Posted by Michael Wonder on 26 Jul 2017
Orchard Therapeutics announces that OTL-101 has received a rare paediatric disease designation
25 July 2017 - Orchard Therapeutics is delighted to announce today that the US FDA granted a rare paediatric disease designation to OTL-101, its lead programme for the treatment of adenosine deaminase severe combined immunodeficiency, commonly known as ADA-SCID or “bubble baby” disease.
TL-101 is developed in collaboration with the University of California, Los Angeles (“UCLA”) and University College London / Great Ormond Street Hospital (“UCL” and “GOSH”).
To date, over 40 ADA-SCID patients have been treated with OTL-101, autologous ex-vivo lentiviral gene therapy at UCLA, Los Angeles and at the GOSH in London, UK. All patients have survived (100% overall survival) with follow-up up to 5 years and the treatment has been shown to restore patients’ immune function, with a favourable safety profile.
