FDA Clears IND for GL-IL2-138 in Cancer and Beyond

• The FDA has cleared an investigational new drug (IND) application for GL-IL2-138.
• GL-IL2-138 is a small-molecule modulator of natural interleukin-2 (IL-2).
• The agent’s tolerability, pharmacokinetics, and pharmacodynamics will now be evaluated in a phase 1 clinical trial.

The FDA has granted clearance to the IND application for GL-IL2-138, an oral small molecule designed to modulate natural IL-2 production through mRNA targeting.¹

"We created the Genetic Leap AI platform to deliver breakthroughs like GL-IL2-138," said Bertrand Adanve, PhD, founder and chief executive officer of Genetic Leap, in a press release. "Innovative medicine that defies known scientific limitations, like a sci-fi drug made real. GL-IL2-138 is like combining Humira ([adalimumab] leading autoimmune biologic) and [pembrolizumab (Keytruda)] (leading cancer biologic) into one oral pill without the downsides and applicable to even more diseases. GL-IL2-138 is poised to be a giant leap for human health, and Genetic Leap is the company making it happen."

IL-2 remains one of the most powerful immunomodulatory cytokines in oncology, historically demonstrating curative potential in select cancers such as melanoma and renal cell carcinoma when administered at high doses. However, clinical application has been severely constrained by its toxicity and limited therapeutic window.

Despite decades of protein engineering efforts, attempts to refine IL-2 receptor selectivity have yet to overcome these challenges.

Rather than delivering exogenous recombinant IL-2, GL-IL2-138 selectively binds endogenous IL-2 mRNA, enabling immune cells to generate natural IL-2 protein in a regulated, physiologic manner. This strategy offers several key advantages over traditional rIL-2 and engineered cytokines, including sustained immunologic activity with reduced systemic toxicity, oral administration with a favorable tolerability profile, and potential applications across oncology, autoimmune, and neurodegenerative indications.

With its IND now active, GL-IL2-138 will enter a randomized, double-blind, placebo-controlled phase 1 trial to assess safety, pharmacokinetics, and pharmacodynamics in humans.

In addition to this IL-2 modulator, Genetic Leap’s portfolio spans novel and known targets, including those that are traditionally undruggable.² These include MYC, PTPN2, IL-1α, IL-1β, and FLCN.

REFERENCES:

1. Genetic Leap receives FDA IND clearance for first-in-class, oral natural IL-2 modulator. News release. Genetic Leap. July 16, 2025. Accessed July 16, 2025. https://tinyurl.com/4p9nzbf2

2. Pipeline. Genetic Leap. Accessed July 16, 2025. https://www.genetic-leap.com/pipeline