Stoke Therapeutics is a technology company.
Stoke Therapeutics has raised $90.0M across 1 funding round.
Stoke Therapeutics has raised $90.0M in total across 1 funding round.
Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company developing precision medicines that upregulate protein expression to treat severe genetic diseases, particularly autosomal dominant haploinsufficiencies where one mutated gene copy fails to produce essential proteins.[1][2][4] Its proprietary TANGO platform uses antisense oligonucleotides (ASOs) to increase output from healthy genes, targeting conditions like Dravet syndrome—a severe childhood epilepsy—with lead candidate zorevunersen (STK-001) in Phase 3 trials, and STK-002 for autosomal dominant optic atrophy (ADOA) in Phase 1.[2][4][6] The company serves patients with rare genetic epilepsies and neurological disorders, addressing unmet needs by restoring protein levels to near-normal without altering DNA.[3][4] Growth momentum includes dosing the first Phase 3 patient in August 2025 for zorevunersen, showing sustained seizure reductions and developmental gains over 36 months, alongside pipeline expansion into SYNGAP1 and other disorders.[6]
Stoke Therapeutics was founded in June 2014 as an early-stage biotechnology company, sparked by unique insights in RNA biology to increase protein output from healthy genes—a novel approach at the time.[4][7] The idea emerged from pioneering work on the TANGO (Targeted Augmentation of Nuclear Gene Output) platform, which modulates pre-mRNA splicing via ASOs to "stoke" expression from non-mutated gene copies, compensating for haploinsufficiencies.[1][4][7] Early traction built through rapid advancement of STK-001 into clinical proof-of-concept for Dravet syndrome, with pivotal moments including ongoing Phase 3 EMPEROR trial initiation and promising long-term data on seizure reduction and neurodevelopmental improvements.[6][7] Headquartered in Bedford, Massachusetts, with offices in Cambridge, the company has evolved from platform discovery to multiple clinical programs, driven by a team committed to RNA-based innovation for genetic diseases.[2][4]
Stoke rides the wave of RNA therapeutics and precision genetic medicine, capitalizing on advances in ASO technology to address haploinsufficiencies—a subset of over 10,000 rare monogenic diseases where 50% protein loss causes pathology.[2][4] Timing aligns with surging demand for disease-modifying treatments amid regulatory support for rare diseases (e.g., FDA incentives) and post-approval successes in RNA platforms like those for spinal muscular atrophy.[3][6] Market forces favoring Stoke include aging populations increasing neurological disorder prevalence, limited competition in upregulation therapies, and biotech funding rebound for high-impact pipelines.[1][3] By validating TANGO across epilepsy, optic atrophy, and beyond, Stoke influences the ecosystem, potentially expanding ASO applications and inspiring platforms for protein restoration in broader genetic disorders.[4][9]
Stoke stands on the cusp of validation with Phase 3 readouts for zorevunersen potentially unlocking first disease-modifying approval for Dravet syndrome, alongside Phase 1 progress for STK-002 and new indications like SYNGAP1.[6] Trends shaping its path include AI-driven target identification, expanded ASO manufacturing scale, and payer focus on rare disease economics, amplifying TANGO's reach to more haploinsufficiencies.[3][4] Influence may evolve from niche biotech to platform leader, transforming lives in genetic medicine if clinical momentum sustains—echoing its founding vision to "stoke" hope through RNA innovation.[1][4]
Stoke Therapeutics has raised $90.0M in total across 1 funding round.
Stoke Therapeutics's investors include RTW Investments.
Stoke Therapeutics has raised $90.0M across 1 funding round. Most recently, it raised $90.0M Series B in October 2018.
| Date | Round | Lead Investors | Other Investors |
|---|---|---|---|
| Oct 1, 2018 | $90.0M Series B | RTW Investments |