuticals
Methylene Blue and Methylene Violet Analogs as Mitochondrial Therapeutic Agents
Mitochondrial dysfunction has been implicated in a number of diseases, including Alzheimer’s disease (AD), movement disorders, diseases of the cardiovascular system, cancer, diabetes, blindness and deafness. Mitochondrial defects are damaging, particularly to tissues with high energy demands such as neural and muscle tissues. Membrane-penetrating...
Published: 2/13/2025   |   Inventor(s): Sidney Hecht, Omar Khdour, Sandipan Roy Chowdhury, Indrajit Bandyopadhyay
Keywords(s): Alzheimer's Disease, Diabetes, Genetic Disorders, Metabolic Disorders, Mitochondrial medicine, Neurodegenerative Disorders, Parkinson's Disease, Pharmaceutical
Category(s): Life Science (All LS Techs), Non-Cancer Therapeutics, Pharmaceuticals
Direct and Effective Method To Reverse The Effects of Fragile X Syndrome
A method of treating Fragile X syndrome by correcting the abnormal DNA clusters causing the characteristic symptoms. Problem: Fragile X syndrome (FXS) affects approximately 1 in 4,000 males and 1 in 8,000 females and is characterized by symptoms such as: Inherited Intellectual Disability Anxiety Attention Deficit Disorder (ADD) Skin, Ovaries...
Published: 3/8/2024   |   Inventor(s): Jennifer Phillips-Cremins, Linda Zhou, Chunmin GE
Keywords(s): Bioengineering, Gene Therapy, Genetic Disorders, Neurodegenerative Diseases, Neurology
Category(s): Technology Classifications > Therapeutics, Technology Classifications > Vaccines
Small Molecule Activators of Valosin-Containing Protein (VCP) as Therapeutics for Neurodegenerative Diseases with Ubiquitinated Protein Aggregates
Small molecule therapeutics that aid in clearing abnormal protein deposits observed in neurodegenerative diseases. Problem: Dementia, characterized by the impairment of at least two brain functions, such as memory loss and judgment, currently has no cure. Two common neurodegenerative diseases that cause dementia are Frontotemporal Dementia (FTD) and...
Published: 2/9/2024   |   Inventor(s): Edward Lee, Benjamin Creekmore, Jessica Phan
Keywords(s): Aging, Genetic Disorders, Neurodegenerative Diseases, Neurology, Orphan Disease, Small Molecule
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
Site-blocking oligos (SBO) upregulate utrophin for treatment of Duchenne Muscular Dystrophy (DMD
Synthetic oligonucleotides blocking natural degradation of utrophin to treat muscular dystrophy. Problem: Duchenne Muscular Dystrophy (DMD) is a severe form of muscular dystrophy affecting males. A genetic mutation causes decreased production of dystrophin, a critical protein connecting cells to surrounding tissue. This leads to improper muscle...
Published: 6/20/2024   |   Inventor(s): Tejvir Khurana
Keywords(s): Genetic Disorders, Neurodegenerative Diseases, Orphan Disease
Category(s): Technology Classifications > Therapeutics
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