Gene Therapy We demonstrate successful correction of F508del and G551D mutations in cystic fibrosis patient-derived intestinal organoids, with restored chloride channel function and forskolin-induced swelling. These results support advancement to IND-enabling studies.
Oncology Our medicinal chemistry program identified a new class of covalent KRAS G12C inhibitors with improved selectivity and reduced off-target binding compared to existing clinical compounds. Phase I trial enrollment is underway.
Autoimmune Integrating transcriptomic, proteomic, and metabolomic data from 450 SLE patients, we identified three previously uncharacterized pathways driving disease flares. Two corresponding drug candidates have entered preclinical development.
Computational Biology We present HelixAI, our proprietary machine learning platform for therapeutic target identification and compound optimization. Benchmarked against traditional workflows, HelixAI reduced the average hit-to-lead timeline from 18 months to 7 months across three internal programs.