HLB, which is seeking approval for new drugs for liver cancer and cholangiocarcinoma, is moving to penetrate the solid tumor market by fronting the next-generation chimeric antigen receptor T-cell (CAR-T) platform "KIR-CAR" from its U.S. subsidiary Verismo Therapeutics as a follow-up anticancer pipeline.
Lee Ji-han, an executive director at HLB, said at the "HLB Forum" held on the 12th at the Sofitel Ambassador Seoul Hotel in Songpa-gu, Seoul, "HLB aims to expand CAR-T from its traditional focus on blood cancers to the solid tumor market," adding, "If our CAR-T platform 'KIR-CAR' proves effective in solid tumors, the likelihood of joint development and technology transfers with global big pharma will grow."
CAR-T is a personalized gene therapy that genetically engineers a patient's immune cells (T cells) to precisely find and attack cancer cells. With a single infusion, the cells proliferate in the body and continuously seek out and attack only cancer cells, earning the name "living anticancer drug."
So far, all CAR-T therapies approved by the U.S. Food and Drug Administration (FDA) are for blood cancers. Starting with Switzerland's Novartis "Kymriah (kymriah)" in 2017, a total of seven products had been commercialized through aucatzyl (aucatzyl) from the United Kingdom's Autolus Therapeutics in 2024. In Korea, Curocell's "Limcato" won approval last month as the country's first CAR-T therapy, entering the blood cancer market.
By contrast, the solid tumor field remains largely untapped. No CAR-T for solid tumors has received FDA approval to date. In solid tumors, CAR-T cells expend excessive energy as they navigate to tumor tissue and attack cancer cells, causing them to fall into exhaustion easily.
Conventional CAR-T therapies use a "single-chain" structure that keeps T cells continuously activated. As a result, T cells operate at a low level even before they encounter cancer cells, and by the time they need to attack, their function is already weakened. In addition, solid tumors have varying target antigens by cancer cell, raising the likelihood that only some cancer cells are eliminated, leading to relapse.
Verismo developed the next-generation platform "KIR-CAR" to overcome these limitations. Applying the signaling mechanism of natural killer (NK) cells, it is designed to activate T cells only when they recognize cancer cells. The company said this reduces T-cell exhaustion and enhances functional persistence, enabling long-term anticancer effects even in solid tumors.
Laura Johnson, chief scientific officer (CSO) at Verismo, said, "KIR-CAR is designed to operate only when it detects the mesothelin target expressed in solid tumors such as ovarian cancer and cholangiocarcinoma," adding, "Through this, we can minimize T-cell exhaustion and induce a more durable antitumor response."
Verismo is currently conducting a phase 1 trial of "SynKIR-110" in solid tumors expressing mesothelin. The company unveiled early clinical results in solid tumor patients at the American Association for Cancer Research (AACR) in April. It said meaningful tumor shrinkage was observed even at low doses, and efficacy signals became more pronounced as the dose increased.
HLB is also betting on the business potential of the KIR-CAR platform. Executive director Lee said, "Big pharma sees potential to use the platform to generate synergies with existing asset," adding, "We are keeping options open for joint development or technology transfers."
Lee added, "If efficacy is confirmed in solid tumors, global drugmakers will likely have sufficient demand to bring in the KIR-CAR platform or pipeline and combine it with their existing anticancer asset."
Verismo was founded by a CAR-T research team at the University of Pennsylvania. Michael Milone, a co-developer of Kymriah, and Donald Siegel, an expert in antibody and cell therapies, co-founded the company.
At a time when the U.S. biotech investment market had cooled, HLB Chairman Jin Yang-gon heard the technology briefing, visited the University of Pennsylvania in person, and decided to make a seed investment. HLB later made Verismo a subsidiary of HLB Innovation.