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§ Public · Bedford, MA, USA
Stoke Therapeutics is a technology company.
Stoke Therapeutics develops RNA-based medicines to address severe genetic diseases by upregulating protein expression. Their proprietary Targeted Augmentation of Nuclear Gene Output (TANGO) platform increases protein output from healthy genes. This innovative approach targets conditions caused by haploinsufficiencies, aiming to restore essential protein levels and mitigate disease.
The company was co-founded in 2014 by Professor Adrian Krainer, Ph.D., and Isabel Aznarez, Ph.D. Their vision stemmed from groundbreaking research into pre-mRNA splicing, identifying an opportunity to develop precision medicines. This insight allowed them to leverage RNA biology, modulating gene expression to treat genetic diseases and establish a new therapeutic paradigm.
Stoke Therapeutics targets patients with severe genetic conditions, including Dravet Syndrome, Autosomal Dominant Optic Atrophy (ADOA), and SYNGAP1, where disease-modifying treatments are critically needed. The company commits to developing therapies that significantly improve patient outcomes. Its long-term vision is to broadly apply the TANGO platform to restore genetic health for individuals with protein-deficient diseases.
Stoke Therapeutics has raised $130.0M across 2 funding rounds.
Stoke Therapeutics has raised $130.0M in total across 2 funding rounds.
Stoke Therapeutics has raised $130.0M in total across 2 funding rounds.
Stoke Therapeutics's investors include RTW Investments, Alexandria Venture Investments, Apple Tree Partners, Bihua Chen, Janus Henderson Investors, Perceptive Advisors, Matthew Hammond, Redmile Group, Sphera Funds Management, Huw Nash.
Stoke Therapeutics has raised $130.0M across 2 funding rounds. Most recently, it raised $90.0M Series B in October 2018.
| Date | Round | Lead Investors | Other Investors | Status |
|---|---|---|---|---|
| Oct 1, 2018 | $90M Series B | RTW Investments | Alexandria Venture Investments, Apple Tree Partners, Bihua Chen, Janus Henderson Investors, Perceptive Advisors, Matthew Hammond, Redmile Group, Sphera Funds Management | Announced |
| Jan 4, 2018 | $40M Series A | HUW Nash | — | Announced |
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]