Skip to main content

Market Overview

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

Share:
Orchard Therapeutics Posts Long-Term Data For Gene Therapy In Inherited Immune System Disorder
  • 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.
 

Related Articles (ORTX)

View Comments and Join the Discussion!

Posted-In: adenosine deaminase severe combined immunodeficiency BriefsBiotech News Health Care Small Cap FDA General

Don't Miss Any Updates!
News Directly in Your Inbox
Subscribe to:
Benzinga Premarket Activity
Get pre-market outlook, mid-day update and after-market roundup emails in your inbox.
Market in 5 Minutes
Everything you need to know about the market - quick & easy.
Fintech Focus
A daily collection of all things fintech, interesting developments and market updates.
SPAC
Everything you need to know about the latest SPAC news.
Thank You

Thank you for subscribing! If you have any questions feel free to call us at 1-877-440-ZING or email us at vipaccounts@benzinga.com