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Cell and Gene Therapies for Hemophagocytic Lymphohistiocytosis
Application Gene/cell therapy to treat hemophagocytic lymphohistiocytosis. Key Benefits Gene therapy approach for treating patients with UNC13D mutated hemophagocytic lymphohistiocytosis. Preliminary in vitro and in vivo data show the ability of the gene therapy to increase UNC13D protein levels and decrease T cell activation. It is administered...
Published: 9/26/2023       Contributor(s): H. (Harold) Trent Spencer, Christopher Doering, Shanmuganathan Chandrakasan, Sarah Takushi
Therapeutic for Potential Elimination of Chronic Hepatitis B
Application A therapeutic technique to eliminate infection of patients suffering from chronic Hepatitis B Virus infection. Key Benefits May provide a cure for chronic HBV infection rather than just simple management. High efficiency of infection targeting may produce minimal side effects. May be used in combination with other HBV inhibitors. Market...
Published: 9/15/2023       Contributor(s): Raymond Schinazi, Leda Bassit, Ting Nie
Repurposing an FDA-Approved Drug for Treating Age-Associated Lung Fibrosis
Application New use of an FDA-approved therapeutic, diroximel fumarate (Vumerity), for treating Idiopathic Pulmonary Fibrosis. Key Benefits Novel use of an FDA-approved drug, diroximel fumarate - DRF (Vumerity), for treating age-dependent lung fibrosis. Lung-targeted inhaled delivery of DRF is effective for boosting lung antioxidant responses...
Published: 9/26/2023       Contributor(s): Louise Hecker, Kosuke Kato
Vaccine-pulsed Dendritic Cells as a Treatment for Aggressive Breast Cancers
Application Immunotherapy for HER2+ and triple-negative breast cancer. Key Benefits Autologous vaccine comprising patient-specific tumor antigens and immune stimulatory components against aggressive breast cancers. Prepared outside of the body and then administered to patients. Preclinical data indicate treatment decreases tumor growth and improves...
Published: 7/28/2023       Contributor(s): Periasamy (Selva) Selvaraj, Luis Munoz, Ramireddy Bommireddy, Christopher Pack, Shaker Reddy, Sampath Ramachandiran
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: 9/18/2023       Contributor(s): Eric Sundberg, Jonathan Du
Engineering Asymmetrically Glycosylated IgG Antibodies
Application A novel method to develop asymmetrically glycosylated therapeutic monoclonal IgG antibodies for preventing and treating diseases like cancer, infectious diseases, and autoimmune disorders. Key Benefits Different IgG antibody glycoforms can be produced, each with its unique effector functions and biological and therapeutic effects. Potential...
Published: 9/18/2023       Contributor(s): Eric Sundberg, Jonathan Du
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: 9/26/2023       Contributor(s): Eric Sundberg, Diego Sastre, Jonathan Du, Jeffrey Ravetch, Stylianos Bournazos
Drug Screening System for Cardioprotection
Application A high throughput screening assay for developing therapies to protect from drug and alcohol-induced cardiotoxicity. Key Benefits A cardiovascular stem-cell-based screening assay that can identify drugs that protect the heart from toxic medications and alcohol. Preliminary data in the laboratory show the system is clinically relevant...
Published: 9/15/2023       Contributor(s): Chunhui Xu, Antonio Rampoldi, Yuhong Du
Guiding the Cartilage Repair Microenvironment
Application A novel method to improve the effectiveness of marrow-mediated cartilage repair. Key Benefits Enhances the efficacy of the marrow stimulation procedure for cartilage repair. Priming the cartilage repair environment to enable subsequent healthy tissue growth. Potential to become a one-step, implementable, and effective treatment for...
Published: 9/15/2023       Contributor(s): Sameh Labib, David Myers, Jay M. Patel, Maddie Hasson, Lorenzo Fernandes
Therapeutic to Treat Age-Dependent Idiopathic Pulmonary Fibrosis
Application Therapeutic for treating age-associated lung fibrosis. Key Benefits Tecfidera (dimethyl fumarate - DMF) is an FDA approved therapy to treat multiple sclerosis via oral administration. A novel formulation for inhaled delivery demonstrated efficacy for a new indication: Idiopathic Pulmonary Fibrosis (IPF), where it promotes the reversal...
Published: 9/6/2023       Contributor(s): Louise Hecker, Kosuke Kato, Priyadarshini Muralidharan, Heidi Mansour
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