BOCG's Family | Innorna Announces U.S. FDA Rare Pediatric Disease Designation Granted to IN022 for the Treatment of Homocystinuria

July 9, 2024


We are delighted to announce that Innorna has been granted the U.S. FDA Rare Pediatric Disease Designation (RPDD) for IN022, their investigational therapy for treating classic homocystinuria (HCU). Congratulations to Innorna for this remarkable achievement!

The following article is from Innorna:

HONG KONG, SHENZHEN, NANJING, CHINA, July 08, 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) has granted Rare Pediatric Disease Designation (RPDD) to its investigational therapy IN022 for the treatment of classic homocystinuria (HCU), a serious or life-threatening genetic disease. This RPDD will greatly facilitate IN022 clinical development and bring this potential therapy to patients as quickly as possible. The RPDD also means that FDA may award a Priority Review Voucher upon the approval of the IN022 product.

About HCU and IN022
HCU is a rare autosomal recessive inherited disorder of sulfur amino acid metabolism primarily caused by deficiency of cystathionine beta synthase (CBS). HCU patients have a wide spectrum of age (older than three years) on presentation, with multisystemic clinical complications, particularly skeletal and connective tissue defects, osteoporosis, dislocated optic lenses, learning difficulties, and developmental delay or thromboembolism. Currently, there is no approved therapeutic drug for this disease. IN022 is designed to address the root cause of HCU resulting from deficiency in CBS. It is expected to restore the function of CBS enzyme, thereby normalizing homocysteine metabolism and potentially ameliorating symptoms in HCU 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 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 foreign biopharmaceutical companies to explore the potential of the technology in broader therapeutic areas. For more information, please visit