How CUR-1916 works
Dravet syndrome is a genetic condition characterized by severe seizures, often starting in the first year of life. Commonly, the condition is caused by a mutation in the SCN1A gene, resulting in little or no functional protein being produced from the faulty copy of the gene.
We all have two copies of every gene, one inherited from our mother and one from our father. In Dravet syndrome, one faulty copy of the SCN1A gene — often caused by a mutation that is not inherited, but unique to a patient — cannot produce enough protein to prevent the condition from developing. The gene provides instructions for making sodium channels, which are central to a cell’s ability to generate and transmit electrical signals.
CUR-1916 aims to boost protein production from the functional SCN1A gene, bringing the overall protein levels to a more normal range.
Gene expression is normally regulated by “natural antisense transcripts,” or NATs, which interact with specific genes to increase or decrease their protein production.
Using the AntagoNAT technology platform, the OPKO-CURNA program identified a NAT that naturally inhibits protein production from the SCN1A gene, called SCN1ANAT.
CUR-1916 is designed to specifically bind to and suppress SCN1ANAT. This frees the healthy gene — SCN1A — from NAT regulation and ultimately results in it producing more protein to compensate for the mutated gene.
Increasing protein levels may address not just the symptoms of Dravet syndrome but its underlying cause as well. This should reduce seizure-related symptoms, and also improve thinking, and behavioral and motor problems associated with Dravet.
CUR-1916 in clinical trials
CUR-1916 has not yet been tested in people. OPKO plans to initiate a Phase 2 clinical trial at three U.S. sites by the end of 2018.
They show that CUR-1916 is highly specific to SCN1ANAT, and that it was able to increase protein production from the SCN1A gene in both animals.
CUR-1916 also improved seizure symptoms in the diseased mice. Further studies are ongoing to confirm whether it might also ease developmental problems.
AntagoNAT was originally developed by CURNA Pharmaceuticals, which was acquired by OPKO in 2011.
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