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Lead program · Recurrent glioblastoma · Research stage

KGEN-001 is built 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.
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Ready

Off-the-shelf healthy-donor product

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Reach

Repeat intraventricular dosing

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Recognize

B7-H3-anchored dual targeting

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Resist

Multi-program resistance

KGEN-001 is a research-stage program. It is not in clinical trials and is not available to patients today.

The complete product

KGEN-001 combines what CNS CAR-T has proven with what durable control still requires.

Early CNS CAR-T studies have established repeat dosing, tumor recognition, and radiographic regression. KGEN-001 builds on that progress by adding ready-to-treat supply and multi-program resistance within the same cell.

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-to-treat

A ready-to-treat CAR-T product removes the patient-specific manufacturing wait and starts with healthy-donor cells.

In recurrent GBM, disease can advance during autologous manufacturing. Patient-derived cells can yield a more exhausted product after matched expansion.

Human-evidence-grounded design

Six coordinated edits address five durability failure modes grounded in human GBM evidence.

Human GBM biology, clinical GBM CAR-T data, and supporting mechanistic evidence defined more than 80 candidate resistance targets and guided selection of six coordinated edits spanning five durability failure modes. The six were selected as one integrated KGEN-001 architecture.

Why recurrent GBM

Recurrent GBM provides a fast, information-rich test of whether KGEN-001 can make control last.

Early GBM CAR-T studies have established repeat CNS dosing and shown radiographic regression. Ommaya access, serial CSF, and longitudinal MRI create a rare opportunity to track treatment, local immune biology, response, and durability.

We can track not only whether a response occurs, but what changes as it holds or fails.

A medically plausible Ommaya reservoir beneath the scalp connects through a catheter to a lateral ventricle, beside a cerebrospinal fluid collection tube.

PUBLISHED GBM CAR-T EXAMPLE · NOT KGEN-001

Axial contrast-enhanced brain MRI from Patient 26 on Day -1 before CAR-T treatment.
Baseline · Day -1
Axial contrast-enhanced brain MRI from Patient 26 on Day +3 after CAR-T treatment showing marked radiographic regression.
Early response · Day +3
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Repeat dosing

Multiple doses can be delivered over time as the disease evolves.

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Serial CSF

Tracks CAR-T abundance, cell state, and local immune biology over time.

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Longitudinal MRI

Shows the depth and persistence of radiographic response.

Response and progression unfold over weeks to months.
Published GBM CAR-T example: Patient 26 showed marked reduction in tumor enhancement at Day +3 and progression on the 1-month MRI. Bagley et al., Nature Medicine, 2025, NCT05168423.

Follow the development of KGEN-001.

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

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