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Stoke Therapeutics Presents Preclinical Data That Demonstrate In-Vitro and In-Vivo Target Engagement and Protein Upregulation in OPA1 Protein Deficiency, the Primary Cause of the Most Common Inherited Optic Nerve Disorder
BEDFORD, Mass.--(BUSINESS WIRE)--Stoke Therapeutics, Inc., (Nasdaq: STOK), a biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced new preclinical data demonstrating in-vitro and in-vivo target engagement and protein upregulation in OPA1 protein-deficient cells. OPA1 protein deficiency is the primary cause of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder