Stoke Therapeutics

To address the genetic cause of severe diseases by becoming the leading company in treating them.

Stoke Therapeutics SWOT Analysis

Updated: October 6, 2025 • 2025-Q4 Analysis

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This analysis for Stoke Therapeutics was created using Alignment.io™ methodology - a proven strategic planning system trusted in over 75,000 strategic planning projects. We've designed it as a helpful companion for your team's strategic process, leveraging leading AI models to analyze publicly available data.

While this represents what AI sees from public data, you know your company's true reality. That's why we recommend using Alignment.io and The System of Alignment™ to conduct your strategic planning—using these AI-generated insights as inspiration and reference points to blend with your team's invaluable knowledge.

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The Stoke Therapeutics SWOT analysis reveals a company at a critical inflection point. Its primary strength lies in the promising clinical data for STK-001 and the underlying TANGO platform, bolstered by a strong cash position and an expert leadership team. However, this is counterbalanced by a significant weakness: an existential dependency on STK-001's success. The market opportunity to expand the TANGO platform is immense, but the external threat from rapidly advancing gene therapies and potential regulatory hurdles is substantial. The core challenge is one of execution. Stoke must flawlessly complete its pivotal trials for STK-001 while simultaneously demonstrating the broader applicability of its science to de-risk the company's future and justify its long-term vision. The path to becoming a leader in genetic medicine is clear but narrow, demanding immense focus and operational excellence.

Ionis Pharmaceuticals Alnylam Pharmaceuticals Sarepta Therapeutics Biogen Vertex Pharmaceuticals

No past cycles

Mission & Vision SWOT Analysis OKR Plan Retrospective AI Strategy Data Acquisition

To address the genetic cause of severe diseases by becoming the leading company in treating them.

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Strengths

PIPELINE: STK-001 shows promising seizure reduction in Dravet studies
PLATFORM: TANGO technology validated by Acadia partnership ($40M upfront)
FINANCES: Strong cash position provides runway through key clinical data
LEADERSHIP: Experienced team with a track record in rare disease drug dev
SAFETY: Favorable safety profile for STK-001 observed in ongoing trials

Weaknesses

CONCENTRATION: Heavy reliance on STK-001 success for near-term valuation
ENROLLMENT: Slower than expected patient enrollment in pivotal studies
COMPETITION: Fintepla (fenfluramine) is an approved, effective therapy
DELIVERY: Intrathecal delivery is invasive and requires specialization
DATA: Long-term efficacy and safety data for TANGO is not yet available

Opportunities

EXPANSION: Positive STK-001 data could validate TANGO for other diseases
LABEL: Potential for best-in-class label including cognitive benefits
AWARENESS: Growing patient and physician awareness of genetic testing
INDICATION: Explore STK-001 for other SCN1A-related epilepsies
PARTNERING: Non-dilutive funding via partnerships for pipeline assets

Threats

REGULATORY: FDA may require more extensive data, delaying potential approval
COMPETITORS: Gene therapies in development could offer one-time cures
MARKET: Biotech market volatility impacts access to future capital needs
PAYER: Pushback on high-cost therapies without overwhelming efficacy data
TRIAL: Risk of trial failure or unexpected safety signals in later stages

Key Priorities

EXECUTION: Flawlessly execute STK-001 pivotal trials to ensure approval
VALIDATION: Leverage STK-001 data to validate and advance the TANGO platform
PIPELINE: Accelerate STK-002 and other preclinical assets into the clinic
CAPITAL: Prudently manage cash runway while exploring strategic partnerships

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Stoke Therapeutics OKR

Updated: October 6, 2025 • 2025-Q4 Analysis

This Stoke Therapeutics OKR plan is sharp and focused, correctly prioritizing what matters most: clinical execution. The objectives—CLINICAL MASTERY, PLATFORM PROMISE, PIPELINE ACCELERATION, and STRATEGIC CAPITAL—are not just goals; they are a clear roadmap derived directly from the SWOT's core challenges. The key results are specific, outcome-oriented, and ambitious, pushing the team beyond mere activity. This plan effectively translates high-level strategy into tangible, measurable actions that directly address the company's primary risks and opportunities. If achieved, these OKRs will not only advance STK-001 but will fundamentally de-risk the entire TANGO platform and secure the company’s financial future, paving the way for market leadership.

