Search Results - metabolic+diseases

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AAV-Based Treatment of Guanidinoacetate Methyltransferase (GAMT) Deficiency (UCLA Case No. 2021-012)
SUMMARY: UCLA researchers in the Departments Molecular & Medical Pharmacology and Surgery in the David Geffen School of Medicine have developed a novel and tissue-specific gene therapy treatment for Guanidinoacetate methyltransferase (GAMT) deficiency. BACKGROUND: Guanidinoacetate Methyltransferase (GAMT) deficiency is caused by a mutation in...
Published: 8/10/2023   |   Inventor(s): Gerald Lipshutz
Keywords(s): Adeno-associated viruses (AAV), Creatine, GAMT deficiency, Gene Therapy, Metabolic Diseases
Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Genetic Diseases, Therapeutics > Metabolism And Endocrinology
Method for Identifying and Optimizing the Development of Heterodimeric Monoclonal Antibodies
Application Method to generate IgG heterodimers that can be used to generate bi-specific monoclonal antibodies for disease treatment. Key Benefits A novel method of screening and developing novel IgG heterodimers used for generating monoclonal antibodies for many diseases. Preliminary data demonstrate efficient production of IgG heterodimers that...
Published: 8/22/2024   |   Inventor(s): Eric Sundberg, Jonathan Du, Tala Azzam
Keywords(s): Antibody, Immunology/Inflammation, Metabolic Diseases, Microbiology/Infectious Diseases, Oncology
Category(s): TechPub Algolia > Therapeutics
Novel Endoglycosidase Derivatives Treating Diseases of the Adaptive Immune System
Application Protein-based therapeutics for the treatment of IgG-mediated diseases. Key Benefits A new class of endoglycosidases for the treatment of IgG-mediated diseases. Four novel compounds have been developed that exhibited robust in vitro activity. Potential to become first-in-class medications for dysregulation of the adaptive immune system,...
Published: 8/22/2024   |   Inventor(s): Eric Sundberg, Diego Sastre, Jonathan Du, Jeffrey Ravetch, Stylianos Bournazos
Keywords(s): Enzyme/Protein, Immunology/Inflammation, Metabolic Diseases, Oncology
Category(s): TechPub Algolia > Therapeutics
Whole-Animal Genetic Model to Mimic Aspects of the Patient Phenotype of Classic Galactosemia
­ Application An animal model for studying the underlying causes of galactosemia and screening for novel drugs to treat the condition. Key Benefits The first animal model for elucidating the underlying causes of galactosemia in infants. Animals with galactosemia causing genetic knockout exhibit similar disease phenotypes and complications...
Published: 7/30/2024   |   Inventor(s): Judith Fridovich-Keil
Keywords(s): Animal Model, Enzyme/Protein, Genetic Disorders, Metabolic Diseases, Rare/Orphan Diseases
Category(s): TechPub Algolia > Research Tools
2020-854 DUAL SELF-REGULATED DELIVERY OF INSULIN AND GLUCAGON BY A HYBRID PATCH
UCLA researchers have developed a novel hybrid microneedle patch that can deliver insulin and glucagon in a glucose-dependent manner. BACKGROUND: Type I diabetes (T1D) is an autoimmune disease in which pancreatic -cells are destroyed and there is a deficiency is insulin secretion. Type 2 diabetes (T2D) is a metabolic disorder resulting from insulin...
Published: 7/19/2023   |   Inventor(s): Zhen Gu
Keywords(s): Drug Delivery, Medical Devices and Materials, Metabolic Diseases, Other Polymers, Polymers, Therapeutics & Vaccines
Category(s): Medical Devices, Platforms > Drug Delivery, Therapeutics, Platforms > Drug Delivery > Other Polymers, Therapeutics > Metabolism And Endocrinology
HDAC Inhibitors for Managing Vascular Conditions and Diabetic Peripheral Neuropathies
Application Treatment of diabetic neuropathy using epigenetic modulators, histone deacetylase inhibitors (HDACis), to ameliorate or prevent vascular conditions and neuropathic pains by nerve damages. Key Benefits Reverses or protects against diabetes-induced nerve damages by direct injection of HDACi. Relatively low regulatory risk because many...
Published: 7/30/2024   |   Inventor(s): Young-Sup Yoon, Ji Woong (Jay) Han, Hyunsuk Shim
Keywords(s): Cell/Gene Therapy, Endocrinology, Metabolic Diseases, Repurposed Drug, Small Molecule
Category(s): TechPub Algolia > Therapeutics
2017-502 Novel Targets and Use of Small Molecule BT-2 to Treat Heart Failure, Obesity and Diabetes
Novel Targets And Use Of Small Molecule BT-2 To Treat Heart Failure, Obesity And DiabetesSUMMARYUCLA researchers from the Cardiovascular Theme in the Departments of Anesthesiology, Physiology and Medicine have discovered new pathways and potential treatment options for heart failure, obesity, and diabetesBACKGROUNDHeart failure is a leading cause of...
Published: 7/19/2023   |   Inventor(s): Yibin Wang, Haipeng Sun
Keywords(s): Cardiovascular, Endocrinology, Metabolic Diseases
Category(s): Therapeutics > Cardiovascular, Therapeutics > Metabolism And Endocrinology
2019-623 USE OF A NATURAL METABOLITE AS A CANCER THERAPY
UCLA Researchers in the Department of Chemistry and Biochemistry have devised a novel off label usage for the commonly available supplement s-adenylmethionine in the treatment of cancer.BACKGROUND: Tumor cells exhibit a wildly different metabolism from the tissue they are derived from. This altered metabolism allows sustained proliferation rates while...
Published: 7/19/2023   |   Inventor(s): Steven Clarke
Keywords(s): Metabolic Diseases, Oncology, Therapeutics & Vaccines
Category(s): Therapeutics > Oncology, Therapeutics > Metabolism And Endocrinology
2020-173 BIOCHEMICAL MARKERS OF PROVOKED VESTIBULODYNIA
UCLA researchers from the Departments of Medicine and Obstetrics and Gynecology have developed novel biochemical markers of provoked vestibulodynia. BACKGROUND:Provoked vestibulodynia (PVD) is a chronic pain disorder that involves hypersensitivity and severe pain localized to the vulvar vestibule. This disorder affects approximately 7 to 16% of the...
Published: 7/19/2023   |   Inventor(s): Jennifer Labus, Andrea Rapkin, Emeran Mayer
Keywords(s): Diagnostic Markers & Platforms, Metabolic Diseases
Category(s): Therapeutics > Metabolism And Endocrinology
2019-833 SMALL MOLECULE HETEROCYCLIC GOAT INHIBITORS
UCLA researchers have developed a potent and stable small molecule inhibitor of the ghrelin O-acyl transferase as potential therapy for obesity and type II diabetes. BACKGROUND:Obesity, and type II diabetes (T2D) resultant from obesity affect millions of people worldwide, and has reached epidemic level in society. Numerous means to counter the trend...
Published: 1/17/2024   |   Inventor(s): Patrick Harran
Keywords(s): Metabolic Diseases, Therapeutics & Vaccines
Category(s): Therapeutics > Metabolism And Endocrinology
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