What happened?

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.

So what

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.

Upward pointing arrows on a blackboard.

Image source: Getty Images.

Meanwhile, Orchard Therapeutics also reported preliminary results from a proof-of-concept study for OTL-201. This experimental treatment targets patients with mucopolysaccharidosis type IIIA (MPS-IIIA), a rare and progressive disorder that affects the nervous system.

Three patients participated in that study, and OTL-201 was well tolerated by them. The biotech also reported encouraging biomarker data from this study. It will host a webinar at 4:30 p.m. EST Tuesday to review and discuss this data.

Now what

It is too early to say what these results might mean for shareholders. Obviously, if Orchard Therapeutics goes on to market these treatments, it would be a big deal for the healthcare company. On the other hand, it still has a long way to go before that happens, if it happens at all, since OTL-201 and OTL-203 could still fail to prove sufficiently effective to pass regulatory muster in larger clinical trials.

It's worth monitoring the development of these programs, but there are other reasons to consider purchasing shares of Orchard Therapeutics. These include the recent approval of Libmeldy in Europe as a treatment for metachromatic leukodystrophy (MLD). Libmeldy is the first therapy approved for early-onset MLD, and the company said it would launch the treatment during the first half of 2021. This  will improve Orchard Therapeutics financial situation significantly, and help the company fund its clinical trials for its promising pipeline candidates. 

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