Stoke Therapeutics, Inc. (NASDAQ: STOK)

Stoke Therapeutics, Inc. is a late-stage clinical company dedicated to addressing the underlying causes of severe diseases by upregulating protein expression with RNA-based medicines. The company uses its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach to develop antisense oligonucleotides (ASOs) that selectively restore protein levels. Its primary focus is on genetic diseases of the central nervous system and the eye, with lead programs targeting haploinsufficiency disorders.

Stoke Therapeutics generates revenue through upfront payments, milestone payments, and royalties from collaboration agreements with biopharmaceutical partners. The company does not currently have any commercialized products or direct product sales revenue. Revenue is derived from licensing and development partnerships, including agreements with Acadia Pharmaceuticals and Biogen, which provide upfront payments, potential milestone payments, and tiered royalties on future net sales of licensed products.

The company operates through the following segments: TANGO platform development and clinical programs.

• TANGO platform development: This segment encompasses the company's proprietary RNA therapeutics platform designed to increase protein expression from healthy genes to compensate for mutated, non-functioning copies. The TANGO approach uses antisense oligonucleotides to modulate pre-mRNA splicing and prevent non-productive alternative splicing, thereby increasing productive mRNA and protein levels. The platform is applied across multiple disease areas, including central nervous system and eye disorders, and is supported by proprietary bioinformatics algorithms and in-house expertise in whole-transcriptome RNA sequencing.

• Clinical programs: This segment includes the advancement of product candidates through preclinical and clinical development. The lead candidate, zorevunersen (STK-001), is in global Phase 3 development for Dravet syndrome under the EMPEROR study. The second candidate, STK-002, is in Phase 1 development for autosomal dominant optic atrophy (ADOA) via the OSPREY study. Additional preclinical programs target haploinsufficiency diseases of the central nervous system, eye, and heart, leveraging the TANGO mechanism to upregulate protein expression from wild-type alleles.

Stoke Therapeutics occupies a pioneering position in the field of genetic medicines for autosomal dominant haploinsufficiencies, a space with limited competition due to the unique mechanism of its TANGO platform. While other companies develop antisense oligonucleotides or gene therapies, few focus on upregulating protein expression via non-productive splicing correction in haploinsufficiency diseases. The company's competitive advantages include its mutation-independent approach, tissue-specific activity, and ability to address multiple mutations in a single gene with a single drug, supported by strong intellectual property and clinical data from its lead programs.

Stoke Therapeutics serves patients with severe genetic diseases, particularly those with Dravet syndrome and autosomal dominant optic atrophy, as well as their families and caregivers. The company collaborates with healthcare providers, treatment centers, and clinical sites to identify and access eligible patients through genetic testing. Its customer base includes pediatric and adult patients diagnosed with specific genetic mutations in the SCN1A and OPA1 genes, who are enrolled in clinical studies or potential future recipients of approved therapies.