MHRA backs gene therapy in battle with 'bubble baby' disease

22 August 2017

The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has granted a Promising Innovative Medicine Designation to OTL-101 for the treatment of adenosine deaminase severe combined immunodeficiency, commonly known as ADA-SCID, or ‘bubble baby’ disease.

This is a major boost for what is the lead program of UK and USA-based clinical-stage biotech Orchard Therapeutics, which is developing transformative gene therapies for patients with severe inherited orphan diseases.

OTL-101 is being developed in collaboration with University College London, Great Ormond Street Hospital (GOSH) and the University of California, Los Angeles (UCLA).

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