Kamau directs homology-directed repair to make precise therapeutic changes in long-lived blood stem cells. We control the process from editing through manufacturing to create medicines designed for lasting benefit from a single intervention.
HBB TARGET
Our approach
Process control supports reproducible precision
Kamau integrates editing, cell processing, analytics, and manufacturing to direct homology-directed repair at chosen genomic sites and make it reproducible at clinical scale.
Direct precise repair
A targeted editing system and donor template guide sequence correction at a chosen genomic site.
Preserve stemness
Process conditions are designed to support cell viability, self-renewal, engraftment potential, and multilineage function.
Define the product
Cells are characterized for identity, precision, potency, quality, and safety before administration.
Learn with every cycle
Each development and manufacturing cycle expands the evidence connecting process, product, and clinical outcomes. Those insights strengthen process control and inform the programs that follow.
Therapeutic reach
One stem cell Multiple therapeutic paths
HSCs sit upstream of red blood cells and diverse immune cell lineages throughout the body. Engineering this common cellular source creates the potential to carry therapeutic change through the progeny it continually produces.
Correct a native sequence
Aim to restore healthy function
Correcting a disease-causing sequence at its native genomic locus preserves the gene’s natural regulation. nula-cel applies this approach to the HBB locus in sickle cell disease, with the goal of restoring production of adult hemoglobin.
Inserting a therapeutic sequence at a selected genomic site can give HSC-derived immune cells a programmed function. Kamau is applying this capability to new approaches in oncology.
Deliver a therapeutic protein
Create a potentially lasting source within the CNS
HSC-derived cells can populate the central nervous system and create the potential for localized delivery of therapeutic proteins. Kamau is exploring this approach across neurological diseases.
Kamau is developing closed, automated manufacturing systems designed for consistency, throughput, and efficiency. As these systems advance, they can expand capacity and support broader patient access.