[Bio Story]②Technology Validation Completed with Global Big Pharma Eli Lilly... Growing Anticipation for Successive Technology Transfers

Interview with Sung Wook Lee, CEO of Algenomics (Part 2)


Material Transfer Agreement with Eli Lilly for Genetic Hearing Loss Treatment

Potential Expansion to CNS and Other Indications with Big Pharma

Algenomics is accelerating its efforts to achieve global license-out agreements, leveraging the technical excellence of its proprietary RNA (ribonucleic acid) editing platform. With major milestones such as successful technology validation with the multinational pharmaceutical company Eli Lilly and the designation as a Regenerative Medicine Advanced Therapy (RMAT) by the U.S. Food and Drug Administration (FDA), discussions about partnerships with global big pharma companies are more active than ever.

The most notable commercialization achievement is the Material Transfer Agreement (MTA) signed with Eli Lilly in the field of genetic hearing loss therapeutics. Eli Lilly conducted a thorough, year-long validation of Algenomics' platform technology using its own in-house researchers. An interesting aspect is that Eli Lilly did not disclose the specific indication at all during the initial stages of the validation process.

Lee Sungwook, CEO of Algenomics, recalled on July 22, "Eli Lilly meticulously validated our technology through their in-house research team for a year. At first, they did not even tell us which disease they were targeting; it was only after the successful validation that we learned it was intended for genetic hearing loss." He continued, "Because it was a specific target that could not be treated with conventional gene overexpression methods, our RNA substitution technology was essential. This demonstrates that global big pharma has directly confirmed the unique competitiveness of our platform, which other technologies cannot achieve."

The partnerships with global pharma companies are not limited to a single indication but are also open to the possibility of expansion into other diseases such as those of the central nervous system (CNS). Because Algenomics' technology is structured as a platform, it provides the versatility needed to address unmet needs across a diverse range of pipelines held by major pharmaceutical companies.

The smooth progress of clinical trials for Algenomics' oncology pipeline is also fueling global license-out discussions. The company's liver cancer and malignant brain tumor treatments were recently awarded RMAT designation by the FDA, officially recognizing their potential as 'first-in-class' drugs.

CEO Lee said, "The RMAT designation indicates that the FDA has validated the innovation and potential of our early clinical results. With a dedicated project manager now assigned by the FDA and entry into an accelerated approval track, the number of global companies showing interest in our oncology pipeline has increased significantly, and technology transfer discussions are now being conducted in much greater depth than before."

To support successful technology transfers and the expansion of indications, Algenomics is also giving top priority to advancing its drug delivery systems. The biggest topic in today's gene therapy market is how to precisely deliver drugs to target organs—such as the heart, brain, or muscles—instead of only the liver, so as to minimize toxicity. To this end, Algenomics is actively engaging in open innovation with leading domestic and international biotech firms, including Glugin Therapeutics, which specializes in adeno-associated virus (AAV) vector research, and Surginex, a lipid nanoparticle (LNP) specialist.

CEO Lee explained, "The key to expanding our technology is securing drug delivery systems that ensure safe delivery to target organs during systemic administration, beyond just local administration, so drugs do not accumulate solely in the liver. By jointly developing these systems with excellent partner companies both in Korea and abroad, we are maximizing their practical value in clinical use."

Regarding the timing of the much-anticipated technology transfer, Lee refrained from making definitive statements but expressed strong confidence. Given that the company has entered into multiple Confidential Disclosure Agreements (CDA) and MTAs with major global companies and is currently undergoing active due diligence, there is speculation that meaningful results could be achieved in the near future. Lee added, "Considering the progress and volatility of the ongoing discussions, I cannot specify an exact date at this moment. However, the current level and atmosphere of discussions are so vigorous and positive that it would not be surprising if a global technology transfer agreement is concluded at any time."

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