We use cookies to help provide you with the best possible online experience. Please read our Privacy Policy for information about which cookies we use and what information we collect on our site. By continuing to use this site, you agree that we may store and access cookies on your device.

I Agree

You are here: Industry Insights > ADA-SCID

News List

Gene therapy successfully treats rare immunodeficiency in paediatric patients europeanpharmaceuticalreview
May 13, 2021
In three Phase I/II trials an experimental gene therapy caused all but two patients with severe combined immunodeficiency to acquire and retain robust immune function for more than two years.
Orchard Therapeutics Presents Two-Year Follow-Up Data Versus Historical Control from Registrational Trial of OTL-101 for the Treatment of ADA-SCID firstwordpharma
February 23, 2019
ADA-SCID is a rare, life-threatening, inherited disease caused by mutations in the ADA gene. Deficiencies of the ADA enzyme leads to dysfunction of cells of the immune system, including B, T and natural killer cells.
Orchard’s OTL-101 receives promising medicine designation for ADA-SCID in UK pharmaceutical-technology
August 24, 2017
Biotechnology company Orchard Therapeutics’ OTL-101 has received a promising innovative medicine designation from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for the treatment of adenosine deaminase severe combined immunodeficiency.