Advancing a robust portfolio of next-generation therapies designed to target the underlying genetic drivers of disease.
From molecular discovery to regulatory approval, our rigorous process ensures safety and efficacy at every stage.
Target identification and validation using our proprietary AI platform.
In vivo safety studies and formulation optimization.
Phase I, II, and III studies evaluating human safety and efficacy.
Global submission, review, and market authorization.
Current status of our priority candidates.
* Pipeline information current as of Q3 2024
Our proprietary NexaGen™ platform leverages advanced CRISPR-Cas9 editing and proprietary lipid nanoparticle delivery systems to address genetic diseases at their source. We don't just treat symptoms; we aim to rewrite the blueprint of disease.
Tissue-specific tropism ensures therapies reach the right cells while minimizing off-target effects.
Rapid identification of optimal gene editor candidates using machine learning models.
95% editing efficiency in pre-clinical models targeting liver tissue.