Ionis Pharmaceuticals Alnylam Pharmaceuticals Sarepta Therapeutics Biogen Vertex Pharmaceuticals

No past cycles

Mission & Vision SWOT Analysis OKR Plan Retrospective AI Strategy Data Acquisition

To address the genetic cause of severe diseases by becoming the leading company in treating them.

CLINICAL MASTERY

Flawlessly execute STK-001 trials to secure approval.

DATABASE: Achieve 100% database lock for the Phase 3 pivotal study on schedule, with zero critical data errors.
SUBMISSION: File the New Drug Application (NDA) with the FDA within 60 days of receiving final top-line data.
ENROLLMENT: Complete enrollment for the long-term extension study to ensure a robust long-term safety dataset.
PUBLISH: Present primary efficacy and safety data from the pivotal program at a major international neurology congress.

PLATFORM PROMISE

Prove TANGO's value beyond a single drug.

VALIDATION: Publish a peer-reviewed paper linking STK-001 outcomes to target engagement and protein upregulation.
DESIGN: Utilize clinical insights and AI modeling to finalize the design of three next-generation ASO candidates.
BIOMARKER: Identify and validate a novel biomarker that correlates with clinical response in Dravet syndrome.
PARTNER: Secure a new strategic partnership for a non-core TANGO asset, validating external interest in the platform.

PIPELINE ACCELERATION

Advance the next wave of genetic medicines.

INITIATE: Dose the first patient in the Phase 1/2 clinical trial for STK-002 for AUTD, our second program.
NOMINATE: Officially declare a development candidate for our lead Ocular disease program from preclinical research.
EXPAND: Identify and validate three new CNS targets for the TANGO platform with strong biological and commercial rationale.
MANUFACTURE: Secure clinical trial manufacturing supply for the STK-002 program through Phase 2 studies.

STRATEGIC CAPITAL

Ensure financial strength through key milestones.

RUNWAY: Maintain a cash runway sufficient to fund operations for at least 24 months at all times during the year.
BUDGET: Deliver the annual operating plan with less than a 5% variance between actual and budgeted spend.
MODEL: Build a comprehensive commercial launch and pricing model for STK-001 to support future financing efforts.
DILIGENCE: Establish a data room and maintain a state of readiness for potential strategic partnership or financing.

METRICS

Patient Enrollment Rate
Cash Runway (Months)
Pipeline Candidates Advanced

VALUES

Commitment to Patients
Scientific Rigor
Urgency and Perseverance
Collaboration
Integrity

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Stoke Therapeutics Retrospective

AI-Powered Insights

Claude

Gemini

GPT

Stoke Therapeutics Q3 2024 Earnings Report and 10-Q filing.
Stoke Therapeutics Corporate Presentation, November 2024.
Press releases from Stoke Therapeutics website (2023-2024).
Analysis of competitors' pipelines in Dravet syndrome and ASO tech.
Review of executive biographies from company website and LinkedIn.

Ionis Pharmaceuticals Alnylam Pharmaceuticals Sarepta Therapeutics Biogen Vertex Pharmaceuticals

No past cycles

Mission & Vision SWOT Analysis OKR Plan Retrospective AI Strategy Data Acquisition

To address the genetic cause of severe diseases by becoming the leading company in treating them.

Toggle Theme

What Went Well

PROGRESS: Positive long-term safety and efficacy data reported for STK-001
ENROLLMENT: Met enrollment targets for key cohorts in the Phase 3 study
PARTNERSHIP: Continued execution on the collaboration agreement with Acadia
FINANCES: Maintained a strong cash position, extending operational runway
PIPELINE: Advanced preclinical programs in ocular and CNS indications

Not So Well

TIMELINES: Overall study completion timelines remain extended and lengthy
SPEND: R&D expenses continue to increase as pivotal trials advance
VALUATION: Stock performance remains volatile, sensitive to market trends
COMMUNICATION: Perception of slow progress compared to faster-moving modalities
DEPENDENCY: Narrative remains heavily focused on a single lead asset

