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

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  • Three years after receiving gene therapy from Orchard Therapeutics (NASDAQ: 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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