Innorna Announces U.S. FDA Rare Pediatric Disease Designation Granted to IN016 for the Treatment of Progressive Familial Intrahepatic Cholestasis

HONGKONG, SHENZHEN, NANJING, CHINA, July 11, 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 (the “FDA”) has granted Rare Pediatric Disease Designation (“RPDD”) to IN016, one of the Company’s lead mRNA product candidates for rare diseases. IN016 is to treat the Progressive Familial Intrahepatic Cholestasis (PFIC), a group of rare genetic disorders that cause a progressive liver disease and can lead to cirrhosis and end-stage liver disease in infants and children. Following IN022, a potential mRNA therapy for treating Homocystinuria (HCU), IN016 marks the second Innorna product candidate to receive the RPDD from FDA. The RPDD will greatly facilitate IN016 and IN022 clinical development and bring these novel mRNA therapies to PFIC and HCU patients more quickly.

Innorna’s advancements in rare diseases are driven by its technology breakthroughs in LNP delivery field. Different from mRNA infectious disease vaccines, the delivery technology used for mRNA rare disease therapies has many challenges, including tissue targeting, delivery efficiency, and safety. To overcome these, Innorna has, through extensive research, discovered and developed the optimal LNP delivery technology for its mRNA rare disease therapies. This breakthrough has enabled a rapid advancement of Innorna's rare disease pipeline, addressing the high unmet medical need of many patients. In addition, a significant progress has also been made in extra-hepatic-targeted delivery technology, which lays the groundwork for future extra-hepatic disease pipeline development.
 

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 focuses on developing best-in-class LNP delivery technology and

advancing innovative RNA therapies to address unmet medical needs of patients globally. Innorna has developed a diversity-oriented lipid library (DOLL) of over 5,000 ionizable lipids, which can be applied in various modalities or scenarios, including mRNA vaccines, protein replacement therapies, in vivo genome editing therapies, and in vivo CAR therapies. Based on its proprietary mRNA and LNP technology platform, Innorna has built extensive internal R&D pipelines for infectious diseases, rare diseases, and immunotherapies. In addition, the company has established partnerships with multiple domestic and global biotechnology/biopharmaceutical companies to explore the potential of the technology in broader therapeutic areas. For more information, please visit www.innorna.com.