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.
Unlock this article instantly by becoming a free subscriber.
You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.