Guangzhou First People's Hospital Completes Southern China's First Gene Therapy for Gaucher Disease, Ushering in a New Era for Rare Disease Treatment in the Greater Bay Area
Lingyi Biotech
Gene Therapy Drug Developer
February 26, 2025, inDirector Wang Shunqing of the Department of Hematology, Guangzhou First People's HospitalUnder the leadership, a patient suffering from the rare genetic disease "Gaucher's disease" has recently successfully undergone gene therapy. This is another milestone achievement completed by the CGT (Cell and Gene Therapy) subspecialty following the breakthrough in the field of thalassemia by the hospital’s hematology department, marking a new phase in the field of gene therapy in South China.
The patient receiving treatment this time has a 12-year history of Gaucher's disease, according toDirector Yiping Zhang, Department of Hematology, Nansha Hospital, Guangzhou First People's HospitalIntroduction: The patient has long relied on expensive enzyme replacement therapy (ERT) to control the condition, but this therapy requires lifelong medication and imposes a heavy financial burden. After learning about the clinical trial of LY-M001 injection gene therapy conducted by the Hematology Department of Nansha Hospital, Guangzhou First People's Hospital, the patient voluntarily applied to join the group and passed the strict screening evaluation.Becoming the first patient in South China to receive this therapyThe patient is currently in good condition after treatment, and the medical team has high expectations for the treatment outcome.
How Gene Therapy "Cures Once and for All" Gaucher Disease?
Gaucher Disease (GD) is an autosomal recessive genetic disorder caused by mutations in the glucocerebrosidase (GBA1) gene. The accumulation of metabolic substrates in patients leads to symptoms such as hepatosplenomegaly, anemia, and bone pain. Director Huang Jinqi of the Hematology Department at Guangzhou First People's Hospital further explained that traditional therapies require lifelong medication. However, LY-M001 injection, the first gene therapy drug in China targeting Type I and Type III Gaucher disease, uses a recombinant adeno-associated virus (rAAV) vector to deliver a functional glucocerebrosidase gene to target organs, achieving long-term stable expression. This degrades harmful substances, aiming for a "one-time treatment, lifelong effectiveness" outcome.
This drug was independently developed by Lingyi Biotech Co., Ltd. and has full independent intellectual property rights. It received dual IND approvals from the National Medical Products Administration (NMPA) and the U.S. FDA in January 2024, and was granted FDA Orphan Drug and Pediatric Rare Disease Designation, highlighting its innovation and clinical value.
Collaborative Innovation among Government, Industry, Academia, and Research:
Building a Biomedical High Ground in Nansha
Guangzhou First People's Hospital - CGT Subspecialty Clinical Trial Center -
Guangdong Medical Valley Transformation Base
December 26, 2024The First People's Hospital of Guangzhou-CGT Subspecialty Clinical Trial Center-Guangdong Medical Valley Transformation Base Officially Unveiled, The success of this treatment is an important achievement of the collaboration among the Nansha Hospital of Guangzhou First People's Hospital, Lingyi Biotech, and Guangdong Medical Valley. The project relies on the policy advantages of the "pilot first" in the Nansha Free Trade Zone, actively responding to the national "Opinions on Supporting Guangzhou Nansha to Relax Market Access and Strengthen Regulatory System Reform", and fully utilizing policy dividends and institutional innovation. As the forefront of reform and opening up, the Nansha Free Trade Zone provides solid policy support for the rapid transformation of gene therapy technology from laboratory to clinical application. As a national biomedical industry incubation platform, Guangdong Medical Valley integrates upstream and downstream industrial chain resources, and provides full-chain platform support for the implementation of innovative cell and gene therapy technologies through the construction of an industrial ecosystem.
Looking Ahead:
Leading New Directions in Rare Disease Treatment with Technology
The First People's Hospital of Guangzhou stated that it will continue to deepen strategic cooperation with research institutions and enterprises, accelerate the clinical transformation and application of gene therapy technology, while enhancing public awareness and attention to rare diseases, improving the medical security system, and lighting up the hope of life for more patients.
Guangdong Medical Valley will further strengthen the foundation of the clinical trial center for cell and gene therapy as a transformation base, accelerate the transformation and application of innovative technologies in the field of cell and gene therapy, continue to uphold the concept of innovation-driven development, continuously introduce top global research teams and innovative enterprises, and constantly promote the internationalization of China-produced gene therapy technologies. It is believed that with the joint efforts of all parties, the Greater Bay Area will surely become an important source of innovation for the global biopharmaceutical industry, making greater contributions to the cause of human health.
About Guangzhou First People's Hospital
The First People's Hospital of Guangzhou is a large-scale comprehensive tertiary Grade A hospital that integrates medical treatment, teaching, research, prevention, and healthcare. Its Department of Hematology is a domestic leader in the field of rare disease diagnosis and treatment. The CGT Subspecialty focuses on the research and practice of innovative therapies, providing cutting-edge treatment options for patients with genetic disorders.
About Guangdong Medical Valley
Guangdong Medical Valley, established in 2014, is a platform focused on investment and incubation in the biopharmaceutical and medical device industries. Its business sectors include the operation of medical industry incubators, equity investments in medical projects, and comprehensive medical support services. Guangdong Medical Valley • Nansha Life Science Park (Phase I), located in the core area of the Nansha Free Trade Zone in Guangzhou and adjacent to Nansha Port, is the first national-level technology enterprise incubator in Nansha District. On September 16, 2021, the General Office of the People's Government of Guangdong Province issued the "Notice on Printing and Distributing the '14th Five-Year Plan' for the Development of the China (Guangdong) Pilot Free Trade Zone," in which Guangdong Medical Valley was listed as a key project in the development of modern service industries (healthcare industry).
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Contact Person for Guangdong Medical Valley Transformation Base:
Ms. Wang from Guangdong Medical Valley
Ms. Su from Guangdong Medical Valley
