RARECast: Orchard Hopes to Bear the Fruit of Gene Therapy

July 1, 2016

Orchard Therapeutics, a London-based gene therapy company with facilities in the United States, unveiled itself in May with $30 million in funding. The company is developing gene therapies to treat rare diseases involving metabolic disorders and immune deficiencies. Orchard uses a patient’s own Hematopoietic stem cells, which are modified with a functioning copy of the missing or faulty gene, before being transplanted back into the patient’s body. We spoke to Andrea Spezzi, chief medical officer for Orchard about the company, its approach to gene therapy, and how its addressing the challenges of bringing such treatments to market.

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