[Bio Story]①ABL Bio's BBB Shuttle Platform Becomes a 'Must-Have'

Sanghoon Lee, CEO of ABL Bio, Interview ①

Grabody-B Expands Beyond Antibodies to siRNA
"Now an Indispensable Technology for Brain Disease Drugs"

"The blood-brain barrier (BBB) penetration platform has shifted from being a 'nice to have' technology to becoming a 'must-have' technology."

Isanghoon Lee, CEO of ABL Bio, diagnosed this paradigm shift in the global new drug development market for brain diseases during an interview with The Asia Business Daily at the company's headquarters in Gangnam-gu, Seoul, on the 26th of last month. He stated, "As recently as 2022, when we signed a technology licensing agreement with Sanofi, the BBB shuttle platform was still considered a nice addition. However, in just a few years, multinational pharmaceutical companies have reached the point where they won't even consider brain disease drugs that lack a BBB shuttle." In response to these changes in global market demand, ABL Bio is expanding the application scope of its core platform, 'Grabody-B,' from traditional antibodies to next-generation modalities (therapeutic approaches) such as small interfering RNA (siRNA).

Isanghoon Lee, CEO of ABL Bio, poses holding a model of the company logo during an interview with this publication at the headquarters in Gangnam-gu, Seoul, on the 26th of last month. Photo by Dongjoo Yoon

Lee assessed, "Since the announcement of the phase 2 clinical trial results for Tronemab, an amyloid-targeting bispecific antibody jointly developed by Genentech and Roche, in October 2024, the market has seen a clear turning point for BBB shuttle technology—akin to a before-and-after era." He explained that, by the time they began discussions with multinational pharmaceutical companies on technology deals, the situation had completely reversed, making access to shuttle platforms that deliver drugs to the brain the top priority for major pharmaceutical companies. Subsequently, ABL Bio signed consecutive technology licensing agreements worth trillions of won with companies such as GSK and Eli Lilly.

As market demand increases, ABL Bio is pursuing a proactive strategy to expand its modalities. Moving beyond its previous focus on delivering antibodies to the brain, the company has now fully entered the development of siRNA brain delivery technology—a major area of interest for global pharmaceutical companies. Lee emphasized, "siRNA has an inherent limitation in that, when administered, the drug is delivered only to the liver and kidneys. To use it as a treatment for brain diseases, it is essential to attach a BBB shuttle that can carry the drug to the brain." He added, "In particular, with the siRNA platform, it’s not enough to simply cross the blood-brain barrier; for the drug to be effective, it must directly enter neurons, the nerve cells in the brain."

Lee highlighted the technical superiority of ABL Bio’s proprietary platform by pointing out the drawbacks of the transferrin receptor-based technology commonly used by competitors. While the transferrin receptor shows relatively low penetration efficiency into brain cells, the insulin-like growth factor 1 receptor (IGF1R) targeted by ABL Bio demonstrates excellent efficiency for neuron entry. Based on these technological strengths, the company is developing a next-generation platform optimized for siRNA delivery by modifying both the form and structure of the shuttle. Specific data on this platform is expected to be released to the market as early as the end of this year or sometime next year.

This expansion of modalities is the result of meticulous preparation combined with intuition. Lee said, "When we met with Eli Lilly at the end of 2022 to discuss antibody technology transfer, the partner company proactively inquired about the possibility of integrating siRNA." He continued, "Coincidentally, at the Keystone Symposium in early the following year, we met with Ionis, a global authority in the siRNA field, and began joint research. The research data we obtained enabled us to present compelling evidence to Lilly and GSK, leading to a series of partnerships."

ABL Bio is not resting on its existing technology and has also begun research on a bispecific antibody shuttle platform that targets two molecules simultaneously in order to maximize penetration efficiency. Through this, the company plans to continuously expand its business into various modalities such as enzymes and proteins, beyond antibodies and siRNA. Collaborative clinical trials with global partners are also progressing smoothly. Lee said, "Joint clinical preparations with Eli Lilly, GSK, and others are proceeding rapidly according to the originally expected timeline. We will mobilize all internal capabilities to support the submission of Investigational New Drug (IND) applications in line with our partners' development schedules."

This content was produced with the assistance of AI translation services.