Innorna Announces FDA Rare Pediatric Disease Designation Granted to IN013 for Treatment of Wilson Disease

Boston, Hong Kong, and Shenzhen, February 25, 2025 – Innorna, a clinical-stage biotechnology company revolutionizing the field with its innovative lipid nanoparticle (LNP) delivery technology, is thrilled to announce that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to its investigational mRNA therapy, IN013, for the treatment of Wilson Disease (WD), also known as Hepatolenticular Degeneration (HLD), a severe genetic disorder.

This milestone underscores the potential of Innorna’s mRNA-LNP technology platform and will significantly accelerate the clinical development of IN013, propelling Innorna closer to providing a transformative therapy for patients with WD.

About RPDD

The FDA grants RPDD to drugs and biologics aimed at treating rare orphan diseases affecting fewer than 200,000 patients in the U.S., with serious or life-threatening impacts predominantly on individuals aged 18 years or younger. Under the FDA’s Rare Pediatric Disease Priority Review Voucher (PRV) program, sponsors receiving approval for a biologics license application for a rare pediatric disease may obtain a voucher for priority review of a subsequent marketing application for a different product. These vouchers can be used by the sponsor or sold to another sponsor, and have recently sold for between $100 million to $150 million.

About Wilson Disease (WD) and IN013

Wilson Disease (WD), or Hepatolenticular Degeneration (HLD), is a rare autosomal recessive disorder resulting from mutations in the ATP7B gene, leading to defective copper metabolism and toxic copper buildup in the liver, brain, kidneys, and other organs. The clinical spectrum ranges widely based on the organs affected and disease severity, predominantly manifesting as hepatic and/or neurological damage, along with ocular abnormalities, hemolysis, kidney injury, and skeletal complications. Existing treatments for WD offer limited symptom relief, often accompanied by hepatorenal toxicity and potential worsening of neurological symptoms.

IN013 leverages Innorna’s cutting-edge mRNA-LNP platform to target the root cause of WD by restoring functional ATP7B protein levels through mRNA technology. By mitigating systemic copper accumulation, IN013 holds promise for alleviating multi-organ symptoms and delivering disease-modifying benefits.

About Innorna

Founded in 2019, Innorna is dedicated to developing a globally leading lipid nanoparticle (LNP) delivery technology platform and innovative RNA therapies to address unmet clinical needs. The company has established a Diversity-Oriented Lipid Library (DOLL), encompassing over 5,000 ionizable lipids for use in developing various innovative therapies, including mRNA vaccines and drugs, gene editing, and cell therapy. Leveraging its proprietary mRNA and LNP technology platform, Innorna has established multiple internal R&D pipelines for infectious disease vaccines, rare diseases, and tumor immunotherapy. The company has also partnered with several multinational biopharmaceutical and biotechnology companies to explore the technology’s potential for broader treatment applications.

Since its establishment, Innorna has gained wide recognition from the investment and industrial sectors and has received numerous awards, including being named among MIT Technology Review’s Global Top 50 Smartest Companies and Fortune China’s Most Socially Influential Startup Companies. At Innorna, we value INNOVATION, INTEGRITY, EFFICIENCY, and OPENNESS. We are committed to exploring the frontier of mRNA application based on platform technologies and leading the revolutionary step toward expanding the clinical application of mRNA in various therapeutic approaches to fulfill the unmet medical needs of patients worldwide.

For more information, please visit www.innorna.com.