Ecosystem integration to improve access to rare disease therapy

TychoBio France runs a high-throughput screening platform integrating long-read sequencing and live cell imaging in multiple disease and toxicity relevant cell types. This platform can be leveraged for any RNA targeting therapeutics. We initially focus on steric blocking antisense oligonucleotides (SB-ASO or SBO) and train models to design safe and efficient ASO targeting multiple RNA regulation mechanisms.

We are interested in partnering with companies working on patient diagnostic and target identification for rare disease, and/or with modified oligonucleotide manufacturers developing cost effective cGMP-grade small scale manufacturing.

At the moment the platform exclusively screens 2'MOE SBOs and does not integrate manufacturing constraints. The goal of the project would be to screen alternative chemistries and integrate yields obtained at various scales to reduce costs for small patient populations.