Stoke Therapeutics to Present at the Cantor Global Healthcare Conference
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Stoke Therapeutics to Present at the Cantor Global Healthcare Conference
A live webcast and archived replay of CEO Ian F. Smith and Chief Patient Officer Jason Hoitt’s Cantor conference talk will be available on Stoke’s website.
BEDFORD, Mass.--(BUSINESS WIRE)--
Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine and has a lead investigational medicine, zorevunersen, in development as a first-in-class potential disease-modifying treatment for Dravet syndrome. Today, the Company announced that Chief Executive Officer Ian F. Smith and Chief Patient Officer Jason Hoitt will present at the Cantor Global Healthcare Conference on Wednesday, September 9, 2026 at 1:00 p.m. ET.
A live webcast of the presentation, along with an archived replay, will be available in the Investors & News section of Stoke’s website at https://investor.stoketherapeutics.com/.
About Stoke Therapeutics
Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine. Using Stoke’s proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke’s first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study. Stoke’s initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for Stoke’s proprietary approach. Stoke is headquartered in Bedford, Massachusetts. For more information, visit https://www.stoketherapeutics.com/ and follow us on LinkedIn.
About Zorevunersen
Zorevunersen is an investigational antisense oligonucleotide that is designed to treat the underlying cause of Dravet syndrome by increasing functional NaV1.1 protein production in brain cells from the unaffected (wild-type) copy of the SCN1A gene. This highly differentiated mechanism of action aims to reduce seizure frequency beyond what has been achieved with anti-seizure medicines and to improve neurodevelopment, cognition and behavior. Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation not associated with gain-of-function in the SCN1A gene, and China’s Center for Drug Evaluation has granted zorevunersen Breakthrough Therapy Designation. Stoke has a strategic collaboration with Biogen (Nasdaq: BIIB) to develop and commercialize zorevunersen for Dravet syndrome. Under the collaboration, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights. Zorevunersen is currently in clinical development, and its safety and efficacy have not been evaluated by any regulatory authority.
Antisense oligonucleotides are short, synthetic pieces of genetic material designed to stick to specific messenger RNA molecules inside cells and block or alter the production of a particular protein. For investors, they matter because they represent a targeted drug approach with potential to treat diseases that other medicines cannot, but they also carry development, safety and regulatory risks that can lead to large swings in a biotech company’s value.
haploinsufficiencymedical
Haploinsufficiency is a genetic situation in which a person has only one working copy of a gene and that single copy does not produce enough of the gene’s product for normal function, so loss or mutation of the other copy causes disease or altered biology. For investors, it matters because haploinsufficiency can determine whether a mutation drives a disorder, affect the size of target patient populations, and influence drug target validation, clinical trial design, and regulatory evaluation — like a machine that fails when it loses half its parts.
orphan drug designationregulatory
Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.
breakthrough therapy designationregulatory
A breakthrough therapy designation is a regulatory fast-track given to a drug or treatment that shows early signs of providing a major improvement over existing options for a serious condition. Think of it as a VIP lane that can speed up development and more intensive guidance from regulators, which matters to investors because it can shorten time to market, reduce development risk and potentially increase a company’s value — though it does not guarantee approval.
phase 3medical
Phase 3 is the late-stage clinical testing step for a new drug or medical treatment, where the product is given to large groups of patients to confirm effectiveness, monitor side effects, and compare it to standard care. Successful Phase 3 results are often the final scientific hurdle before regulators decide on approval and market launch—like passing a final exam before graduation—and can sharply change a company's valuation and future revenue prospects.
