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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
First in-vivo proof-of-concept for TANGO antisense oligonucleotides in an ocular disease Results presented at the American Society of Gene and Cell Therapy
About this update from Stoke Therapeutics, Inc.
[{"type":"text","content":"\nFirst in-vivo proof-of-concept for TANGO antisense oligonucleotides in an ocular disease \n\n\nResults presented at the American Society of Gene and Cell Therapy Annual Meeting further validate the company’s mutation-independent approach to amplifying protein expression to treat severe genetic diseases\n\n BEDFORD, Mass.--(BUSINESS WIRE)--\nStoke 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. This is the first proof-of-concept data for TANGO antisense oligonucleotides (ASOs) in an ocular disease. The results further validate the company’s mutation-independent approach to amplifying protein expression to treat severe genetic diseases. These data will be presented today in a virtual poster session at the American Society of Gene and Cell Therapy (ASGCT) 2020 Annual Meeting.\n\n\n“These data provide early evidence of the potential to address the underlying cause of autosomal dominant optic atrophy, an optic nerve disorder that causes progressive and irreversible vision loss starting in the first decade of a child’s life. There are currently no approved treatments for ADOA,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “Our TANGO technology represents a unique, mutation-independent approach to treating the underlying cause of a variety of genetic diseases, particularly in the central nervous system and the eye. The ADOA program is one of several under consideration for future prioritization, and we look forward to nominating a second product candidate later this year.”\n\n\nADOA affects approximately one in 30,000 people globally with a higher incidence in Denmark of one in 10,000 due to a founder effect. An estimated 65% to 90% of cases are caused by mutations in the OPA1 gene.\n\n\nThe data presented today demonstrate in-vitro and in-vivo proof-of-concept for TANGO ASOs in an ocular disease. Highlights from today’s presentation include:\n\n\n\nDose-dependent decreases in non-productive OPA1 mR...