ReFOLD Bio

ReFOLD Bio discovers gene editing targets for protein misfolding diseases that affect hundreds of millions of patients worldwide. ReFOLD identifies novel refolding edits that restore protein function without correcting the original mutation. This approach unlocks a blue ocean: patients carrying mutations that no existing gene therapy can address. Targets are validated directly in patient-derived organoids, producing assets with substantially higher translational confidence than industry standard.

The validity of our technology is proven in Cystic Fibrosis, a $13B therapeutic market where a curative one-time treatment remains the stated unmet need. Here, we have already identified three novel, previously undescribed targets that restore protein function.

ReFOLD is not a single-asset play. Each indication our discovery engine validates becomes a new partnerable asset. Multiple priority indications have been identified that combine high unmet need with a large market uniquely unlocked by our technology. Projected total addressable market for currently identified targets is ~$17.5B.

Our team combines world-class CRISPR base editing expertise, direct access to the patient-derived organoid biobanks used in our screens, and an established CF clinical trial network. We are advised by Prof. Hans Clevers (pioneer of organoid technology, former Head of pRED at Roche), Prof. MD. Kors van den Ent (lead clinician, HIT-CF trial), and Dr. Gijs van den Brink (Partner at Forbion, former SVP pRED).