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OTL-101 (Orchard Therapeutics)

Products Next higher product group gene therapy_oo
Period Status 2017-08-22 development pc existent
Organisation Organisation Orchard Therapeutics Ltd.
  Group Orchard Therapeutics (Group)
  Original research University College London (UCL)

Orchard Therapeutics Ltd.. (8/22/17). "Press Release: Orchard Therapeutics Announces that OTL-101 Has Received a Designation as Promising Innovative Medicine by UK’s Regulatory Agency". London.

Orchard Therapeutics Limited (“Orchard”), a clinical-stage biotechnology company developing transformative gene therapies for patients with severe inherited orphan diseases, today announces that the UK Medicines and Healthcare Products Regulatory Agency (“MHRA”) granted a Promising Innovative Medicine 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. OTL-101 is developed in collaboration with University College London / Great Ormond Street Hospital (“UCL” and “GOSH”) and the University of California, Los Angeles (“UCLA”).

This designation indicates that ADA-SCID is a life-threatening condition with high unmet need despite currently available treatment options and that OTL-101 is likely to offer major clinical benefits to patients.

Prof. Bobby Gaspar, Orchard’s Chief Scientific Officer and Professor of Paediatrics at University College London / Institute of Child Health, commented: “We are delighted by the designation of OTL-101 as a Promising Innovative Medicine as it recognizes the significant unmet need in ADA-SCID as well as the major potential clinical benefits of OTL-101 over alternative treatment options”.

The designation as Promising Innovative Medicine is the first step of a two-step process under which OTL-101 can benefit from the Early Access to Medicine Scheme (“EAMS”). Nicolas Koebel, Senior Vice President for Business Operations at Orchard, added: “With this PIM designation we can potentially make OTL-101 available to UK patients sooner under the Early Access to Medicine Scheme”.

To date, over 40 ADA-SCID patients have been treated with autologous ex-vivo lentiviral gene therapy at Great Ormond Street Hospital (GOSH) in London, UK and UCLA in Los Angeles, California. 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.

Orchard’s development pipeline of autologous ex-vivo gene therapies includes novel treatments for primary immune deficiencies (such as ADA-SCID) and inherited metabolic disorders (such as Sanfilippo syndrome type A).


ADA-SCID is a rare inherited disorder of the immune system. The incidence of ADA-SCID is currently estimated between 1 in every 200,000 to 1,000,000 live births according to literature sources. The actual incidence could be higher. 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 Orchard Therapeutics Ltd.

Orchard Therapeutics is a privately-held clinical-stage biotechnology company based in London, UK and in Foster City, California. Orchard is developing transformative gene therapies for patients with severe and life-threatening inherited orphan diseases. Orchard works in partnership with some of the world's leading centres in gene therapy, including University College London, Great Ormond Street Hospital for Children NHS Foundation Trust, the University of Manchester and Central Manchester University Hospitals NHS Foundation Trust, the University of California Los Angeles and Boston Children’s Hospital. In 2016 the company was named a Fierce 15 Company by Fierce Biotech.


Orchard Therapeutics Limited
Sylvie Blanchier-Franklin
+44 (0) 20 3823 2149

Record changed: 2018-05-07


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