Spherical Nucleic Acid (SNA™) Technology
Dermelix has executed a research and development agreement with Exicure, Inc., for the worldwide rights to an innovative genetic therapy approach called Spherical Nucleic Acid (SNA) technology for the treatment of rare genetic skin diseases. By configuring oligonucleotides in three dimensions, SNA’s have been proven to deliver high payloads of genetic targeting therapies into the skin in preclinical and clinical models of skin disease, which has been challenging with traditional oligonucleotide approaches. Dermelix will utilize SNAs to target dysregulated genes in a number of rare skin diseases, beginning with Netherton Syndrome.
Netherton Syndrome Background
Netherton Syndrome (NS) is a rare and severe autosomal recessive disorder caused by loss-of-function mutations in the SPINK5 gene. SPINK5 encodes the serine protease inhibitor LEKTI, which is involved in skin barrier function. NS affects approximately 1 in 200,000 children born each year and is characterized by severely inflamed, red, scaled, itchy skin. NS patients are at increased risk of mortality in the first year of life due to recurrent infections and dehydration as a result of the impaired skin barrier. Currently there are no approved treatments for NS patients and off-label use of standard-of-care treatments are of limited benefit. Visit the NIH.gov website for more information on Netherton Syndrome.