Press Release 2024. 07. 30
HONGKONG, SHENZHEN, NANJING, CHINA, July 30, 2024—
Innorna, a clinical-stage biotech company pioneering its proprietary lipid nanoparticle (LNP) technology to develop novel RNA therapeutics, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to its investigational therapy IN016, for the treatment of progressive familial intrahepatic cholestasis (PFIC). IN016 had also been granted with Rare Pediatric Disease Designation (RPDD) on July 10, 2024. Having obtained both RPDD and ODD would greatly facilitate IN016 clinical development/approval process, therefore bring this potential therapy to PFIC patients quickly.
About PFIC and IN016
Progressive familial intrahepatic cholestasis (PFIC) is a heterogeneous group of rare genetic disorders associated with defects in bile acid secretion or transport, resulting in unwanted bile accumulation within the liver. Typical symptoms include jaundice, hepatomegaly, pruritus, splenomegaly, and diarrhea. Over time, this disease can evolve into severe liver damage, fibrosis, cirrhosis, and is associated with high mortality rates. While symptoms can be managed to some extent, more effective treatment is in great need for PFIC. IN016 is designed to address the root cause of PFIC resulting from gene mutations, potentially restoring defective proteins, normalizing bile excretion, and possibly improving symptoms in PFIC patients.
About Innorna
Founded in 2019, Innorna aims to develop 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 multi-national 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. Please visit the Innorna website at www.innorna.com for more information.
