Creating medicines that endure
Kamau engineers long-lived stem cells to create precise therapeutic changes with the potential to carry forward over time.
Platform
The power to make lasting change
Kamau harnesses the cell’s homology-directed repair pathway in self-renewing hematopoietic stem cells, combining site-specific engineering with the control of an ex vivo process. Because these cells give rise to red and immune cell lineages throughout the body, they create a common foundation for therapeutic approaches across hematology, oncology, and neurology.
Durability by design
Therapeutic changes made in long-lived stem cells have the potential to carry forward over time.
Site-specific precision
Kamau guides the repair process to a selected genomic site, either restoring a native sequence under its natural regulation or introducing a new therapeutic function.
Control throughout
Cells are selected, modified, characterized, and verified before infusion. Evidence from development and manufacturing connects process, product, and patient outcomes, strengthening the platform over time.
Phase 1/2 • NCT04819841 • FDA Fast Track and Orphan Drug designations
nula-cel
Correcting the root cause of sickle cell disease
Correcting the root cause of sickle cell disease
Nula-cel is an investigational stem medicine designed to directly correct the sickle-causing mutation in a patient’s own blood stem cells and restore production of adult hemoglobin. nula-cel is an investigational stem medicine designed to directly correct the sickle-causing mutation in a patient’s own blood stem cells and restore production of adult hemoglobin.
RESTORE is a Phase 1/2 clinical trial evaluating nula-cel in people with severe sickle cell disease at clinical centers across the United States.
RESTORE is a Phase 1/2 clinical trial evaluating nula-cel in people with severe sickle cell disease at clinical centers across the United States.
Our vision
A world
with more cures
with more cures
