Publication · 2025 · PDF
Science
Explore conditions, clinical trials, and the latest research.
These pages contain non-exhaustive scientific information about uses of products that have not yet been approved by regulatory authorities, as their efficacy and safety for these uses have not yet been established. The information is provided for educational and scientific exchange purposes, and does not suggest products should be used in a manner not consistent with their respective label approved by the regulatory authorities, which may differ by geography. Any publication reflects the state of knowledge at the time it was published. The information is not medical advice and cannot replace the independent medical judgement of a healthcare professional.
Conditions
Current Research
The conditions we work on all stem from clear genetic causes: mutations, missing enzymes, misfolded proteins. The biological precision of genetic conditions gives us a real starting point to build therapies that target the root cause and not just the symptoms. Many of these conditions are progressive, overlooked, and still lack approved treatments. We’re here to change that by following science with urgency and designing with purpose because the people living with these diseases can’t afford to wait. They’re the reason we move fast, ask hard questions, and never compromise on what matters most.