Search Results - Therapeutic

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Factor IX Variants With Enhanced Biological Functions
Market NeedHemophilia B, also known as Christmas disease, is an x-linked genetic disorder caused by missing or defective Factor IX (FIX), a clotting protein. It is estimated that hemophilia occurs 1 in every 5000 births with 20,000 estimated to have hemophilia in the US alone. The severity of decrease in FIX levels in Hemophilia B patients varies f...
Published: 2/27/2018   |   Inventor(s): Valder Arruda, Rodney Camire, Benjamin Samelson-jones
Keywords(s):  
Category(s): Therapeutic
Chimeric Allo-Antigen Receptors (CALLARs) directed to Factor VIII autoantibodies
Market NeedHemophilia A is an inherited X-linked disease caused by Factor VIII (FVIII) deficiency and is a life-threatening bleeding disorder. The disease is associated with frequent hemarthosis and arthropathy that causes significant morbidity for patients. Factor replacement therapy using recombinant human FVIII (rhFVIII) is the standard of care ...
Published: 2/27/2018   |   Inventor(s): Valder Arruda, Sarah Richman, Benjamin Samelson-jones, Michael Milone
Keywords(s):  
Category(s): Therapeutic
Methods For The Generation Of Self Renewing Endodermal Precursor Populations Derived From Human Embryonic Stem Cells And Induced Pluripotent Stem Cells
Market NeedCell-based therapies for disease of endodermally derived organs such as the liver, pancreas, and intestines have gained significant interest. In addition to cell-based treatment strategies, there is a proven value in screening drugs in cellular models of the organ systems during development process. Commonly this screening is performed o...
Published: 2/27/2018   |   Inventor(s): Paul Gadue, Deborah French, Xin Cheng
Keywords(s):  
Category(s): Therapeutic
AAV-Mediated GRN Gene Delivery For Therapy Of Frontotemporal Dementia Due To Progranulin Mutations
Market NeedFrontotemporal dementia (FTD) is a highly heritable form of early onset dementia with a prevalence in 20-50 year olds with dementia similar to that of Alzeimer’s disease (15 in 100,000). On average it begins in the sixth decade of life and has poor life expectancy that is on par with Alzheimer’s. It is caused by frontotempora...
Published: 2/27/2018   |   Inventor(s): Beverly Davidson, Dfne Amado, Alice Chen-plotkin, Julianne Rieders
Keywords(s):  
Category(s): Therapeutic
Gene-Editing Of FMR1 Using CRISPR/Cas9
Market NeedFragile X syndrome is an X-chromosome-linked disease that affects males more severely than females and is the most common cause of inherited intellectual disability, often co-occurring with Autism spectrum disorder. According to the CDC, around 1 in every 6000 males and 7000 females are born with the condition. Patients usually present w...
Published: 2/27/2018   |   Inventor(s): Beverly Davidson, Carolyn Yrigollen, Alejandro mas Monteys
Keywords(s):  
Category(s): Therapeutic
Allele Specific Genome Editing For Huntington's Disease Therapy
Market NeedHuntington’s Disease (HD) is an orphan neurodegenerative disease characterized by dementia, declining cognitive function, chorea that gradually spreads to all muscles, hypokinesia, rigidity, and other symptoms of psychomotor decline. Mean onset of symptoms is 30-50 years, however there is a juvenile onset form of HD that occurs und...
Published: 2/27/2018   |   Inventor(s): Beverly Davidson, Alejandro mas Monteys, Shauna Ebanks
Keywords(s):  
Category(s): Therapeutic
Repurpose PARP-1 Inhibitors for Type 2 Diabetes and Cystic Fibrosis Related Diabetes
Expand to Two Additional Indications for Existing DrugMarket NeedCurrent therapies for type 2 diabetes (T2D), including the closely similar cystic fibrosis related diabetes (CFRD), only address the symptoms, not the underlying pathophysiology of the disease. So instead of halting disease progression, the growing number of patients continue to have ...
Published: 5/10/2017   |   Inventor(s): Qianghua Xia, Struan Grant
Keywords(s): Cystic Fibrosis, Diabetes
Category(s): Therapeutic
Protect Medical Device Implants Against Oxidative Damage
Oxidation Resistant Bioprosthetic TissuesMarket NeedOver 200,000 patients worldwide receive bioprosthetic heart valves. Compared to mechanical valves, bioprosthetics do not require patient anti-coagulation treatment. Although the risk of thromboembolism is significantly reduced, bioprosthetic valves demonstrate poor durability. Structural deteriora...
Published: 5/9/2017   |   Inventor(s): Robert Levy, Ivan Alferiev, Stanley Stachelek, Jeanne Connolly, Abigail Christian
Keywords(s): Anti-Oxidant
Category(s): Therapeutic
Harnessing viral strategies to suppress inflammation
Anti-inflammatory action through chromatin modulationMarket NeedInflammation is a hallmark of diseases such as sepsis, endotoxaemia, arthritis, acute respiratory disease, and multiple organ failure. Steroidal and non-steroidal anti-inflammatory medications (NSAIDS) both carry different risks, including risk of infection due to immune suppression wi...
Published: 2/23/2017   |   Inventor(s): Matthew Weitzman, G. scott Worthen, Daphne c. Avgousti, Christin Herrmann, Andrew Paris
Keywords(s): Inflamation
Category(s): Therapeutic
Enhancing platelet production from megakaryocytes in-vitro
Sorting megakaryocytes by the presence of Factor V selects for cells with high platelet yieldMarket NeedPlatelets are critical as a first line treatment for trauma and hematological diseases. The demand for platelet transfusions is increasing along with the aging population and supply is often stressed in times of increased need. In fact, it has be...
Published: 2/23/2017   |   Inventor(s): Deborah French, Paul Gadue, Mortimer Poncz, Rodney Camire, Xiuli Sim
Keywords(s): Hematology
Category(s): Therapeutic
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