UK’s Regulatory Agency Grants Promising Innovative Medicine Designation to 'Bubble Baby' Disease Treatment

Orchard Therapeutics Limited, a clinical-stage biotechnology company developing transformative gene therapies for patients with severe inherited orphan diseases, announces that the UK Medicines and Healthcare Products Regulatory Agency (MHRA) granted a Promising Innovative Medicine Designation to OT...

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Bibliographic Details
Published inPharmaceutical Processing
Main Author Orchard Therapeutics
Format Trade Publication Article
LanguageEnglish
Published Rockaway Advantage Business Media 23.08.2017
Subjects
Medicine
Regulatory agencies
University colleges
Los Angeles California
United Kingdom > UK
United States > US
California
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Summary:Orchard Therapeutics Limited, a clinical-stage biotechnology company developing transformative gene therapies for patients with severe inherited orphan diseases, announces that the UK Medicines and Healthcare Products Regulatory Agency (MHRA) granted a Promising Innovative Medicine Designation to OTL-101, its lead program for the treatment of adenosine deaminase severe combined immunodeficiency, commonly known as ADA-SCID or “bubble baby” disease. Prof. Bobby Gaspar, Orchard’s Chief Scientific Officer and Professor of Pediatrics 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”.
ISSN:1049-9156