• Enabled by WuXi Biologics' industry-leading integrated technology platform, velaglucerase-beta for injection has successfully advanced from concept to commercialization.
• Velaglucerase-beta for injection, as the first and the only locally developed enzyme replacement therapy (ERT) for Gaucher disease in China, marks a significant breakthrough for CANbridge Pharmaceuticals and WuXi Biologics in advancing R&D of innovative rare disease therapeutics in China.
• Velaglucerase-beta for injection is the first innovative biological product in China to pass segmented manufacturing inspection.

WuXi Biologics (2269.HK), a leading global Contract Research, Development and Manufacturing Organization (CRDMO), congratulates its partner CANbridge Pharmaceuticals on the approval of its innovative velaglucerase-beta for injection (Gaurunning) by the China National Medical Products Administration (NMPA) for the treatment of adolescents aged 12 and above and adults with type I and type III Gaucher disease. This marks China's first and only locally developed enzyme replacement therapy (ERT) for Gaucher disease.

Gaucher disease is a rare inherited genetic lysosomal storage disorder characterized by clinical manifestations such as hepatosplenomegaly, anemia, bone pain, and neurological symptoms, which can be life-threatening in severe cases. Recombinant human glucocerebrosidase ERT has been the standard treatment for Gaucher disease for 30 years, significantly improving major non-neurological signs and symptoms, as well as improving the quality of life for patients. It is estimated that the global prevalence of Gaucher disease is 0.7 to 1.75 per 100,000 people. Gaucher disease has been included in China's "First List of Rare Diseases".

Velaglucerase-beta for injection is the first collaboration project on rare diseases between CANbridge and WuXi Biologics, advancing from concept to commercialization by leveraging WuXi Biologics' leading integrated technology platform. The project utilized innovative technology to construct high-expression cell line and achieved significant improvements in both yield and quality through WuXiUP^TM, an ultra-high productivity continuous bioprocessing platform of WuXi Biologics. The overall yield increased by more than 110 times, and the specific enzyme activity increased by more than 50%, ensuring the affordability for Gaucher disease patients. Additionally, WuXi Biologics developed cell-based bioassay methods for commercial product release and stability testing, addressing the technical challenges of enzyme testing and effectively ensuring product quality control. Additionally, velaglucerase-beta for injection is the first innovative biological product in China to pass segmented manufacturing inspection.

Dr. Chris Chen, CEO of WuXi Biologics, stated, "We are honored to enable CANbridge to achieve the significant milestone of approval for velaglucerase-beta for injection, which represents a critical advancement in rare disease drug innovation in China. Developing therapies for rare diseases is a long and challenging journey, requiring relentless joint efforts to bridge the gap between early-stage discovery and patients' access to medication. The successful commercialization of velaglucerase-beta for injection not only reflects CANbridge's decade-long dedication to rare diseases but also exemplifies the value of WuXi Biologics' integrated platform and CRDMO business model. Furthermore, our shared commitment to addressing the unmet clinical needs of rare disease patients has resulted in China's first locally developed ERT for Gaucher disease. We look forward to enabling more global partners to accelerate the development of high-quality rare disease therapies, and making them accessible and affordable for more patients suffering from rare diseases."

Dr. James Xue, Founder, Chairman, and CEO of CANbridge Pharmaceuticals, stated, "Enzyme replacement therapy is a crucial part of CANbridge' global pipeline. The approval of velaglucerase-beta for injection in China is a milestone. By leveraging the integrated technology platform, velaglucerase-beta for injection will significantly reduce development costs, making safe and effective domestically produced ERT accessible and affordable for patients. This product is also the first collaboration project between CANbridge and WuXi Biologics in rare diseases. We extend our gratitude to our partners, including WuXi Biologics, who have empowered the development and manufacturing of velaglucerase-beta for injection. This milestone is not only CANbridge' third rare disease drug to be launched following Hunterase® and Livmarli®, but also the first in-house developed commercialized product. In the future, we will continue to focus on the localized research, manufacturing, commercialization, and accessibility of rare disease drugs, bringing innovative treatments to patients in China."

Since its establishment, WuXi Biologics has been committed to enabling global innovative biotech companies to accelerate the rare disease drugs from concept to commercialization, benefiting rare disease patients worldwide. As ofthe end of 2024, there are 21 rare disease projects on the company's platform. Additionally, the company actively promotes public awareness and supports the rare disease patient community through various philanthropic initiatives. Its global "Run for Health" campaign has engaged over 2,500 participants to raise awareness for rare diseases. In China, the company's "Rare Disease Student Education Support Program" has provided scholarships to over 100 students affected by rare diseases over the past four years, fostering their academic growth.