Innorna Announces FDA Rare Pediatric Disease and Orphan Drug Designations Granted to IN013 for Treatment of Wilson Disease

Innorna, a clinical-stage biotechnology company revolutionizing mRNA therapeutics with its innovative lipid nanoparticle (LNP) delivery technology, today announced the U.S. Food and Drug Administration (FDA) has granted both Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) to its investigational mRNA therapy, IN013, for treating Wilson Disease (WD). This dual achievement accelerates the clinical development of IN013, advancing Innorna’s mission to deliver transformative therapies for WD patients.

About FDA Designations

The Rare Pediatric Disease Designation (RPDD) incentivizes therapies for serious or life-threatening diseases affecting fewer than 200,000 U.S. patients, primarily those aged 18 or younger. Sponsors may qualify for a Priority Review Voucher (PRV) upon approval to expedite FDA review of a subsequent drug application.

The Orphan Drug Designation (ODD) supports therapies targeting rare diseases (affecting fewer than 200,000 U.S. patients) by providing seven years of market exclusivity, tax credits for clinical trials, and waiver of certain FDA fees.

About Wilson Disease (WD) and IN013

Wilson Disease (WD), or Hepatolenticular Degeneration (HLD), is a rare and life-threatening autosomal recessive disorder caused by mutations in the ATP7B gene, leading to toxic copper accumulation in the liver, brain, kidneys, and other organs. Symptoms include hepatic and neurological damage, ocular abnormalities, kidney injury, hemolysis, and skeletal complications. Current treatments offer limited efficacy and carry risks such as hepatorenal toxicity or exacerbated neurological symptoms.

IN013, developed using Innorna’s mRNA-LNP platform, targets the root cause of WD by restoring functional ATP7B protein levels. By reducing systemic copper buildup, IN013 aims to alleviate multi-organ damage and deliver disease-modifying benefits.

About Innorna

Founded in 2019, Innorna is committed to developing state-of-the-art LNP delivery systems and RNA therapies to address unmet medical needs. Its proprietary Diversity-Oriented Lipid Library—with over 5,000 ionizable lipids—enables breakthroughs in mRNA vaccines, gene editing, and cell therapies.

With its innovative mRNA-LNP platform, Innorna has built a robust pipeline targeting infectious diseases, rare genetic disorders, and cancer immunotherapy. Beyond its internal R&D efforts, the company collaborates with global-leading biotechnology partners to expand the reach of its transformative technology.

Innorna’s achievements have earned recognition as one of MIT Technology Review’s Global Top 50 Smartest Companies and Fortune China’s Most Socially Influential Startups. Guided by INNOVATION, INTEGRITY, EFFICIENCY, and OPENNESS, Innorna drives its mission to revolutionize mRNA applications and make a global impact.

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