Positive Updates for a New Potential Gene Therapy for Wiskott-Aldrich Syndrome

Wiskott-Aldrich Syndrome

Wiskott-Aldrich Syndrome (WAS) is a rare immune disorder which causes platelets in the blood to function abnormally. It also causes autoimmunity. Patients suffer from severe bleeding which can occur in various areas of the body including the brain and the gut. Bleeding and severe infections are the most common causes of death in WAS, with the median survival age being only 14 without treatment.

Some patients do have the option of undergoing a stem cell transplant however the procedure has a high level of risk of morbidity and even mortality.

Thankfully, researchers have been investigating a potential gene therapy for WAS which so far has had extremely promising results. It could become an alternative treatment option for WAS patients.


OTL-103 is a gene therapy that is stem-cell based. It is an ex vivo and autologous therapy being developed by Orchard Therapeutics. An interim analysis for this therapy has offered continued support for its continued development for WAS.

The interim results from the company’s registrational trial utilized a fresh cell formulation of OTL-103. Patients treated with the therapy had a significant reduction in the frequency and severity of both infections and bleeding episodes. No severe bleeding events were documented after the administration of the gene therapy. The follow-up period ranged from .5 to 5.6 years. Overall this data showed the gene therapy was both safe and effective for WAS patients.

These interim results were published in Lancet Hematology. The full data set should be released this year.

Open Label Study

Orchard is also conducting an open label study to evaluate a cryopreserved formulation of the therapy. They plan to enroll 6 patients in total, and they’ve just announced that the very first WAS patient has been dosed with the therapy. Ultimately, the company hopes to commercialize this formulation of the therapy, pending approval of course.

The primary endpoint of this evaluation is successful engraftment. This will be evaluated at 6 months.

Pending continued positive results, Orchard hopes to file for approval of this therapy in both the United States and Europe in the year 2021.

You can read more about this gene therapy investigation for WAS here.

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