Extending precise stem cell engineering across diseases
Kamau is advancing nula-cel in sickle cell disease and exploring the same core capabilities across hematology, oncology, and neurology. Each program begins with long-lived HSCs and is designed for lasting benefit from a single intervention.
Our Pipeline
A growing pipeline of stem medicines
Program
Therapeutic area
Approach
Stage
nula-cel
Sickle cell disease
Direct correction of the sickle-causing mutation at the native HBB locus
Phase 1/2 clinical
Oncology research portfolio
Selected oncology indications
Programmed therapeutic functions in immune cell progeny
Research
Neurology research portfolio
Selected neurological indications
HSC-derived delivery of therapeutic proteins within the CNS
Research
Phase 1/2 • NCT04819841 • FDA Fast Track and Orphan Drug designations
nula-cel
A direct approach to gene correction
A direct approach to gene correction
nula-cel is designed to use homology-directed repair to correct the sickle-causing mutation at the native HBB locus and restore production of adult hemoglobin.
It is being evaluated in the Phase 1/2 RESTORE clinical trial and has received FDA Fast Track and Orphan Drug designations.
It is being evaluated in the Phase 1/2 RESTORE clinical trial and has received FDA Fast Track and Orphan Drug designations.
Enabling technologies
Building for broader access and scale
Kamau is advancing conditioning, manufacturing, and data capabilities designed to make stem medicines more scalable, consistent, and accessible.
01
Targeted conditioning
Myeloablative conditioning is the current standard of care for stem cell gene therapy. Kamau is advancing more targeted, precise conditioning approaches with the aim of supporting outpatient treatment and extending access to broader patient populations, potentially including newborns.
02
Scalable manufacturing
Kamau is a process-engineering company. Every program shares the same target cell, editing chemistry, manufacturing process, and release assays, allowing each new program to begin with a clinical-grade process. Automated, closed systems are designed to increase yield and consistency while lowering the cost of each dose.
03
Continuous platform learning
Each development and manufacturing cycle expands the data connecting process parameters, product attributes, and clinical outcomes. These insights sharpen process control across the pipeline.
Research
Expanding the therapeutic reach of HSCs
HSCs give rise to red blood cells and diverse immune cell lineages throughout the body. Engineering this common cellular source creates multiple paths to therapeutic impact.
Oncology
A potential lasting source of engineered immune cells
Engineering HSCs creates the potential for a self-renewing source of T cells, NK cells, and macrophages carrying programmed therapeutic functions. Kamau is exploring this approach with the aim of supporting durable, multilineage immune activity in cancer.
Neurology
Therapeutic delivery within the CNS
HSC-derived microglia and plasmablasts can take up long-term residence in the central nervous system. Kamau is engineering these cells as localized sources of antibodies, enzymes, and neurotrophic factors within the CNS.
