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Stoke Therapeutics Inc (STOK)

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Upturn Advisory Summary
02/26/2026: STOK (3-star) is a STRONG-BUY. BUY since 4 days. Simulated Profits (-1.88%). Updated daily EoD!
1 Year Target Price $41.7
1 Year Target Price $41.7
| 7 | Strong Buy |
| 2 | Buy |
| 1 | Hold |
| 0 | Sell |
| 0 | Strong Sell |
Key Highlights
Company Size Mid-Cap Stock | Market Capitalization 2.03B USD | Price to earnings Ratio 52.21 | 1Y Target Price 41.7 |
Price to earnings Ratio 52.21 | 1Y Target Price 41.7 | ||
Volume (30-day avg) 10 | Beta 1.11 | 52 Weeks Range 5.35 - 38.69 | Updated Date 02/26/2026 |
52 Weeks Range 5.35 - 38.69 | Updated Date 02/26/2026 | ||
Dividends yield (FY) - | Basic EPS (TTM) 0.68 |
Analyzing Revenue: Products, Geography and Growth
Revenue by Products
Earnings Date
Report Date - | When - | Estimate - | Actual - |
Profitability
Profit Margin 19.73% | Operating Margin (TTM) -405.3% |
Management Effectiveness
Return on Assets (TTM) 5.2% | Return on Equity (TTM) 15.02% |
Valuation
Trailing PE 52.21 | Forward PE 204.08 | Enterprise Value 1114659914 | Price to Sales(TTM) 9.86 |
Enterprise Value 1114659914 | Price to Sales(TTM) 9.86 | ||
Enterprise Value to Revenue 5.58 | Enterprise Value to EBITDA 26.37 | Shares Outstanding 57117150 | Shares Floating 41863444 |
Shares Outstanding 57117150 | Shares Floating 41863444 | ||
Percent Insiders 4.06 | Percent Institutions 123.07 |
Upturn AI SWOT
Stoke Therapeutics Inc

