Orchard Therapeutics Posts Long-Term Data For Gene Therapy In Inherited Immune System Disorder

Three years after receiving gene therapy from Orchard Therapeutics ORTX in an early-stage clinical trial, 100% of patients with an inherited disease that damages the immune system were still alive.
Data from a study that used Orchard’s gene therapy OTL-101 to treat adenosine deaminase severe combined immunodeficiency (ADA-SCID) were published in the New England Journal of Medicine.
Results showed 100% overall survival and more than 95% event-free survival (defined as survival in the absence of enzyme replacement therapy reinstitution or rescue allogeneic hematopoietic stem cell transplant (HSCT)) at two and three years.
Results also showed sustained ADA gene expression, metabolic correction, and functional immune reconstitution in 48 out of the 50 patients.
The data came from three-phase 1/2 studies conducted in the U.S. and U.K., and through a compassionate use program in the U.K., thirty of the U.S. patients received Orchard’s OTL-101.
All of the patients experienced adverse events, but Orchard described them as mostly mild or moderate.
However, four patients did experience immune reconstitution inflammatory syndrome, a serious complication that can occur when the body recovers from treatment with antiretroviral drugs.
The condition flared up in patients between three months and 22 months after receiving the gene therapy.
Orchard said the complications were considered unrelated to the gene therapy, and the patients were treated successfully with supportive therapy.
Price Action: ORTX shares are up 0.39% at $5.16 during the market trading session on the last check Tuesday.

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Orchard Therapeutics Posts Long-Term Data For Gene Therapy In Inherited Immune System Disorder

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