국립암센터 면역세포유전자치료제전주기기술개발연구단

R&D

Opening New Possibilities in Therapeutics for Hard-to-Treat Solid Tumors

Research Programs

CAR-T / TCR-T Clinical Research

CAR-T and TCR-T therapies are genetically engineered T-cell therapies
designed to selectively recognize and eliminate cancer cells.

Evaluation Endpoints

Safety of
Cell Infusion
Pharmacological
Profile
Preliminary
Antitumor
Response (PoC)

Assessment of the Clinical Feasibility of CAR-T/TCR-T Therapies

This clinical research aims to evaluate the safety of cell infusion, pharmacological profile (including PK and PD), and preliminary antitumor responses in patients with solid tumors to assess the feasibility of clinical application of CAR-T/TCR-T therapies.

R&D

Project Overview

Explore each research project.

EGFRvIII/IL-13Rα2 tandem CAR-T

Seoul National University Hospital
Nrf2 KD GD2 CAR-T

Pusan National University Yangsan Hospital
Liver Cancer-Targeting CAR-T

Seoul National University
PDL1/EphA2
tandem CAR-T

VaxCell-Bio
IL-13Rα2 CAR-T

National Cancer Center
Armored GPC3
CAR-T

National Cancer Center

Research Overview

EGFRvIII/IL-13Rα2 tandem CAR-T / Indication: Glioblastoma
Indication
Glioblastoma Unmet Medical Need

The current standard of care is surgical resection followed by concurrent radiotherapy and chemotherapy,but recurrence rates remain high.
CAR-T and TGF-β inhibitor

Key Features

Dual targeting of EGFRvIIIandIL-13Rα2

Tumor-specific expression of EGFRvIII and preferential expression of IL-13Rα2 in glioblastoma
→ Minimizes the risk of on-target off-tumor toxicity
Simultaneous targeting of EGFRvIII and IL-13Rα2
→ overcomes tumor heterogeneity and antigen escape

Combination therapy with a TGF-β inhibitor

TGF-β inhibitor relieves immunosuppression within the TME
TGF-β inhibitor improves CAR-T cell survival and persistence
→ Enhances antitumor efficacy by improving CAR-T cell cytotoxicity and remodeling the TME
Research Overview

Nrf2 KD GD2 CAR-T / Indication: Neuroblastoma
Indication
Neuroblastoma
Unmet Medical Need

More than 45% of patients present with metastatic disease at diagnosis, resulting in poor clinical outcomes

Although standard treatments include chemotherapy, surgical resection, autologous hematopoietic stem cell transplantation, radiotherapy, and immunotherapy, patients with relapsed or refractory neuroblastoma continue to have limited therapeutic options.
CAR-T

Key Features

GD2 and anti-GD2 scFv

High GD2 expression on neuroblastoma cells
→ High tumor specificity with reduced toxicity to normal tissues.
scFv derived from the anti-GD2 monoclonal antibody clone 14G2a
→ Reduces risk of novel scFv development with well-established manufacturing processes and quality standards

Nrf2 KD CAR-T

Nrf2-KD CAR-T cells co-expressing a GD2 CAR and shNrf2
→ Reduces CAR-T cell exhaustion, thereby improving antitumor activity in a ROS-rich TME
(* KD: knockdown, reduced Nrf2 protein expression)
Research Overview

C12 B7H3 CAR-T / Indication: Hepatocellular Carcinoma
Indication
Hepatocellular Carcinoma
Unmet Medical Need

Curative treatment is often not feasible with hepatic resection, radiofrequency ablation, or TACE.

Targeted therapies provide limited benefit.

Immune checkpoint inhibitors have shown only modest improvement.
CAR-T

Key Features

Liver Cancer-targeting CAR-T

Simultaneous targeting of tumor cells and tumor-associated endothelial cells
→ improves CAR-T cell infiltration into the TME

Converter CAR Technique

Co-expression of Converter enhances CAR-T function
※ The Converter is a fusion protein composed of the extracellular domain of CTLA-4 and the intracellular domain of CD28. Replacement of CTLA-4 intracellular domain with the CD28 intracellular domain enables B7 binding to convert the native CTLA-4 inhibitory signal into a CD28-mediated costimulatory signal.

Signal-responsive cytokine-secreting CAR-T

Upon antigen recognition, CAR-T secrete cytokines
→ enhances CAR-T cell activity within the antigen-expressing TME
연구개요

PDL1/EphA2 tandem CAR-T / 적응증: 위암, 간암 및 난소암
적응증
재발성/불응성 위암, 간암 및 난소암 치료의 미충족 수요

수술, 방사선, 화학요법제에 한계가 있음

표적치료제, 면역관문억제 치료제에 역할 제한적
CAR-T
특징
이중 표적 PD-L1 및 EphA2의 차별성
PD-L1 및 EphA2를 동시에 표적

암세포 인지 특이성 향상

종양 조직 내 항원 이질성 및 항원 소실에 따른 종양의 면역회피 극복
CAR tandem 인지부의 차별성
anti-PD-L1 scFv와 anti-EphA2 monobody

각각 PD-L1과 EphA2에 적절한 친화력

면역원성이 낮고, 해리력이 매우 빠름

→ 비표적 작용에 따른 독성 (off-target toxicity) 감소
Research Overview

IL-13Rα2 CAR-T / Indications: Leptomeningeal Metastases, CNS Metastatic Disease
Indications and Route
Leptomeningeal Metastases(LM)
Unmet Medical Need

Radiotherapy and surgery have shown limited therapeutic benefit for LM
CNS Metastatic Disease Clinical feasibility of CSF-directedtherapy

Clinical benefit demonstrated with intrathecal trastuzumab of meningeal carcinoma in HER2-positive metastatic breast cancer

Clinical efficacy reported for EGFR-targeted therapy in EGFP-mutant NSCLC with LM
CAR-T

Key Features

IL-13Rα2 and IL-13 ligand

Tumor-selective expression of IL-13Rα2 with minimal expression in normal tissues
High selectivity of the engineered IL-13 ligand
→ Minimizes the risk of on-target off-tumor toxicity while maximizing antitumor efficacy in LM

intracerebroventricular administration

CSF-directed ICV administration
Research Overview

Armored GPC3 CAR-T / Hepatocellular carcinoma, Head and neck cancer
Indications
Head and neck cancer
Unmet Medical Need

Surgery, radiotherapy, and chemotherapy have limited efficacy in patients

Immune checkpoint inhibitors have shown only modest clinical benefit
Potential suitability of CAR-T therapy for head and neck cancer

Relatively high immune cell infiltration

Favorable CAR-T cell infiltration into the TME
CAR-T

Key Features

GPC3 and anti-GPC3 scFv

Tumor-selective GPC3 expression with minimal expression in normal tissues
Specific recognition of GPC3-expressing tumor cells by an anti-GPC3 scFv

→ minimizes the risk of on-target off-tumor toxicity

Armored CAR-T

Armored CAR-T cells are engineered to express molecules that enhance antitumor activity, such as cytokines, immune resistance factors, or tumor-targeting factors
(As of July 2025, Genes X, A, B, and C are currently being evaluated for their ability to enhance the antitumor efficacy of CAR-T cells.)