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.