- 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.
© 2025 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
Posted In:
Benzinga simplifies the market for smarter investing
Trade confidently with insights and alerts from analyst ratings, free reports and breaking news that affects the stocks you care about.
Join Now: Free!
Already a member?Sign in
