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Lead program · Recurrent glioblastoma · Preclinical development

KGEN-001 is designed to make control last.

A healthy-donor, dual-target, multiplex-edited CAR-T designed for repeat CNS dosing and durable function under tumor pressure.

A functional engineered CAR-T cell beside a translucent brain with an Ommaya reservoir connected to a lateral ventricle for repeat delivery into cerebrospinal fluid.

Four requirements. One product.

Ready

Banked healthy-donor product

Reach

Repeat CNS dosing

Recognize

Dual-target tumor recognition

Resist

Multiplex resistance to suppression

KGEN-001 is in preclinical development. It is not in clinical trials and is not available to patients today.

The integrated product

Keep what works in CNS CAR-T. Build what is missing.

Repeat local delivery and tumor recognition provide a clinical foundation. KGEN-001 integrates those precedents with banked healthy-donor supply and coordinated resistance to tumor suppression.

A finished KGEN-001 cell connects healthy-donor manufacturing and Ommaya-mediated ventricular delivery with dual tumor recognition and resistance to suppressive cytokines and metabolites arising from the tumor environment.
Four coordinated product decisions, integrated in one therapy.

Why Ready

Timing matters. So do the cells you start with.

In recurrent GBM, waiting for treatment can mean losing ground. Cancer and prior therapy can also take a toll on a patient’s own T cells. KGEN-001 is designed to address both challenges, using healthy-donor CAR-T cells banked in advance rather than made for each patient.

Off-the-shelf availability

Published clinical study

80%

Experienced radiographic progressionbetween enrollment and first infusion

12 of 15 treated patients

Chart shows the 14 patients with measurable disease.

Progression threshold: ≥25% increase in tumor measurements (sum of products of perpendicular diameters). Approximate reconstruction of the published figure.

Zhang et al. · TX103 phase 1 · Nature Medicine, 2026

Starting-cell quality

Published mouse study

2.5×

Survival extension relative to control

Healthy-donor versus patient-derived CAR-T cells in an orthotopic GBM mouse model

Normalized summary of the published comparison. Patient-derived survival extension is set to 1.0×.

Salim et al. · orthotopic GBM8 modelInt. J. Cancer, 2026

Why Resist

Six coordinated edits are designed to address five barriers to CAR-T durability.

Human GBM biology, clinical GBM CAR-T evidence, and mechanistic support defined more than 80 candidate targets. The selected edits were combined into one integrated KGEN-001 cell product.

Why GBM first

Because an early response is not enough.

At recurrence, glioblastoma leaves patients with little time and few effective options. CAR-T has shown it can drive rapid regression. The challenge is making that activity last.

GBM brings together an urgent need, early clinical activity, and a clear durability gap. Repeat CNS dosing and serial MRI make it an unusually direct test of our thesis: can a cell therapy engineered to resist suppression keep working when the tumor pushes back?

Urgent need.A clinical signal to build on.A durability gap to close.

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.

NBTS Clinical Trial Finder ClinicalTrials.gov

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KGEN-001 is a research-stage program and is not approved for clinical use.