(Reuters) – Sangamo Therapeutics Inc’s shares sank 27 percent on Thursday after its gene-editing drug failed to show promise in an early-stage trial testing it in patients with ultra-rare disorders.
Data suggested that genome-edited liver cells may be able to generate active IDS enzyme, a lack of which is a genetic mutation that leads to Hunter syndrome. Its symptoms include hearing loss, poor peripheral vision and diarrhea.
The drug was also being tested in patients with Mucopolysaccharidosis Type I, an ultra-rare disorder.
Reporting by Manas Mishra in Bengaluru; Editing by Sai Sachin Ravikumar