Learnings

EXECUTION: Long-term studies require immense operational discipline to manage
INVESTORS: The market requires consistent data flow to maintain confidence
PLATFORM: Early data provides crucial insights for next-gen ASO designs
PATIENTS: High unmet need drives strong patient retention in extension studies
REGULATORY: Early and frequent interaction with agencies is paramount

Action Items

DATA: Prepare for timely top-line data readout from pivotal studies
FINANCE: Continue prudent cash management to fund operations to approval
PIPELINE: Finalize plans to move STK-002 into clinical development
COMMERCIAL: Begin building foundational pre-commercialization strategy
PARTNER: Evaluate opportunities for ex-US partnerships for STK-001

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Organization	SWOT Analysis	OKR Plan	Top 6	Retrospective

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Stoke Therapeutics Market

AI-Powered Insights

Claude

Gemini

GPT

Stoke Therapeutics Q3 2024 Earnings Report and 10-Q filing.
Stoke Therapeutics Corporate Presentation, November 2024.
Press releases from Stoke Therapeutics website (2023-2024).
Analysis of competitors' pipelines in Dravet syndrome and ASO tech.
Review of executive biographies from company website and LinkedIn.

Founded: 2014
Market Share: 0% (Clinical Stage)
Customer Base: Patients with severe genetic diseases (e.g., Dravet)
Category:
Biotechnology
SIC Code: 2836
NAICS Code: 541714 Research and Development in Biotechnology (except Nanobiotechnology)
Location: Bedford, Massachusetts
Zip Code: 01730
Congressional District: MA-6 LYNN
Employees: 150

Competitors

Ionis Pharmaceuticals View Analysis

Alnylam Pharmaceuticals View Analysis

Sarepta Therapeutics View Analysis

Biogen View Analysis

Vertex Pharmaceuticals Request Analysis

Products & Services

No products or services data available

Distribution Channels

N/A (Direct via clinical trial sites)

Stoke Therapeutics Business Model Analysis

AI-Powered Insights

Claude

Gemini

GPT

Stoke Therapeutics Q3 2024 Earnings Report and 10-Q filing.
Stoke Therapeutics Corporate Presentation, November 2024.
Press releases from Stoke Therapeutics website (2023-2024).
Analysis of competitors' pipelines in Dravet syndrome and ASO tech.
Review of executive biographies from company website and LinkedIn.

Problem

Severe genetic diseases lack effective treatments
Haploinsufficiency is a common disease cause
Existing drugs only address disease symptoms

Solution

TANGO platform to upregulate protein expression
ASO medicines targeting the genetic root cause
Disease-modifying therapies for CNS/ocular

Key Metrics

Clinical trial enrollment and completion rates
Regulatory submission and approval milestones
Cash runway and operational burn rate

Unique

Novel mechanism restoring protein from healthy gene
Focus on haploinsufficiencies, a broad target class
Potential for cognitive benefits beyond symptoms

Advantage

Lead asset STK-001 with years of clinical data
Strong IP portfolio around TANGO platform
Deep expertise in oligonucleotide drug development

Channels

Clinical trial sites and investigators
Scientific publications and conferences
Patient advocacy group partnerships

Customer Segments

Patients with Dravet syndrome & AUTD
Neurologists and genetic disease specialists
Future: Patients with other genetic diseases

Costs

High R&D expenses for clinical trials
Personnel costs for specialized talent
Contract manufacturing (CMO) expenses

Stoke Therapeutics Product Market Fit Analysis

Updated: October 6, 2025

Stoke Therapeutics pioneers a new class of medicine to treat the underlying cause of severe genetic diseases. By precisely upregulating protein expression, its platform offers the potential to not only manage symptoms like seizures but also improve cognition and quality of life for patients and their families, offering hope where there was none.

DISEASE MODIFICATION: Addressing the root genetic cause of Dravet.

BEYOND SEIZURES: Potential for cognitive and behavioral improvements.

SUSTAINED EFFICACY: A simple, repeatable dosing regimen for long-term benefit.

