/PRNewswire/ -- Genetic Leap, a clinical stage, AI-native biotech pioneering RNA genetic therapeutics, announced today 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).

/PRNewswire/ -- 临床阶段的AI原生生物技术公司Genetic Leap，专注于RNA基因治疗领域的创新，今日宣布美国食品药品监督管理局（FDA）已批准其在研新药（IND）申请，涉及公司的小分子天然白介素-2（IL-2）调节剂GL-IL2-138。

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.

IL-2 是一种多效性细胞因子，能够调节免疫耐受和激活之间精细的稳态平衡。IL-2 在应对多种重大疾病方面的巨大潜力已得到普遍认可。重组 IL-2 蛋白 (rIL-2) 是首个获得 FDA 批准的高剂量有效抗癌免疫疗法，但其毒性在历史上限制了它的使用。

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..

低剂量rIL-2在自身免疫和神经退行性疾病中具有临床效果。但制药公司多次尝试去除rIL-2突变体的毒性均以失败告终，这使得IL-2赢得了“不可成药靶点”的名声。

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:

Genetic Leap 通过开发一种小分子药物来应对这一艰巨挑战，该药物结合内源性 IL-2 mRNA，驱动免疫细胞可控且系统地生产天然 IL-2 蛋白，与重组 IL-2 相比具有诸多优势，包括：

Superior potency and durability

卓越的效力和持久性

Oral dosing with high tolerability

口服给药，耐受性良好

Broad 'pipeline-in-a-pill' potential across oncology, immunology (I&I), and neurodegeneration

广泛的“药丸中的管道”潜力，涵盖肿瘤学、免疫学（I&I）和神经退行性疾病

'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.

“我们创建了 Genetic Leap AI 平台，以实现像 GL-IL2-138 这样的突破，”Genetic Leap 创始人兼首席执行官 Bertrand Adanve 博士说道。“这是一种创新药物，突破了已知的科学限制，就像科幻药物变成现实一样。GL-IL2-138 就像是将 Humira（领先的自体免疫生物药）和 Keytruda（领先的癌症生物药）结合成一种口服药丸，没有它们的缺点，并且适用于更多疾病。”

GL-IL2-138 is poised to be a giant leap for human health, and Genetic Leap is the company making it happen.'                 .

GL-IL2-138 将成为人类健康领域的巨大飞跃，而 Genetic Leap 是实现这一目标的公司。

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.

该药物现在将进入随机、双盲、安慰剂对照的1期临床试验，以评估其在人体中的耐受性、药代动力学和药效学。

About Genetic Leap

关于Genetic Leap

Genetic Leap is innovating at the cutting edge of AI and RNA genetic therapeutics to redefine drug development and more quickly address the health needs of millions of people. For more information, please visit

Genetic Leap处于人工智能和RNA基因治疗的前沿，创新性地重新定义药物开发，更快地满足数百万人的健康需求。欲了解更多信息，请访问