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Synthetic Peptide Mimetic for the Treatment of Rare Hematalogical Disorders

Technology #19-0178

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Researchers
David Williams
Managed By
Kyle Bartholomew
Commercialization Manager 919.962.5921
Patent Protection
Provisional Patent Application Filed

We have recently developed a cell penetrating, synthetic peptide for the treatment of cancer, sickle cell anemia and ß-thalassemia, that disrupts the MBD2:NuRD complex in human cells. This is based on the observation that enforced expression of a small peptide from the GATAD2A protein could disrupt complex formation and block DNA methylation, making this peptide a potential alternative to currently prescribed DNA methylation inhibitors, but without the harsh side effects. Additionally, this peptide can block the DNA methylation dependent gene silencing of fetal hemoglobin caused by MBD2, representing a novel therapeutic approach to ß-hemoglobinopathies.<!–[if supportFields]><![endif]–> This inhibition has been a long-standing goal of the hemoglobin gene regulation field of science. This novel peptide has been modified to increase its stability, efficacy, and distribution properties. Disruption of the MBD2:NuRD complex has also been shown to play a role in slowing tumor progression, and as a result, this peptide could also be developed as an oncology therapy.

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