Ready
Healthy-donor cells
Prepared in advance. No patient-specific manufacturing.
KGEN-001 · Recurrent GBM · Preclinical development
Healthy-donor cells.
Engineered to resist.
KGEN-001 is designed to make control last in glioblastoma.
A healthy-donor, dual-target CAR-T with six coordinated base edits, designed for repeat dosing into cerebrospinal fluid.
KGEN-001 at a glance
Reach
Repeat CNS dosing
Delivered directly into cerebrospinal fluid.
Recognize
Two targets, one receptor
Designed to recognize two tumor markers.
Resist
Six coordinated base edits
Designed to resist multiple tumor shutdown signals.
The disease clock
Making patient-derived CAR‑T takes time.
Recurrent GBM does not wait.
KGEN-001 starts with healthy-donor cells prepared in advance, so treatment would not depend on manufacturing a new product for each patient.
Published TX103 clinical study
>25%Tumor growth
before first infusion.In 12 of 15 patients (80%)
Enrollment~40 daysFirst infusion
Progression: at least a 25% increase in tumor measurements, based on the sum of products of perpendicular diameters.
Zhang et al., Nature Medicine, 2026The starting cell
Speed is not enough.
The starting cell matters.
Fewer exhaustion-associated cells
After matched expansion. Rounded values.
Patient-derived GBM mouse model
2.5×Greater survival extension
Healthy-donor versus GBM-patient CAR-T.
Compares survival extension relative to control CAR-T, not total survival.
Engineered resistance
Five resistance programs.
Six coordinated base edits.
Human GBM biology defined what KGEN-001 must resist.
Checkpoint signalsSuppressive cytokinesSuppressive metabolitesT-cell death signalsChronic TCR stimulation
What do these protections address?
- Checkpoint signals
- Signals that reduce T-cell activity through immune checkpoints.
- Suppressive cytokines
- Immune messages in the tumor environment that inhibit T-cell function.
- Suppressive metabolites
- Tumor-associated chemicals, such as adenosine, that dampen T-cell activity.
- T-cell death signals
- Signals through death receptors that can trigger the loss of therapeutic T cells.
- Chronic TCR stimulation
- Repeated stimulation of the cell’s native T-cell receptor, including by tumor-associated macrophages, can wear cells down. The engineered CAR provides a separate route for tumor recognition.
Why GBM first
A direct test of sustained tumor control.
Recurrent GBM combines urgent need with a clinical foundation for CAR-T delivery to the brain. KGEN-001’s development plan builds toward measuring how long activity lasts.
See the clinical durability gapIntended clinical development approach
- Deliver to the CNS
- Develop for administration into cerebrospinal fluid, building on published experience with brain-tumor CAR-T.
- Evaluate repeat dosing
- A prepared donor-cell product is intended to support more than one administration.
- Follow activity over time
- Serial MRI and cerebrospinal-fluid sampling would help assess tumor control and cell behavior.
Planned development concept. KGEN-001 remains preclinical; a clinical dosing regimen has not been established.
For patients and families
KGEN-001 is in preclinical development. It is not in clinical trials and is not available to patients today.
Any future clinical-trial information will be shared here.
For information about clinical trials that may be recruiting, visit the National Brain Tumor Society Clinical Trial Finder or ClinicalTrials.gov.
Discuss the KGEN-001 program.
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KGEN-001 is a research-stage program and is not approved for clinical use.