“Cystinosis is a debilitating and progressive disease, and new treatment options are sorely needed. The current standard of care does not avert deterioration; at best, it can attenuate symptoms. That’s why gene therapy is particularly exciting: It has the potential to change the course of disease — and the lives of patients — by addressing the underlying cause of cystinosis,” said Birgitte Volck, MD, PhD, President of Research and Development at AVROBIO. “We believe we can engineer patients’ own stem cells so they sustainably produce the functional protein that is needed to prevent a toxic buildup of cystine and halt progression of the disease. We are so pleased that this investigational gene therapy is now in the clinic in collaboration with Dr. Stephanie Cherqui at UC San Diego.”

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