Why Orchard Therapeutics Stock Is Jumping Today

Shares of gene-therapy specialist Orchard Therapeutics (NASDAQ: ORTX) were trading sharply higher on Tuesday after the company released updates on two of its candidate treatments. As of 2:30 p.m. EST, Orchard stock was up by 10.7%, after having been up by as much as 21.9% earlier in the session.

Orchard Therapeutics focuses on developing treatments for rare diseases with unmet needs. The company's candidate OTL-203 is a potential therapy for mucopolysaccharidosis type I (MPS-I), a rare and progressively debilitating disorder. Patients with MPS-I fall into one of two categories: Those with the so-called Hurler subtype (MPS-IH) suffer from such symptoms as neurocognitive impairment and skeletal deformities, leading to impaired growth in early childhood.

Treatment options for MPS-IH are currently limited. In a proof-of-concept study that included eight patients with the condition, OTL-203 was generally well tolerated and showed encouraging efficacy. In particular, patients treated with OTL-203 showed stable cognitive performance, stable motor function, and improved range of motion compared to their conditions prior to treatment, among other improvements.

Source Fool.com