On July 16, 2025 Genetic Leap, a clinical stage, AI-native biotech pioneering RNA genetic therapeutics, reported that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for GL-IL2-138, the company’s small-molecule modulator of natural interleukin-2 (IL-2) (Press release, Genetic Leap, JUL 16, 2025, View Source [SID1234654414]).
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IL-2 is a pleiotropic cytokine that regulates the careful homeostatic balance between immune tolerance and activation. The tremendous potential of IL-2 to address multiple major diseases is universally recognized. Recombinant IL-2 protein (rIL-2) was the first FDA-approved immunotherapy proven effective against cancer at high dose, though toxicity has historically limited its use. And low dose rIL-2 is clinically effective in autoimmune and neurodegeneration diseases. But numerous attempts by pharma companies to remove toxicity from rIL-2 muteins have flopped, earning IL-2 its reputation as an impossible target.
Genetic Leap addressed this herculean challenge by developing a small molecule drug that binds endogenous IL-2 mRNA to drive controlled, systemic production of natural IL-2 protein by immune cells, which has many advantages over rIL-2 including:
Superior potency and durability
Oral dosing with high tolerability
Broad "pipeline-in-a-pill" potential across oncology, immunology (I&I), and neurodegeneration
"We created the Genetic Leap AI platform to deliver breakthroughs like GL-IL2-138," said Dr. Bertrand Adanve, Founder and CEO of Genetic Leap. "Innovative medicine that defies known scientific limitations, like a sci-fi drug made real. GL-IL2-138 is like combining Humira (leading autoimmune biologic) and 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."
The drug will now be tested in a randomized, double-blind, placebo-controlled phase 1 clinical trial to evaluate its tolerability, pharmacokinetics and pharmacodynamics in humans.