Taiwan’s Gene Therapy Pioneer Dr. Wuh-Liang Hwu Partners with TFBS Bioscience to Advance Gene Therapy for Rare Disease

Taipei, Taiwan–11th June 2025— Dr. Paul Wuh-Liang Hwu, MD, PhD, Professor Emeritus of Pediatrics and Medical Genetics at National Taiwan University, and Distinguished Research Fellow at the Center for Precision Medicine, China Medical University Hospital, has recently launched a new gene therapy development project targeting Sialidosis, a rare genetic disorder. The research utilizes adeno-associated virus serotype 9 (AAV9) as a vector to deliver the sialidase gene into the human body, offering a potential effective treatment for patients. TFBS Bioscience, serving as the manufacturing partner, will provide a one-stop service for AAV9 vector process development, GMP-grade clinical trial material manufacturing, and quality and safety testing to support Phase I/II clinical trials.

Sialidosis is a rare lysosomal storage disorder caused by a deficiency in sialidase, which disrupts the metabolism of sialic acid, leading to an accumulation of undegraded sialylated oligosaccharides in cells. This buildup affects the central nervous system, skeletal system, and reticuloendothelial system. Symptoms include gait disturbances, vision loss, myoclonus, ataxia, leg tremors, and seizures. Myoclonus worsens over time, eventually impairing a patient’s ability to sit, stand, or walk without assistance, often requiring the use of a wheelchair. Currently, there is no cure for Sialidosis; patients rely mainly on supportive therapies to manage symptoms. If clinical trials prove this AAV9-based gene therapy to be safe and effective, it could become a new therapeutic option for patients.

Professor Hwu is a leading figure in gene therapy in Taiwan. During his tenure at the Department of Pediatrics and Institute of Genetics at National Taiwan University Hospital (NTUH), he successfully developed the world’s first AAV-based gene therapy for Aromatic L-amino Acid Decarboxylase (AADC) deficiency. It was the first gene therapy in Taiwan to be licensed to a global pharmaceutical company, receiving marketing authorization from the EU in 2022 and the US in 2024. His accomplishment in gene therapy has inspired the scientists domestically and internationally. Following his retirement from NTU, Professor Hwu has joined China Medical University Hospital in Taichung, Taiwan to continue his pursuit in gene therapy development for various rare genetic diseases, including Sialidosis.

TFBS Bioscience began as a biosafety testing organization and has since become Taiwan’s first—and only—GMP-certified viral vector CDMO. It provides comprehensive one-stop services for biopharmaceuticals and cell and gene therapy products, covering key stepsfrom method development to manufacturing and quality control.

Having already brought real benefits to patients through his pioneering AADC gene therapy, Professor Hwu is now leveraging his experience to tackle Sialidosis. With the support of the NTUH Center for Frontier Medicine, his team has successfully demonstrated therapeutic efficacy in a mouse model. Forward-looking planning is now underway with the goal of accelerating the path to clinical application and bringing hope to patients suffering from this rare disorder.

TFBS Bioscience, selected as the manufacturing partner, demonstrates Taiwan’s robust capabilities in gene therapy development and manufacturing. This collaboration offers new hope to Sialidosis patients worldwide, proving that Taiwan can play a critical role in the advancement of gene therapy from research to product realization.