Search Results - therapeutic

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Epigenetic Methylation inhibitors displaying anti-tumor activity
Background Diffuse intrinsic pontine glioma (DIPG) is a lethal malignant tumor that arises from the middle portion of the brain stem and usually affects children with a poor prognosis, as there are no surgical options for providing relief to patients. Conventional chemotherapy as well as radiation therapy provide minimal life extension and palliative...
Published: 11/30/2022   |   Inventor(s): Frank (Hal) Ebetino, Venkatesan Srinivasan, Rintaro Hashizume, Robert Boeckman, Jr.
Keywords(s):  
Category(s): Therapeutic
Gene Therapy for Reflux Disease in the Esophagus
Background Almost one-third of adults in the US experience symptoms of gastroesophageal reflux disease (GERD) and patients with chronic GERD have an increased risk for the development of esophageal adenocarcinoma. Although proton pump inhibitors (PPIs) have shown effective treatment for GERD, up to 45% of patients with GERD remain symptomatic on standard...
Published: 11/8/2022   |   Inventor(s): David Dean, Zhongren (David) Zhou
Keywords(s):  
Category(s): Therapeutic
Macrocyclic Peptide Phage Display
­Background Peptides represent a growing class of therapeutics with potential to treat different conditions such as diabetes and various forms of cancer. However, therapeutic peptides have two intrinsic drawbacks: poor cell permeability and susceptibility to proteolytic degradation, which represent major stumbling blocks for peptide drug development....
Published: 8/5/2022   |   Inventor(s): Rudi Fasan, Andrew Owens, Jacob Iannuzzelli
Keywords(s):  
Category(s): Therapeutic
Method for using cerebrospinal fluid to deliver gene therapeutics to the inner ear and rescue hearing
Background An estimated 2.45 billion of individuals are predicted to have mild-to-complete hearing impairment by 2050. Current ameliorating therapy using hearing aids or cochlear implants remain insufficient in restoring the full capacities of auditory function. Gene therapy has shown promising results in animal models but the delivery of genetic material...
Published: 7/25/2022   |   Inventor(s): Maiken Nedergaard
Keywords(s):  
Category(s): Therapeutic
A method for treating a myotonic disorder
Background Myotonic disorders are a group of genetic diseases characterized by the presence of myotonia, which is defined as failure of muscle relaxation after activation. The presentation of these disorders can range from asymptomatic electrical myotonia, as seen in some forms of myotonia congenita (MC), to severe disability with muscle weakness,...
Published: 7/25/2022   |   Inventor(s): John Lueck, Charles Thornton, Lily Cisco
Keywords(s):  
Category(s): Therapeutic
Platform based on RNA splicing to produce functional human dystrophy gene for treatment of Duchene Muscular Dystrophy
Background Duchenne muscular dystrophy (DMD) is among the most common fatal genetic diseases in children, occurring in 1 of every ~3,500 live male births. DMD is caused by mutations in the dystrophin gene which is required to maintain muscle fiber integrity. DMD is a debilitating muscle disorder characterized by progressive muscle weakness, cyclic...
Published: 7/25/2022   |   Inventor(s): Douglas Anderson
Keywords(s):  
Category(s): Therapeutic
Small molecule for treatment of Acute Myelogenous Leukemia
­Background Acute myeloid leukemia (AML), also known as acute myelogenous leukemia, is a fast-growing form of cancer of the blood and bone marrow. There are about 30,000 new cases of AML diagnosed per year in the USA, and the incidence is rising given the aging of the population. The current standard-of–care repertoire of therapies is often...
Published: 7/25/2022   |   Inventor(s): Rakesh Singh, Deepak Sahasrabudhe
Keywords(s):  
Category(s): Therapeutic
Septin inhibitors for treatment of cancer
Background Effective therapeutic options are limited for the treatment of liver, lung, renal, ovarian, endometrial and pancreatic cancer. Successive second- and third-line therapies do not add meaningful responses besides adding numerous life-debilitating toxicities. In order to improve the survival rate of patients diagnosed with these lethal malignancies,...
Published: 7/25/2022   |   Inventor(s): Richard Moore, Rakesh Singh, Kyu Kwang Kim, Rachael Turner
Keywords(s):  
Category(s): Therapeutic
Method for improving delivery of immunotherapeutic agents to the CNS
­Background Up to 1 billion people, suffer from neurological disorders, from Alzheimer (AD) and Parkinson disease, strokes, multiple sclerosis and epilepsy to migraine, brain injuries and neuroinfections. Most therapies fail mainly due to the poor penetration of the blood-brain barrier (BBB). Therefore, improving the delivery of drugs to the CNS...
Published: 7/25/2022   |   Inventor(s): Maiken Nedergaard, Benjamin Plog, Humberto Mestre, Tuomas Olavi Lilius, Andreas Tue Ingemann Jensen, Steven Goldman
Keywords(s):  
Category(s): Therapeutic
RNA-based antisense platform for the treatment of human diseases
­Background The central dogma of molecular biology demonstrates the two steps of gene expression, DNA transcription and mRNA translation, which generate functional proteins for any living cells. In any disease state, phenotypic changes are driven by insufficient production of protective proteins or overproduction of pathogenic proteins made by...
Published: 7/11/2022   |   Inventor(s): Peng Yao, Omar Hedaya
Keywords(s):  
Category(s): Therapeutic
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