Helex is driving synthetic RNA-driven therapeutics to solve rare genetic conditions by partnering with drug developers. The Helex Platform is powered by intelligent data-driven deep-learning modeling—using extensive high-throughput, next-generation sequencing data derived from CRISPR edits on human cell lines. Helex not only gives reliable predictions through the Helex Platform but also validates them in-house using cell-based assays and next-generation sequencing.