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Gene therapy for ATP1A3-Related Neurologic Disorders

Gene therapy for ATP1A3-Related Neurologic Disorders

Unmet Need Children with Alternating Hemiplegia of Childhood can have episodes of temporary paralysis, dystonia, and severe behavioral problems. The prevalence of AHC is estimated to be 1 in one million to 1 out of…

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Method for increasing gene expression in mammalian cells from transfected plasmids

Method for increasing gene expression in mammalian cells from transfected plasmids

Unmet Need Mammalian cell-based protein expression systems enable the production of mammalian proteins in the most native structure and activity. Thus, they have been commonly used for producing antibodies and therapeutic proteins, and more recently,…

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Gene therapy for long-term decrease of intraocular pressure

Gene therapy for long-term decrease of intraocular pressure

Unmet Need It is estimated that in the US alone, approximately 3 million individuals are living with glaucoma, making it the leading cause of blindness in the US. There currently is no cure for glaucoma,…

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Gene therapy and small molecule treatment of chronic pain without addiction

Gene therapy and small molecule treatment of chronic pain without addiction

Unmet Need Chronic pain is an escalating issue worldwide – in the U.S., an estimated one of five adults are affected. Chronic pain is difficult to treat, often requiring powerful opioids to provide relief. These…

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CRISPR-based antiviral treatment of coronavirus infections

CRISPR-based antiviral treatment of coronavirus infections

Unmet Need The global market for antiviral drugs is a large one, topping $60B, and is getting larger due to the ongoing COVID-19 pandemic. Spurred on by a $3B investment from the U.S. government into…

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A method of treating neurodegenerative diseases by preventing mitochondrial dysfunction

A method of treating neurodegenerative diseases by preventing mitochondrial dysfunction

Unmet Need Alzheimer's Disease (AD) is a devastating neurological disorder that results in significant cognitive impairment and memory loss. A common feature in late onset AD patients is the accumulation of amyloid plaques in the…

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A method for treating critical limb ischemia and other ischemic injuries

A method for treating critical limb ischemia and other ischemic injuries

Unmet Need The most severe stage of peripheral artery disease, a chronic vascular disease characterized by impaired circulation to patients’ lower extremities, is critical limb ischemia (CLI), and it affects approximately two million people in…

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An injectable hydrogel scaffold that delivers gene therapies locally

An injectable hydrogel scaffold that delivers gene therapies locally

Unmet Need Gene therapy has applications ranging from tissue engineering to vaccine development to infectious disease. Most approaches to deliver nucleic material involve the systemic intravenous delivery of condensed nucleic acid (e.g., within a virus…

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A CRISPR-based system for modifying the length of 3’ untranslated regions (3’UTR) of an mRNA transcript that offers novel treatment strategies for prostate cancer and other diseases

A CRISPR-based system for modifying the length of 3’ untranslated regions (3’UTR) of an mRNA transcript that offers novel treatment strategies for prostate cancer and other diseases

Unmet Need Abnormal length changes in the 3’ untranslated region (3’UTR) of mRNAs are associated with many diseases such as systemic lupus erythematosus, α-thalassemia, facioscapulohumeral muscular dystrophy, and cancer. While existing technologies can manipulate expression…

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A technique that promotes the stringent control of gene expression within specific cell types

A technique that promotes the stringent control of gene expression within specific cell types

Unmet Need A major challenge of gene therapy is to target only the intended cell- and/or tissue-type. For this reason, many gene therapies are delivered using adeno-associated virus (AAV) vectors with gene-specific promoters; however, off-target…

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Gene therapy for Crb1 retinopathies

Gene therapy for Crb1 retinopathies

Value Proposition Current treatments for retinal disease include laser surgeries, injectable corticosteroids, and other medications which may slow the process of retinal degeneration but are not able to stop it. The Crb1 protein has been…

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Isolated organ viral, theraputic or cell therapy delivery

Isolated organ viral, theraputic or cell therapy delivery

Value proposition Cardiovascular diseases (CVD) remain the leading cause of death worldwide. The number of CVD patients with heart failure (HF) in the US is approaching 6.5 million adults and is estimated to increase by…

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