SQY Therapeutics’
Technological Platform:
Our unique technological platform allows us to access custom synthesis of a new superior generation
of antisense oligonucleotides (ASOs) from the tricyclo-DNA (tc-DNA) class.
With recognized expertise in the field of rare neuromuscular diseases, our R&D team relies on an innovative and universal high throughput screening cascade to effectively analyze the efficacy of hundreds of tc-DNA ASOs and identify lead compounds to progress.
Drug candidates are studied in tri-dimensional (3D) human DMD engineered muscles (mini-muscle) generated from patient’s cells or genetically modified.
Such innovative disease models closely mimic mature dystrophic muscle tissue architecture, benefit from the appropriate human genetic context and offer the flexibility necessary for the development of personalized therapeutic approaches based on the nature of the genetic anomalies considered. Our universal multi-phase funnel screening cascade aim to identify and triage effective drug candidates with appropriate safety profile.
The technological platform of SQY Therapeutics aims to accelerate the discovery of drug candidates and their translation to patients.