Before State

Uncontrolled, life-threatening seizures
Significant developmental delays daily
Constant fear for caregivers and family

After State

Reduced seizure frequency and severity
Potential for cognitive, motor gains
Hope for a more independent future

Negative Impacts

High risk of SUDEP (sudden death)
Poor quality of life for patient/family
Lifelong dependency and high care costs

Positive Outcomes

Improved patient survival and development
Reduced caregiver burden and anxiety
Lower long-term healthcare utilization

Key Metrics

Patient Retention Rates

>90% in open-label extension

Net Promoter Score (NPS)

N/A (Qualitative patient feedback)

User Growth Rate

Measured by clinical trial enrollment rate

Customer Feedback/Reviews

Positive feedback from advocacy groups

Repeat Purchase Rates

N/A (Long-term dosing in trials)

Requirements

Confirmed SCN1A gene mutation diagnosis
Enrollment in a clinical trial site
Commitment to long-term treatment

Why Stoke Therapeutics

Targeted antisense oligonucleotide (ASO)
Intrathecal administration every few months
Restore protein via TANGO technology

Stoke Therapeutics Competitive Advantage

First disease-modifying approach for Dravet
Potential for benefits beyond seizure control
Simple, repeatable dosing regimen

Proof Points

Observed seizure reduction in Phase 1/2a
Cognitive improvements in some patients
Strong safety profile in ongoing studies

Stoke Therapeutics Market Positioning

AI-Powered Insights

Claude

Gemini

GPT

Stoke Therapeutics Q3 2024 Earnings Report and 10-Q filing.
Stoke Therapeutics Corporate Presentation, November 2024.
Press releases from Stoke Therapeutics website (2023-2024).
Analysis of competitors' pipelines in Dravet syndrome and ASO tech.
Review of executive biographies from company website and LinkedIn.

Strategic pillars derived from our vision-focused SWOT analysis

TANGO PLATFORM

Dominate RNA modulation for genetic upregulation

DRAVET LEADERSHIP

Secure STK-001 as the standard of care

PIPELINE EXPANSION

Systematically target new CNS & ocular diseases

OPERATIONAL EXCELLENCE

Build scalable clinical & manufacturing capabilities

What You Do

Develops RNA-based medicines to upregulate proteins.

Target Market

Patients with severe, rare genetic diseases.

Differentiation

Proprietary TANGO platform targets RNA splicing.
Focus on restoring normal protein levels, not replacing.

Revenue Streams

Collaboration revenue (Acadia)
Future product sales upon approval

Stoke Therapeutics Operations and Technology

AI-Powered Insights

Claude

Gemini

GPT

Stoke Therapeutics Q3 2024 Earnings Report and 10-Q filing.
Stoke Therapeutics Corporate Presentation, November 2024.
Press releases from Stoke Therapeutics website (2023-2024).
Analysis of competitors' pipelines in Dravet syndrome and ASO tech.
Review of executive biographies from company website and LinkedIn.

Company Operations

Organizational Structure: Functional hierarchy with project-based clinical teams.
Supply Chain: Relies on CMOs for oligonucleotide manufacturing.
Tech Patents: Portfolio covers TANGO platform and specific ASOs.
Website: https://www.stoketherapeutics.com/

Top Clients

Acadia Pharmaceuticals (Partner)

Board Members

Stoke Therapeutics Competitive Forces

Threat of New Entry

Moderate. High R&D costs and IP are significant barriers, but novel platforms for genetic medicine are continually emerging from academia and startups.

Supplier Power

Moderate to High. Specialized CMOs for clinical-grade oligonucleotides are limited, giving them pricing leverage and creating potential bottlenecks.

Buyer Power

Low (Patients/Physicians), High (Payers). Patients have few options, but payers (insurers) will have significant power to negotiate prices on high-cost therapies.

Threat of Substitution

High. Gene therapies from competitors like Encoded Therapeutics or Sarepta could offer a one-time curative treatment, substituting Stoke's chronic therapy.

Competitive Rivalry

High. While few direct TANGO competitors exist, the broader genetic medicine space (gene therapy, ASOs) is intense with well-funded players.

