ORYZON starts preclinical collaboration on Kabuki syndrome with Kennedy Krieger Institute and Johns Hopkins University

Oryzon Genomics, S.A., a clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with strong unmet medical need, announced today the start of a preclinical collaboration on Kabuki Syndrome with researchers from Kennedy Krieger Institute and Johns Hopkins University led by Dr. Jacqueline Harris, Director of the Epigenetics Clinic at Kennedy Krieger Institute and an assistant professor in pediatrics, neurology and genetics at the Johns Hopkins University School of Medicine and Dr. Hans Bjornsson, Founder of the Epigenetic and Chromatin Clinic and associate professor of pediatrics and genetics at the Johns Hopkins University School of Medicine.

Kabuki syndrome (KS) is an autosomal dominant/X-linked disorder that affects multiple organ systems including neuro, immune, auditory and cardiac systems. Patients show characteristic distinctive facial features, growth retardation, and mild to moderate intellectual disability and autoimmune disorders. The majority (>70%) of molecularly confirmed cases of KS have loss-of-function variants in KMT2D gene. This gene, aka MLL2, catalyzes the addition of methyl groups to lysine 4 of histone 3, which are marks associated with open chromatin, thus regulating the expression of critical target genes.

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