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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…
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…
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…
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…
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…
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…
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…