| Valuation method | Value, $ | Upside, % |
|---|---|---|
| Artificial intelligence (AI) | 49.21 | 62 |
| Intrinsic value (DCF) | 2034.23 | 6605 |
| Graham-Dodd Method | n/a | |
| Graham Formula | n/a |
Stoke Therapeutics, Inc. (NASDAQ: STOK) is an innovative biopharmaceutical company pioneering antisense oligonucleotide (ASO) therapies to address severe genetic diseases. Leveraging its proprietary Targeted Augmentation of Nuclear Gene Output (TANGO) platform, Stoke designs ASOs to precisely upregulate protein expression, targeting the root causes of rare genetic disorders. The company’s lead candidate, STK-001, is in Phase I/IIa trials for Dravet syndrome, a debilitating form of epilepsy, while STK-002 is in preclinical development for autosomal dominant optic atrophy. Stoke has also partnered with Acadia Pharmaceuticals to advance RNA-based treatments for neurodevelopmental diseases. Headquartered in Bedford, Massachusetts, Stoke represents a high-potential player in the gene modulation space, combining cutting-edge science with a focus on unmet medical needs in neurology and rare diseases.
Stoke Therapeutics presents a high-risk, high-reward opportunity for investors focused on genetic medicine innovation. The company’s TANGO platform and ASO pipeline offer compelling science, but clinical and regulatory risks remain significant given its early-stage assets. Financially, Stoke operates at a loss (net income: -$88.98M in latest reporting) with substantial R&D burn, though its $128M cash position provides near-term runway. The collaboration with Acadia Pharmaceuticals mitigates some development risk. Investors should monitor STK-001’s clinical progress closely—positive data could drive upside, while setbacks may pressure the stock given its speculative valuation (market cap: ~$511M). The 1.198 beta suggests higher volatility than the broader market.
Stoke Therapeutics competes in the niche but rapidly growing ASO and genetic medicine sector, differentiated by its TANGO platform’s ability to upregulate (rather than suppress) protein expression—a key distinction from most ASO approaches. While Ionis Pharmaceuticals and Sarepta Therapeutics dominate the ASO space with commercialized products, Stoke’s focus on protein upregulation for haploinsufficiency disorders (like Dravet syndrome) provides a unique niche. However, it faces competition from gene therapy (e.g., Neurocrine Biosciences) and small-molecule (e.g., Zogenix, now part of UCB) approaches targeting similar indications. Stoke’s partnership with Acadia strengthens its CNS-focused pipeline but doesn’t eliminate the risk of larger biotechs with deeper resources (e.g., Biogen, Roche) entering the space. The company’s modest market cap reflects its early stage; success with STK-001 could position it as an acquisition target, while clinical failures would leave it vulnerable to cash constraints given its limited diversified pipeline.