Analysis of AI Strategy

Updated: October 6, 2025 • 2025-Q4 Analysis

The Stoke Therapeutics AI SWOT analysis underscores a significant untapped opportunity. The company possesses a core strength in its unique, proprietary genetic and clinical datasets—the essential fuel for any meaningful AI initiative. However, this is starkly contrasted by its primary weakness: a lack of internal AI talent and infrastructure. The opportunity to leverage AI for target discovery and ASO design is a potential game-changer, capable of dramatically accelerating its pipeline and solidifying its leadership. Conversely, the threat of more AI-savvy competitors outpacing them is very real. Stoke doesn't need to become an AI company, but it must urgently become an AI-enabled biotech. The strategic imperative is to partner smartly and build targeted capabilities to transform its data asset into a durable competitive advantage, starting with trial recruitment and drug design.

Ionis Pharmaceuticals Alnylam Pharmaceuticals Sarepta Therapeutics Biogen Vertex Pharmaceuticals

No past cycles

Mission & Vision SWOT Analysis OKR Plan Retrospective AI Strategy Data Acquisition

To address the genetic cause of severe diseases by becoming the leading company in treating them.

Strengths

DATA: Proprietary clinical and genetic data from trials to train models
TARGETS: Well-defined genetic targets are ideal for computational analysis
CHEMISTRY: Existing oligonucleotide chemistry data for predictive modeling
FOCUS: Narrow therapeutic focus simplifies data collection and modeling

Weaknesses

TALENT: Lack of a dedicated, in-house AI/ML research and engineering team
INFRASTRUCTURE: No scaled computational infrastructure for large model training
INTEGRATION: Siloed data across clinical, preclinical, and CMC functions
STRATEGY: No publicly articulated, cohesive AI strategy to guide investment

Opportunities

DISCOVERY: AI-driven identification of new TANGO-addressable disease targets
OPTIMIZATION: Machine learning to design more potent and safer ASO sequences
ENROLLMENT: AI tools to identify and recruit eligible patients for trials
BIOMARKERS: Discovering novel digital or fluidic biomarkers from trial data

Threats

COMPETITION: Larger biopharma leveraging AI to accelerate their own pipelines
LEADERSHIP: Competitors may identify and pursue Stoke's targets faster with AI
PRIVACY: Risk of data breaches with sensitive patient genetic information
RELIANCE: Over-reliance on black-box AI models without biological validation

Key Priorities

DESIGN: Implement AI to optimize the design of next-generation ASO candidates
IDENTIFY: Use machine learning to rapidly identify new genetic disease targets
RECRUITMENT: Deploy AI platforms to accelerate patient identification/enrollment
PARTNER: Collaborate with AI-native drug discovery firms to leapfrog capabilities

Create professional SWOT analyses in minutes with our AI template. Get insights that drive real results.

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Stoke Therapeutics Financial Performance

AI-Powered Insights

Claude

Gemini

GPT

Stoke Therapeutics Q3 2024 Earnings Report and 10-Q filing.
Stoke Therapeutics Corporate Presentation, November 2024.
Press releases from Stoke Therapeutics website (2023-2024).
Analysis of competitors' pipelines in Dravet syndrome and ASO tech.
Review of executive biographies from company website and LinkedIn.

Profit: -$175.4 Million (Net Loss, TTM)

Market Cap: $350 Million

Stock Performance

Annual Report: SEC EDGAR Database

Debt: $10.2 Million (Total Debt)

ROI Impact: Metrics are pre-commercial; focus is on clinical value.

SWOT Index

Composite strategic assessment with 10-year outlook

/ 100

Focused Challenger

ICM Index

STRATEGIC ADVISOR ASSESSMENT

Stoke's score reflects a high-ambition mission (societal impact) constrained by clinical-stage achievability risks. Its SWOT is balanced, with significant platform upside offset by single-asset dependency and competitive threats. The OKRs are strong and execution-focused. High AI potential exists but is unrealized. The company is a focused challenger with a clear but high-risk path to transforming a specific segment of genetic medicine. Success hinges entirely on STK-001 trial outcomes, creating a binary risk profile.

SWOT Factors

Upside: 74.0 Risk: 78.0

OKR Impact

AI Leverage

Top 3 Strategic Levers

Flawless execution of STK-001 pivotal trials to de-risk platform

Accelerate STK-002 into the clinic to diversify pipeline

Leverage AI for ASO design and new target identification

AI Disclosure

This report was created using the Alignment Method—our proprietary process for guiding AI to reveal how it interprets your business and industry. These insights are for informational purposes only and do not constitute financial, legal, tax, or investment advice.

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