Roche Holding AG / Skyhawk Therapeutics Inc.

Oncology and Neurodegenerative Diseases (RNA splicing platform)

Disease Overview

RNA splicing modulation is a novel small-molecule modality that corrects exon inclusion/exclusion of disease-causing transcripts. The approach has been validated by Biogen/PTC's Spinraza and Roche/PTC's Evrysdi (risdiplam) for spinal muscular atrophy. Skyhawk's SkySTAR platform aims to extend small-molecule splicing modulation across oncology drivers (e.g., MYC, oncogenic isoforms) and neurodegenerative targets where altered splicing contributes to pathology.

Competitive Landscape

When Roche/Genentech inked the deal in July 2019, RNA splicing was a hot frontier validated by Biogen/Ionis's Spinraza (nusinersen, ASO) and the impending Roche/PTC risdiplam approval (now Evrysdi)—both in SMA. Skyhawk's differentiation was a small-molecule, orally bioavailable approach targeting splicing across oncology and CNS, expanding well beyond the SMA paradigm. Direct competitors building small-molecule splicing platforms included Stoke Therapeutics (TANGO ASO platform for haploinsufficiency, e.g., Dravet), PTC Therapeutics (oral splicing modifiers), Remix Therapeutics, and Arrakis Therapeutics (RNA-targeted small molecules). Biogen had also signed Skyhawk earlier in January 2019 for neurology targets, signaling broad pharma validation. The Genentech alliance mattered because it gave Skyhawk a second top-tier partner with both oncology and neuroscience commercial muscle, plus a $2B+ biobucks value—one of the largest platform deals of 2019—while preserving Skyhawk's optionality to partner additional targets. James Sabry of Roche characterized splicing modulation as 'a novel approach for difficult-to-treat diseases'.