Gene Editing, R&D

Sangamo claims a major milestone in gene editing, treating first patient with its zinc finger nuclease tech

While the leading CRISPR biotechs have been focused on a long, slow windup to their first human studies, one of the original players in the gene editing field has scored boasting rights for a first-in-human experiment.

Sangamo Therapeutics $SGMO has treated its first patient in a Phase I/II trial for a rare disease called Hunter syndrome, or mucopolysaccharidosis type II — MPS II. Physicians targeted the liver with SB-913, which uses Sangamo’s zinc finger nuclease tech delivered via AAV cells with a corrective gene. The ZFNs snip the targeted liver cells to insert the corrective gene at a specified point.

Sandy Macrae, CEO

By repairing the cells Sangamo says they should be good to go in producing an enzyme called iduronate-2-sulfatase, which is used to rid the cells of carbohydrates that accumulate in a toxic load, the hallmark of MPS II.

“Even with regular infusions of ERT, which has markedly improved functional health outcomes, patients endure progressive damage to heart, bones and lungs. Many patients with MPS II die of airway obstruction, upper respiratory infection or heart failure before they reach the age of 20,” said Paul Harmatz, a pediatric gastroenterologist and a principal investigator for the CHAMPIONS study.

Gene editing is an emerging field in the biotech world, and the top players here — including Editas, Intellia and CRISPR Therapeutics — have been scrapping over control of the CRISPR/Cas IP, even as researchers continue to work on new and better ways to go about this kind of DNA surgery. Sangamo, meanwhile, has labored in this field for more than 20 years with its ZFN tech, while TALEN is the focus at Cellectis.

The AP identified Sangamo’s patient 1 as 44-year-old Brian Madeux.

“It’s kind of humbling” to be the first to test this, Madeux told Reuters. “I’m willing to take that risk. Hopefully it will help me and other people.”


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