Press Release 2025. 03. 14
Boston, Hong Kong, Shenzhen - March 14, 2025 - Innorna, a pioneering clinical-stage biotechnology company revolutionizing the mRNA field with its innovative lipid nanoparticle (LNP) delivery technology, is proud to announce that its investigational mRNA therapy, IN013, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of Wilson Disease (WD), also known as Hepatolenticular Degeneration (HLD). This achievement, coupled with the previously awarded Rare Pediatric Disease Designation (RPDD), marks a significant milestone in the development of IN013, bringing Innorna closer to delivering transformative therapies to patients affected by Wilson Disease.
About Orphan Drug Designation
The FDA’s Office of Orphan Products Development awards orphan drug designation to drugs and biologics aimed at preventing, diagnosing, or treating rare diseases affecting fewer than 200,000 individuals in the U.S. This designation provides a range of incentives, including up to seven years of market exclusivity upon approval, tax credits for qualifying clinical trials, and exemption from certain user fees.
About Wilson Disease and IN013
Wilson Disease (WD), or Hepatolenticular Degeneration (HLD), is a rare and life-threatening autosomal recessive disorder caused by ATP7B gene mutations. These mutations lead to defective copper metabolism and toxic accumulation of copper in the liver, brain, and other vital organs. The disease presents a spectrum of symptoms, with primary manifestations involving hepatic and neurological damage, along with ocular abnormalities, kidney injury, hemolysis, and skeletal complications. Existing treatments often fall short, offering limited relief and introducing significant risks such as hepatorenal toxicity or exacerbated neurological symptoms.
IN013, built on Innorna’s advanced mRNA-LNP platform, is designed to address the root cause of WD by restoring functional ATP7B protein levels. By effectively reducing systemic copper accumulation, IN013 has the potential to alleviate multi-organ symptoms and offer patients meaningful, disease-modifying benefits.
About Innorna
Founded in 2019, Innorna is committed to developing state-of-the-art LNP delivery technology and RNA-based therapies to address unmet medical needs. The company’s proprietary Diversity-Oriented Lipid Library (DOLL), housing over 5,000 ionizable lipids, has laid the foundation for breakthroughs across mRNA vaccines, gene editing, and cell therapy.
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 garnered widespread recognition, including accolades from MIT Technology Review as one of the Global Top 50 Smartest Companies and Fortune China as one of the Most Socially Influential Startups. The company’s core values - INNOVATION, EFFICIENCY, INTEGRITY, and OPENNESS - drive its mission to revolutionize mRNA applications and make a global impact.
For more information, visit www.innorna.com
