Stoke Therapeutics Enrolls First Patient in a Natural History Study of People Living with Autosomal Dominant Optic Atrophy (ADOA)
– ADOA is primarily caused by mutations in the OPA1 gene that result in progressive and irreversible vision loss in both eyes starting in the first decade of life –
– The FALCON study is designed to evaluate disease progression and its effects on patients –
– Data will support clinical development plans for STK-002, a potential new medicine that targets the underlying cause of ADOA –
Excerpt from the Press Release:
BEDFORD, Mass.–(BUSINESS WIRE)–Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced enrollment of the first patient in a prospective natural history study of people ages 8 to 60 who are living with autosomal dominant optic atrophy (ADOA). ADOA is the most common inherited optic nerve disorder. It is a rare disease that causes progressive and irreversible vision loss in both eyes starting in the first decade of life. There are currently no approved treatments for ADOA.
Most cases of ADOA are caused by mutations in one allele of the OPA1 gene, which result in half of the normal OPA1 protein production. FALCON is a two-year prospective natural history study in patients who have a confirmed diagnosis of ADOA that is caused by an OPA1 mutation. The study is designed to evaluate the rate of change in structural and functional ophthalmic assessments. Data from the FALCON study will support the clinical development of STK-002, Stoke’s proprietary antisense oligonucleotide (ASO) in preclinical development for the treatment of ADOA.
“ADOA is a severe and progressive disease that, for many patients, leads to legal blindness,” said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. “There are currently no available treatments for ADOA. We look forward to partnering with the ADOA community and clinicians to learn more about this disease as we work to develop the first potential medicine to treat the underlying cause of ADOA.”
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