Mar 11, 2026，Shanghai CirCodeBiomed Co., Ltd. (hereinafter referred to as "CirCode") announced amajor breakthrough for its second core innovative circular RNA (circRNA) drug,HM2003 Injection, which has officially obtained Investigational New Drug (IND)clearance from the U.S. Food and Drug Administration (FDA) and concurrentlybeen granted the Rare Pediatric Disease Designation (RPDD) for undisclosedindication. Prior to this, HM2003 had already received the FDA Orphan DrugDesignation (ODD) for Thromboangiitis Obliterans (TAO). The approval of INDcoupled with two special designations marks an important milestone for HM2003’sglobal clinical development and further demonstrates the internationalcompetitiveness of CirCode’s circRNA technology platform.
HM2003 is developed based on CirCode’s core technologyplatform fully recognized by the FDA. Endowed with the core advantages of highstability and low immunogenicity from its covalently closed-loop structure, aswell as a mature R&D and translation system, this platform has been fullyvalidated in the development of HM2002, the company’s world’s first-in-classcircRNA drug. HM2002 has not only obtained 3 clinical trial approvals fromChina’s National Medical Products Administration (NMPA) and 1 from the FDA, butalso been awarded the FDA Fast Track Designation, emerging as a benchmark forcircRNA drugs in the field of ischemic heart disease. As the second coreproduct, HM2003 has achieved efficient R&D by leveraging this matureplatform and won multiple important FDA accreditations in a short period, whichfully confirms the replicability and expandability of CirCode’s technologyplatform.
The FDA IND clearance paves the way for HM2003 toinitiate clinical trials in the United States, laying a core regulatory foundationfor verifying its safety and efficacy in target populations worldwide. The RPDDdesignation is a high recognition by the FDA of the drug’s potential to addressthe significant unmet medical needs in rare pediatric diseases. Upon thesubsequent marketing approval of this indication, CirCode will be eligible toreceive a transferable Priority Review Voucher (PRV), which can shorten theFDA’s standard review period for any of the company’s drugs from 10 months to 6months and holds important strategic and commercial value. In the meantime, thecompany can also acquire R&D benefits such as FDA clinical protocolassistance and registration fee waivers. The previously obtained TAO-ODDdesignation enables HM2003 to get policy supports including tax credits forclinical research and market exclusivity in the field of this rare vasculardisease. These multiple FDA accreditations form a synergistic policy benefit,building a multi-dimensional R&D support system for HM2003 and greatlyaccelerating its clinical progress and approval timeline.
TAO and other-related limb ischemia, the key indicationsof HM2003, are both major areas of unmet medical needs urgently to be addressedworldwide. TAO mainly affects young populations, easily leading to limbischemia, ulceration and even amputation, and there is currently no curativetherapy targeting its pathological mechanism. Relying on the unique advantagesof circRNA technology, HM2003 improves limb ischemia through specific molecularmechanisms, providing a brand-new innovative treatment option for patients withthese two diseases and is expected to fill the global gap in clinicaltreatment.
The CEO of CirCode, Chenxiang TANG stated: "Theacquisition of both FDA IND clearance and RPDD designation for HM2003, togetherwith the previously obtained TAO-ODD, is a dual confirmation of the maturity ofCirCode’s circRNA technology platform and the clinical value of the drug.Following HM2002, HM2003’s rapid breakthrough in global regulatory recognitiononce again verifies the internationalization capability of the company’sR&D system. Going forward, we will fully leverage the policy benefits frommultiple FDA accreditations to speed up the global clinical trials of HM2003,striving to bring innovative therapies to patients with TAO and other relatedlimb ischemia at an early date. In the future, CirCode will continue to deepenits layout in the field of circRNA drugs, expand its R&D pipeline throughtechnological innovation, and create more core products to meet the unmetmedical needs worldwide."
“As an innovative biotech company focused oncircRNA drug development, CirCode has built a comprehensive core technologysystem and a global patent protection layout. Two of the company’s coreproducts have entered the global regulatory and clinical development stage,with its R&D pipeline covering multiple major disease areas includingischemic heart disease and limb ischemic diseases. Adhering to technologicalinnovation as the core, the company will accelerate the global R&D andclinical translation of circRNA drugs, and continuously demonstrate theinnovative strength of Chinese biopharmaceuticalenterprises in the global cutting-edge biotechnology field.”