OTL-101 granted Rare Pediatric Disease designation

The US Food and Drug Administration has granted a Rare Pediatric Disease designation to OTL-101, for the treatment of adenosine deaminase severe combined immunodeficiency, commonly known as ADA-SCID or “bubble baby” disease.

OTL-101, UK clinical-stage biotech firm Orchard Therapeutics’ lead program, is being developed in collaboration with the University of California, Los Angeles (UCLA) and University College London (UCL)/Great Ormond Street Hospital (GOSH)..

ADA-SCID is a rare inherited disorder of the immune system. ADA-SCID is caused by mutations in the gene encoding for the enzyme adenosine deaminase, which result in a severe deficiency in white blood cells and life-threatening infections. In the absence of treatment, ADA-SCID is fatal within the first year of life.