Date: 2016-09-21
Type of information: Presentation of results at a congress
phase:
Announcement: presentation of results at the 17th biennial meeting of the European Society for Immunodeficiencies (“ESID”) in Barcelona, Spain (21–24 September 2016) and at the European Society of Gene and Cell Therapy (“ESGCT”) conference in Florence, Italy (18–21 October 2016)
Company: Orchard Therapeutics (UK)
Product: autologous CD34+ haematopoietic stem cells transduced ex vivo with EFS lentiviral vector encoding for the human ADA gene
Action
mechanism: gene therapy.
Disease: adenosine deaminase deficient severe combined immunodeficiency (ADA-SCID)
Therapeutic area: Rare diseases - Genetic diseases
Country:
Trial details:
Latest
news: * On September 21, 2016, Orchard Therapeutics announced that some of its academic collaborators will be presenting an update from ongoing clinical studies with autologous CD34+ haematopoietic stem cells transduced ex vivo with EFS lentiviral vector encoding for the human ADA gene for the treatment of patients diagnosed with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). The clinical data, being generated by University College London / Great Ormond Street Hospital for Children NHS Foundation Trust (“GOSH”) and the University of California Los Angeles (“UCLA”), will be presented at the 17th biennial meeting of the European Society for Immunodeficiencies (“ESID”) in Barcelona, Spain (21–24 September 2016) and at the European Society of Gene and Cell Therapy (“ESGCT”) conference in Florence, Italy (18–21 October 2016). The two clinical studies with ex-vivo EFS lentiviral-vector-based gene therapy have been designed as non-randomized studies to evaluate the safety and efficacy of treatment. Main endpoints include overall survival, event-free survival, immune system reconstitution, ADA enzyme activity and safety parameters. Clinical data highlights: - As of September 2016, a total of 39 patients with ADA-SCID have been treated at GOSH and UCLA, with a follow-up of 1–55 months At ESID, Professor Bobby Gaspar and Dr Claire Booth, both from UCL, will present on 21 and 23 September 2016, respectively. Further data will be presented by Professor Donald Kohn, from UCLA, at ESGCT on 20 October 2016.
- The data presented at ESID and ESGCT are based on an analysis of clinical data from 32 patients
- All patients have survived (100% overall survival) and 31 out of 32 patients show immune reconstitution, with so far a favourable safety profile.
