Wuh-Liang Hwu, Honorary Professor at National Taiwan University College of Medicine, has recently initiated the development of a novel gene therapy targeting the rare inherited disorder Sialidosis. The therapy utilizes adeno-associated virus serotype 9 (AAV9) as a vector to deliver the gene encoding neuraminidase (sialidase) into the human body, offering a promising treatment option for patients. The manufacturing partner for this new therapy is TFBS Bioscience, which will provide end-to-end support including AAV9 vector process development, GMP-grade clinical trial material manufacturing, as well as quality and safety testing services for Phase I/II clinical use.
Sialidosis is a rare lysosomal storage disorder caused by a deficiency of the enzyme neuraminidase, resulting in the accumulation of undegraded sialylated oligosaccharides within cells. This metabolic block disrupts normal function across the central nervous system, skeletal system, and reticuloendothelial system. Symptoms may include gait abnormalities, vision loss, myoclonus (muscle jerks), ataxia, leg tremors, and seizures. These symptoms progressively worsen, often leading to severe mobility impairment requiring the use of a wheelchair. Currently, there is no cure for sialidosis, and patients rely on supportive therapies to manage symptoms.
Professor Hwu is a trailblazer in Taiwan’s gene therapy field. During his tenure at the Departments of Pediatrics and Medical Genetics at National Taiwan University Hospital, he developed the world’s first AAV gene therapy for Aromatic L-amino Acid Decarboxylase (AADC) deficiency. This was also Taiwan’s first gene therapy licensed to an international pharmaceutical company, achieving regulatory approval in the European Union (2022) and the United States (2024). After retiring from NTU, Professor Hwu joined China Medical University Hospital, where he continues to advance gene therapy programs for other rare genetic diseases.
TFBS Bioscience, originally established as a biosafety testing provider, is now Taiwan’s first and only CDMO capable of GMP-grade viral vector manufacturing. The company offers comprehensive CDMO services for biopharmaceuticals and cell and gene therapies, spanning method development, production, and quality control.
Professor Hwu’s gene therapy program for sialidosis, supported by the Center for Translational Medicine at NTU Hospital, has already shown promising results in mouse models. With forward-looking planning underway, if clinical trials confirm the therapy’s safety and efficacy, it could offer a much-needed new treatment option for patients living with this devastating rare disease.
Resource: 台灣基因治療之父胡務亮攜手啟弘生技，推動罕見疾病的基因治療
