Advancements in Gene Therapy for Rare Ocular Disease and Metabolic Disorders with Guang Qu NGGT Corporation

Empowered Patient Podcast

Guang Qu, Co-Founder of NGGT Corporation is focused on developing gene therapy products using dual functional vector strategies. Guang highlights the importance of distinguishing between recessive and dominant mutations in rare diseases and explains how their approach differs from other gene therapy approaches. The two leading indications caused by a gene mutation that NGGT is working on are Bietti's Crystalline Dystrophy (BCD), a rare ocular disease, and phenylketonuria (PKU), a metabolic disease.

Guang explains, "For our strategies and our product development strategies, I think we are leveraging our experience in the gene therapy field. Later, I will talk more about myself, Dr. Lixin Jiang, and our team. The other thing we are leveraging is our fully integrated team in gene therapy product development, which involves R&D, research and development, CGMP manufacturers, from tox development and clinical regulatory functions and the medical teams. So with all of the teams we built up in the last couple of years, we're in very good positions in developing our gene therapy products."

"So, of the two currently leading indications, one is involved in ocular disease, what we call the BCD, Bietti's Crystalline Dystrophy. This disease is caused by a gene mutation. The gene mutation causes lipid metabolic problems. The particular gene is called the CYP4V2 gene. The mutation directly leads to the lipid metabolic process being interrupted. Therefore, lipids are precipitated in the different ocular cells, such as the cornea and the retina. That's most of the disease cell layers affected."

#AAV #GeneEditing #GeneTherapy #PKU #BCD

Download the transcript here