[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 evolved from a 'nice to have' technology to a 'must have' technology."

Isanghoon Lee, CEO of ABL Bio, offered this diagnosis of the changing paradigm 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 May 26. He stated, "As recently as 2022, when we signed a technology transfer agreement with Sanofi, the BBB shuttle platform was considered a nice-to-have feature. However, in just a few years, multinational pharmaceutical companies have reached a point where they will not even consider introducing brain disease drugs without a BBB shuttle." In response to this shift in global market demand, ABL Bio is expanding the application of its core platform, Grabody-B, from conventional antibodies to next-generation modalities 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 that "after the phase 2 clinical results of the amyloid-targeting bispecific antibody trontinemab, jointly developed by Genentech and Roche, were announced in October 2024, the market underwent a clear turning point—much like the division between BC and AD—regarding BBB shuttle technology." He explained that by the time discussions for technology agreements with multinational pharmaceutical companies took place, the situation had completely reversed, making the acquisition of a shuttle platform for drug delivery to the brain the top priority for major pharma companies. Subsequently, ABL Bio has signed consecutive technology transfer agreements worth trillions of won with companies such as GSK and Eli Lilly.

Amid growing market demand, ABL Bio is pursuing a proactive strategy to expand its modalities. Moving beyond the conventional approach of delivering antibodies to the brain, the company has fully entered the development of siRNA brain delivery technology, which has emerged as a key area of interest for global pharmaceutical companies. Lee stressed, "siRNA has an inherent limitation in that, when administered, the drug is delivered only to the liver and kidneys. Therefore, attaching a BBB shuttle to deliver the drug to the brain is essential for using siRNA as a treatment for brain diseases." He added, "In particular, the siRNA platform must do more than simply penetrate the blood-brain barrier; for therapeutic efficacy, the drug must directly enter the neurons, which are the nerve cells of the brain."

Lee pointed out the disadvantages of transferrin receptor-based technologies commonly used by competitors and emphasized the technical superiority of ABL Bio's proprietary platform. He explained that the transferrin receptor has a relatively low rate of penetration into brain cells, while the insulin-like growth factor 1 receptor (IGF1R), which ABL Bio targets, is highly efficient at delivering drugs into neurons. Based on this technological strength, the company is developing a next-generation platform optimized for siRNA delivery by modifying the form and structure of its shuttle. ABL Bio plans to release concrete data to the market by the end of this year or next year at the earliest.

This expansion of modalities is the result of thorough 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. Coincidentally, at the Keystone Symposium in early the following year, we met with Ionis, a world-renowned expert in the siRNA field, and began joint research. We were able to drive successive partnerships by presenting the research data we secured to both Lilly and GSK."

ABL Bio is not content to rest on existing technology and has begun research on a bispecific antibody shuttle platform that targets two sites simultaneously to maximize penetration efficiency. Through this, the company plans to continue expanding its business into various modalities, including enzymes and proteins, beyond antibodies and siRNA. Collaborative clinical trials with global partner companies are also progressing smoothly. Lee said, "Joint clinical preparations with Eli Lilly, GSK, and others are proceeding rapidly according to the originally anticipated timeline, and we will mobilize all of our internal resources 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.