Building Gene Therapies that Address Gain and Loss of Function Simultaneously

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For some genetic diseases, there is not only the need to replace the function of a gene that is lost, but to also address toxicities that a mutated gene may cause. There is currently no available treatment targeting diseases that result from both loss and gain of function mutations. NGGT uses dual-functional vectors to simultaneously remove harmful, mutated genes and replace them with normal, healthy genes to restore cellular function. We spoke to Guang Qu, chief operating officer and co-founder of NGGT, about the company’s approach to gene therapies, how it is leveraging its platform technologies to cost-effectively accelerate development of these therapies, and the pipeline it is building.