Rare diseases
Patients worldwide
Lack approved therapies
Are children
At Immitra Bio, we are pioneering a novel genetic medicine platform by combining two of the most powerful biomedical technologies: Hematopoietic Stem Cells (HSCs) and CRISPR-Cas9 gene editing. Our innovative approach enables precise and durable gene modifications within blood stem cells, unlocking transformative treatments for rare diseases with high unmet medical needs.
Hematopoietic stem cells possess an extraordinary ability to self-renew and differentiate into all blood and immune cells. By harnessing these regenerative properties, we integrate cutting-edge CRISPR-Cas9 technology to correct or introduce therapeutic genes directly into the patient’s own cells. This approach offers lifelong benefits by providing a sustainable in vivo factory for producing functional proteins, addressing the root cause of genetic disorders rather than just managing symptoms.
Our proprietary gene-integration platform, HematoGen, is designed to be a scalable solution, capable of addressing multiple rare diseases that currently have no viable treatment options.