Business Wire

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

Share

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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20250427120055/en/

Contacts

Innorna Co. Ltd.
info@innorna.com

About Business Wire

For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.

www.businesswire.com

Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

Ant International Partners with Barclays on Global Treasury Management with Proprietary AI-Powered FX Model9.5.2025 16:57:00 EEST | Press release

Ant International has entered a partnership with leading UK bank Barclays to enhance efficiency and resilience in global treasury management for businesses. Under the partnership, the two sides will combine innovative solutions, including Ant’s proprietary Time-Series Transformer (TST) AI FX Model, to help businesses reduce FX-related costs and risks against global volatilities. At the initial stage of the collaboration, Ant International has successfully completed the first batch of its intra-group FX transactions with Barclays. Ant International’s TST Model is a transformer architecture-based big data model with close to 2 billion parameters. By integrating the latest time series forecasting algorithms, the TST Model predicts patterns over time. Ant also created new pre-training and Supervised Fine-Tuning (SFT) frameworks to train the model and improve its predictions over time. The TST Model now forecasts the company's cashflow and FX exposure on an hourly, daily and weekly basis, w

Monument Re Transfers €1.4bn Greycastle Portfolio to RGA and Strengthens European Life Insurance Consolidation Platform9.5.2025 15:00:00 EEST | Press release

Monument Re Limited (“Monument”) announces today that it has transferred a legacy €1.4bn reinsurance portfolio, comprising annuity and other life insurance liabilities acquired as part of the 2020 Greycastle transaction, to RGA Americas Reinsurance Company, Ltd. (“RGA”). This transaction releases capital resources that Monument will redirect to its core strategy of consolidation in European life insurance markets. The transaction completed on 2May 2025 following approval by the Boards of Directors of both Monument and RGA and non-objection from the Bermuda Monetary Authority. Monument has taken significant steps in recent months to strengthen its business operations by consolidating its European group support functions in Dublin and by aligning with the recently strengthened regulatory regime in Bermuda. With its strong financial position and best in class capabilities in the Group, Monument remains ideally positioned to build on its success to date and grow its European footprint, del

IFF Completes Divestiture of Nitrocellulose Business9.5.2025 15:00:00 EEST | Press release

IFF (NYSE: IFF) today announced that it has completed the divestiture of its nitrocellulose business, including Walsrode Industrial Park in lower Saxony, Germany, to Czechoslovak Group (CSG). The business manufactures nitrocellulose strictly for industrial purposes, serving customers primarily in coatings and printing inks, and had been part of IFF’s Pharma Solutions business unit. “The divestiture of our nitrocellulose business builds upon our deleveraging journey and enables us to focus on our core businesses,” said Erik Fyrwald, IFF CEO. “I’d like to thank our nitrocellulose colleagues for their dedication and wish them continued success as part of CSG.” Welcome to IFF At IFF (NYSE: IFF), we make joy through science, creativity and heart. As the global leader in flavors, fragrances, food ingredients, health and biosciences, we deliver groundbreaking, sustainable innovations that elevate everyday products—advancing wellness, delighting the senses and enhancing the human experience.Le

CPAC Systems AB Announces Strategic Minority Investment in Flying Fish9.5.2025 13:33:00 EEST | Press release

CPAC Systems AB, a leader in advanced control systems and embedded vessel and commercial vehicle technology, today announced a strategic minority investment in Flying Fish Maritime Innovations B.V., a pioneer in advanced shared water mobility solutions and robust, cost-effective hydrofoil technology. The investment underscores both companies’ commitment to redefining water-based transportation through innovation, sustainability, and seamless integration. It marks the beginning of a deeper collaboration aimed at enabling smarter, cleaner, and more connected mobility on the water — for both recreational and commercial applications. “We are thrilled to support Flying Fish and their impressive work in redefining water mobility,"saidMarcus Wingolf, CEO of CPAC Systems. "Our investment in Flying Fish represents a strategic alignment of our technical proficiencies and innovative ambitions. This partnership opens exciting new possibilities for integration and sustainable transformation across

Kioxia Receives IEEE Corporate Innovation Award9.5.2025 10:00:00 EEST | Press release

Kioxia Corporation, a world leader in memory solutions, today announced that it has received the IEEE Corporate Innovation Award from the Institute of Electrical & Electronics Engineers (IEEE), the world’s largest technical professional organization dedicated to advancing technology for the benefit of humanity. This award recognizes Kioxia's outstanding contribution in the field of electrical and electronics engineering through its BiCS FLASH™ technology, a low-cost, high-capacity 3D flash memory innovation. The award ceremony was held on April 24 in Tokyo. The IEEE Corporate Innovation Award is a globally-recognized honor bestowed upon organizations that have developed innovative technologies, products or services that have made a substantial contribution to the advancement of electrical and electronics engineering. Since its inception in 1985, the award has been presented to leading electronics manufacturers and IT companies worldwide, and Kioxia is proud to be the seventh Japanese c

In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.

Visit our pressroom
World GlobeA line styled icon from Orion Icon Library.HiddenA line styled icon from Orion Icon Library.Eye