Orchard Therapeutics Announces Presentations from UCL/GOSH and UCLA Academic Collaborators on Clinical Data in ADA-SCID Using Ex-Vivo Lentiviral Gene Therapy at September 2016 ESID and ESGCT Congresses

London, UK, September 21, 2016 / B3C newswire / -- Orchard Therapeutics announces 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 ("UCL") / 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
-       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

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.

Presentations at ESID

  • Session name: Opening keynote lecture
  • Title of presentation: PID across frontiers - new paradigm
  • Presenter: Professor Bobby Gaspar
  • Date: Wednesday 21 September 2016
  • Time: 17.40-17.55 CET
  • Session name: PID across genetic frontiers
  • Title of presentation: Evolution of gene therapy for primary immune deficiencies
  • Presenter: Dr Claire Booth
  • Date: Friday 23 September 2016
  • Time: 09.00-09.30 CET

Presentation at ESGCT

  • Session name: Ex-vivo HSC based gene and cell therapy
  • Title of presentation: Gene therapy for primary immune deficiencies: ADA-SCID and XCGD
  • Presenter: Professor Donald Kohn
  • Date: Thursday 20 October 2016
  • Time: 14.00 CET

ADA-SCID is a rare inherited disorder of the immune system. The incidence of ADA-SCID is currently estimated between 1 in every 375,000-660,000 live births. ADA-SCID is caused by mutations in the gene encoding for the adenosine deaminase enzyme, which result in a severe deficiency in white blood cells and life-threatening infections.

About Great Ormond Street Hospital for Children NHS Foundation Trust
Great Ormond Street Hospital for Children Foundation Trust is the UK's leading centre for treating sick children, with the widest range of specialists under one roof. With the UCL Institute of Child Health, we are the largest centre for paediatric research outside the US and play a key role in training children's health specialists for the future. The Great Ormond Street Hospital Children's Charity needs to raise vital funds to enable the hospital to continue to provide the very best care for its young patients and their families through helping to rebuild and refurbish the hospital, buy vital equipment, fund pioneering research and provide support for staff and families.

About UCL
UCL was founded in 1826. It was the first English university established after Oxford and Cambridge, systematic teaching of law, architecture and medicine. It is among the world's top universities, as reflected by performance in a range of international rankings and tables. UCL currently has more than 35,000 students from 150 countries and over 11,000 staff. Annual income is more than £1 billion. Follow UCL on Twitter @uclnews. Watch UCL's YouTube channel YouTube.com/UCLTV.

About UCLA
UCLA is an international leader in the breadth and quality of its academic, research, health care, cultural, continuing education and athletics programmes. With more than 43,300 undergraduate and graduate students, it is the largest university in California. The UCLA College and 12 professional schools offer more than 5,000 courses, 124 undergraduate majors and 91 minors, 98 master's programmes, and 109 doctoral and professional programmes. Seven alumni and six faculties have been awarded the Nobel Prize. UCLA performs well in a wide variety of national and international university rankings.

About Orchard Therapeutics
Orchard Therapeutics is a clinical stage biotechnology company dedicated to bringing transformative ex-vivo gene therapies to patients with serious and life-threatening orphan diseases.


Sylvie Blanchier
+44 (0) 7469 935464

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