We are pioneering curative treatments for rare genetic disorders by addressing the root cause of disease at the molecular level.
Many severe diseases are caused by a single mutation in a patient's DNA. Our gene therapy platform utilizes next-generation CRISPR-Cas9 technology to precisely locate and correct these mutations.
By delivering a functional copy of the gene or repairing the defective one, we aim to provide a one-time, curative treatment that restores normal cellular function and halts disease progression.
Viral vectors are optimized to carry the therapeutic payload.
The therapy targets affected cells with high specificity.
Defects are corrected, restoring protein production.
Combining advanced editing tools with superior delivery mechanisms.
Proprietary nuclease variants engineered for higher fidelity and reduced immunogenicity, allowing for precise DNA double-strand breaks and repair.
Next-generation Adeno-Associated Virus (AAV) capsids designed using machine learning to maximize tissue tropism and minimize required dosage.
A flexible platform approach that supports both direct administration to the patient and modification of cells outside the body.
Advancing a diverse portfolio of candidates towards clinical approval.
Beta Thalassemia
Hemophilia A
Cystic Fibrosis
Duchenne MD
*Pipeline status as of October 2023. Subject to regulatory updates.
We are actively seeking collaborations to accelerate the development of life-saving gene therapies.