AVROBIO, Inc. Expands Rare Disease Pipeline with Gene Therapy to Treat Cystinosis. Avrobio, Inc. is a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases. “This cystinosis program is a strong strategic fit... We are pleased to partner with Dr. Stephanie Cherqui and her team at the University of San Diego (UCSD)," said Geoff MacKay, AVROBIO’s President and CEO.
A life-threatening childhood disease and CIRM-funded team seeking the stem cell cure. Such cure is Clay Emerson’s biggest hope for his young daughter Brooke, who has cystinosis, a genetic disease that over time causes damage to many organs. UCSD researcher Dr. Stephanie Cherqui, received $5 million in CIRM funding to bring her team’s therapy to clinical trials in people.
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