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Gene therapy for ATP1A3-Related Neurologic Disorders
Unmet Need Alternating Hemiplegia of Childhood (AHC) is a rare neurologic disorder affecting children, causing episodes of temporary paralysis, dystonia, and severe behavioral issues, with prevalence estimates ranging from 1 in 1 million to 1…
Novel epigenome editors for multiplexed gene regulation
Unmet Need Epigenome editors activate or inhibit gene expression without modifying the underlying DNA sequence. Consequently, these editors can reversibly fine-tune gene expression without the risk of permanently altering the genome or off-target editing. Epigenome…
Methods and compositions for treating protein aggregation diseases
Unmet Need Neurodegenerative disease affects more than 50 million people per year. Certain neurodegenerative diseases, like Alzheimer’s disease, result from the accumulation of amyloid-forming proteins in the brain, which results in cognitive impairment and has…
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…
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…
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…