Company Overview
History and Background
Stoke Therapeutics Inc. was founded in 2014, emerging from the Broad Institute of MIT and Harvard. The company is focused on developing a new class of medicines to treat severe genetic diseases by precisely increasing the expression of target genes. A significant milestone was its initial public offering (IPO) in July 2019, which provided capital for its research and development efforts. The company has evolved by advancing its lead product candidate for Dravet syndrome and expanding its pipeline to other genetic epilepsies and diseases.
Core Business Areas
- Gene Expression Modulation: Stoke Therapeutics focuses on developing small molecule drugs designed to upregulate the expression of specific genes. This approach aims to correct the underlying cause of genetic diseases by increasing the amount of functional protein produced by the body. Their platform utilizes proprietary technology to identify and synthesize these molecules.
Leadership and Structure
Stoke Therapeutics operates with a dedicated leadership team comprising experienced professionals in drug development, biotechnology, and business. The organizational structure is typical of a clinical-stage biopharmaceutical company, with departments focused on research and development, clinical operations, regulatory affairs, manufacturing, and commercialization.
Top Products and Market Share
Key Offerings
- STK-001: STK-001 is Stoke Therapeutics' lead product candidate, designed to treat Dravet syndrome, a severe form of epilepsy. It works by increasing the expression of the SCN1A gene, which is crucial for normal brain function. STK-001 is currently in clinical development. Specific market share data for STK-001 is not yet applicable as it is an investigational drug. Key competitors in the Dravet syndrome treatment space include companies developing symptomatic treatments like Epidiolex (GW Pharma/Jazz Pharmaceuticals) and various anti-seizure medications. However, STK-001 aims to address the root genetic cause.
Market Dynamics
Industry Overview
The rare genetic disease therapeutics market is characterized by high unmet medical needs, significant scientific innovation, and substantial investment. Companies in this space often focus on gene therapy, enzyme replacement therapy, and novel molecular approaches to address diseases with limited or no existing treatment options. The regulatory pathway can be complex but often offers incentives for orphan drug development.
Positioning
Stoke Therapeutics is positioned as an innovator in the field of gene expression modulation for genetic diseases. Its proprietary platform and focus on increasing protein expression represent a differentiated approach compared to some gene editing or gene replacement therapies. The company's strength lies in its scientific foundation and its targeted approach to specific genetic disorders.
Total Addressable Market (TAM)
The TAM for genetic diseases is vast and growing as understanding of genetic underpinnings expands. For Dravet syndrome alone, the TAM is estimated in the hundreds of millions to low billions of dollars annually, depending on patient population estimates and pricing strategies for novel therapies. Stoke Therapeutics is positioned to address a significant portion of this market with its potential disease-modifying therapy.
Upturn SWOT Analysis
Strengths
- Proprietary gene expression modulation platform
- Focus on a novel therapeutic approach for genetic diseases
- Strong scientific foundation from the Broad Institute
- Lead candidate STK-001 targeting a significant unmet need (Dravet syndrome)
Weaknesses
- Clinical-stage company with no approved products
- High dependence on the success of STK-001 and pipeline candidates
- Significant R&D expenses without current revenue
- Need for substantial future funding
Opportunities
- Expansion of pipeline to other genetic diseases with similar mechanisms
- Potential for partnerships with larger pharmaceutical companies
- Advancements in genetic diagnostics leading to earlier identification of patients
- Favorable regulatory pathways for orphan drugs
Threats
- Clinical trial failures or delays
- Competition from other novel therapeutic modalities (e.g., gene editing, ASOs)
- Challenges in manufacturing and scaling up production
- Reimbursement challenges for high-cost novel therapies
Competitors and Market Share
Key Competitors
- GW Pharmaceuticals plc (now part of Jazz Pharmaceuticals) (GWPH)
- BioMarin Pharmaceutical Inc. (BMRN)
- Sarepta Therapeutics, Inc. (SRPT)
- uniQure N.V. (QURE)
- Geneuro SA (private)
Competitive Landscape
Stoke Therapeutics' advantage lies in its unique approach to gene expression modulation, which differs from gene replacement or gene editing. However, competitors are well-established in the rare disease space with approved therapies and significant clinical development programs. Stoke's ability to demonstrate efficacy and safety in STK-001 will be critical to gaining a competitive foothold. Other companies are exploring similar genetic targets or different modalities to treat the same or related diseases.
Growth Trajectory and Initiatives
Historical Growth: Stoke Therapeutics' historical growth has been characterized by the expansion of its scientific team, the advancement of its proprietary platform, and the progression of its lead candidate, STK-001, through preclinical and early clinical stages. This growth has been fueled by significant venture capital and public market financing.
Future Projections: Future growth projections are contingent on the successful completion of clinical trials for STK-001 and other pipeline assets, leading to regulatory approval and commercialization. Analyst estimates would focus on potential peak sales for approved products and the expansion of the company's therapeutic reach into other genetic diseases.
Recent Initiatives: Recent initiatives likely include the continued enrollment and execution of clinical trials, ongoing research into new therapeutic targets, and potential business development activities to expand its portfolio or secure strategic partnerships.
Summary
Stoke Therapeutics Inc. is a clinical-stage biopharmaceutical company with a promising novel approach to treating severe genetic diseases by modulating gene expression. Its lead candidate, STK-001 for Dravet syndrome, shows early potential. The company's strengths lie in its innovative platform and scientific backing, but it faces significant risks inherent to drug development, including clinical trial success and future funding needs. Continued progress in clinical trials and strategic pipeline expansion are key to its future success, while competition and regulatory hurdles pose ongoing threats.
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Sources and Disclaimers
Data Sources:
- Company filings with the U.S. Securities and Exchange Commission (SEC)
- Industry analysis reports
- Financial news and data providers
- Biotechnology research databases
Disclaimers:
This analysis is for informational purposes only and does not constitute financial advice. Stock market investments are subject to risks, and investors should conduct their own due diligence before making any investment decisions. The information provided is based on publicly available data and industry knowledge, which can change rapidly. Market share data and TAM are estimates and may not be precise.
AI Summarization is directionally correct and might not be accurate.
Summarized information shown could be a few years old and not current.
Fundamental Rating based on AI could be based on old data.
AI-generated summaries may have inaccuracies (hallucinations). Please verify the information before taking action.
About Stoke Therapeutics Inc
Exchange NASDAQ | Headquaters Bedford, MA, United States | ||
IPO Launch date 2019-06-19 | CEO & Director Mr. Ian F. Smith A.C.A., C.P.A. | ||
Sector Healthcare | Industry Biotechnology | Full time employees 128 | |
Full time employees 128 | |||
Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, engages in the development of treatments for severe genetic diseases by upregulating protein expression. The company utilizes its proprietary Targeted Augmentation of Nuclear Gene Output (TANGO) approach in developing antisense oligonucleotides (ASOs) to selectively restore protein levels. Its lead product candidates include STK-002, which is in preclinical stage for the treatment of autosomal dominant optic atrophy; and Zorevunersen (STK-001), an investigational new medicine for the treatment of Dravet syndrome is being evaluated in phase I/II clinical trials. The company also develops programs focused on various targets, including haploinsufficiency diseases of the central nervous system and eye. It has a license and collaboration with Biogen Inc. for the development and commercialization of zorevunersen medicine for the treatment of Dravet syndrome; and Acadia Pharmaceuticals Inc. for the discovery, development, and commercialization of novel RNA-based medicines for the treatment of genetic neurodevelopmental diseases. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was incorporated in 2014 and is headquartered in Bedford, Massachusetts.

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