Jun 24, 2024
by Suyeon Jang
Great News from Ingenium Therapeutics – Making Strides in Korea and the U.S.
Ingenium Therapeutics is making meaningful progress on both sides of the Pacific, with promising updates coming out of both Korea and the United States.
Most recently in Korea, Ingenium’s NK cell therapy received compassionate use approval—a special designation that allows patients with serious or life-threatening conditions to access treatments still under clinical development. The approval has led to a first successful patient administration, marking a key milestone for the company.
Ingenium’s NK therapy, currently being developed for acute myeloid leukemia (AML), was approved for Phase 2 clinical trials in Korea last year. Additional programs for non-small cell lung cancer and liver cancer are moving toward clinical entry, and a therapy for retinoblastoma remains in the preclinical stage.
The company is optimistic about the broad potential of NK cell therapy across multiple cancer types—but is currently focusing its efforts on AML as the lead indication.
This recent approval isn’t just a clinical step forward—it also signals the beginning of commercialization. Even though the treatment is still in trials, it can now be provided to patients and generate revenue. With an estimated 500 AML patients annually in Korea, and a treatment cost of around KRW 100 million (~$75,000 USD) per patient, Ingenium sees a path to meaningful early-stage revenue—while potentially saving lives.
There’s more good news: thanks to the Advanced Regenerative Bio Act passed earlier this year, patients who are not enrolled in clinical trials may also be eligible to receive cell therapies, under strict safety and ethical guidelines. Ingenium is expected to benefit from this legislative change as access expands for patients in need.
Ingenium Therapeutics Earns Orphan Drug Status from U.S. FDA
We’re excited to share that Ingenium Therapeutics’ NK cell therapy has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA)—a major milestone in our global development journey.
This designation is expected to significantly shorten both the timeline and cost of global clinical trials—by up to 50%, accelerating our path to market.
But what does this mean in real terms? Let’s break it down from two key perspectives:
For Investors
ODD allows clinical trials to proceed with smaller patient populations, helping to reduce costs while maintaining regulatory rigor.
Upon approval, it grants seven years of market exclusivity in the U.S., offering a powerful competitive advantage.
Licensing partners also benefit from the same incentives, making the technology more attractive to potential collaborators.
Bottom line: ODD opens the door for faster commercialization and strategic licensing opportunities.
For Patients
ODD was designed to support therapies for rare and underserved conditions—diseases often overlooked due to limited market size.
This means greater access to innovative treatments for patients who’ve long had few or no viable options.
It’s about more than medicine—it’s about hope, access, and impact.
At Ingenium, we see this as a pivotal step toward realizing the full potential of NK cell therapies for patients worldwide. We're moving fast, with purpose—and we're just getting started.
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