Stoke Therapeutics
↗Bedford, USA (Moving to Waltham, MA in late 2026)
Stoke Therapeutics is a clinical-stage biotechnology company pioneering a new way to treat the underlying causes of severe genetic diseases by upregulating protein expression with RNA-based medicines. The company's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform is designed to restore missing proteins by increasing the amount of mRNA that is translated into protein.
Stoke's approach targets haploinsufficiencies—conditions where one copy of a gene is mutated and the remaining functional copy does not produce enough protein. By using antisense oligonucleotides (ASOs) to modulate RNA splicing, Stoke aims to deliver disease-modifying therapies for conditions with high unmet needs, particularly in the central nervous system and the eye.
CLASSIFICATION
Company Type:Biotech
Therapeutic Areas:
Industry:Biotechnology
Sub-Industry:RNA Therapeutics / Rare Disease
SIZE & FINANCIALS
Employees:51-200
Revenue:$200M-$250M
Founded:2014
Ownership:public
Status:operating
FUNDING
Stage:Post-IPO
Total Raised:$500M+
Investors:Apple Tree Partners, RTW Investments, Redmile Group, Janus Henderson Investors
STOCK
Exchange:NASDAQ
Ticker:STOK
Market Cap:$1.96B
PIPELINE
Stage:Phase 3
Lead Drug Stage:Phase 3 (Zorevunersen for Dravet Syndrome)
Modalities:RNA, Antisense Oligonucleotides (ASO)
Active Trials:4
Trial Phases:Phase 1: 1 | Phase 2: 2 | Phase 3: 1
FDA Approvals:0
EMA Approvals:0
CORPORATE STRUCTURE
Key Partnerships:Biogen (Global collaboration for zorevunersen outside US/Canada/Mexico), Acadia Pharmaceuticals (Discovery/development of RNA-based medicines for CNS targets)
COMPETITION
Position:Leader in RNA-based protein upregulation for haploinsufficiency.
Competitors:Encoded Therapeutics, Jazz Pharmaceuticals, UCB (Fintepla), Ionis Pharmaceuticals
LEADERSHIP
Key Executives:
Ian F. Smith - CEO
Isabel Aznarez - Co-Founder & SVP Research
Barry Ticho - Chief Medical Officer
Scientific Founders:Adrian Krainer, Ph.D., Isabel Aznarez, Ph.D.
Board Members:Arthur Tzianabos (Chairman), Adrian Krainer, Edward M. Kaye, Garry E. Menzel
LINKS
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