VectorBuilder supports first gene therapy trial for Menkes disease

VectorBuilder – a supplier in gene delivery technologies – has announced that its partnership with cell and gene therapy company, Lantu Biopharma, has led to the first human trial of an innovative gene therapy drug candidate for Menkes disease.

Key highlights:

• Lantu Pharma has developed an adeno-associated virus (AAV) drug, LTGT06, as a potential treatment for Menkes disease.
• Partnering with VectorBuilder has led to the first human trial for the gene therapy drug candidate.
• The trial human subject was a three year old boy who was diagnosed with Menkes disease when he was less than a year old.

Menkes disease is a type of disorder that is caused by a defect in the ATP7A gene, and affects the way the body processes and distributes copper levels. It is characterised by sparse hair, failure to grow in height and gain weight.

The adeno-associated virus (AAV) based drug, LTGT06, was developed by Lantu with crucial support from VectorBuilder. The trial subject was a three-year-old boy whose father gained worldwide attention for not giving up on his son.

Xu Haoyang, from China’s Yunan province, was less than a year old when diagnosed with Menkes disease, a congenital disorder arising from mutations in the ATP7A gene. Patients show severe neurological defects and typically do not live beyond three years. There is currently no cure for Menkes.

To save his son, Haoyang’s father Xu Wei, who has a high school education, taught himself chemistry and biology to build a home laboratory to synthesise copper histidine, a chemical shown to reduce Menkes symptoms but is not available in China. The father’s story of love and determination made international news while also generating debate over DIY medicine.

VectorBuilder formed a partnership with Lantu to develop AAV-based drug candidates for Menkes, providing CRO and CDMO services including AAV vector design and optimisation, and clinical-grade AAV manufacturing. Lantu studied the efficacy and safety of the candidates and collaborated with 920 Hospital and Hope of Health Hospital in Kunming to perform an investigator-initiated trial (IIT) to test the lead candidate, LTGT06, on Haoyang. After receiving official approval, the drug was administered shortly after the boy’s third birthday this year.

Xu Wei said: “I knew that copper histidine can only slow down Menkes, and gene therapy is the only hope for reversing the disease. We are now beginning to notice improvements. I am so grateful to see Haoyang’s condition improve for the first time in three years, rather than deteriorating. I am hopeful he will continue to get better."

VectorBuilder’s chief scientist, Dr. Bruce Lahn, commented: “VectorBuilder has supported gene therapy programs for many pharma and biotech companies along their entire pipelines, from early-stage R&D all the way to CDMO manufacturing. I am very pleased that we can play a role in the development of LTGT06. It is a testament to our technical capabilities and comprehensive end-to-end service capacity in the gene delivery space.”