Orchard Therapeutics launched to treat rare child disease

摘要

University College London Business and venture capital firm F-Prime Capital Partners have launched a biotechnology company to create gene therapies to treat rare childhood diseases. The lead programme for Orchard Therapeutics will be for severe combined immunodeficiency caused by adenosine deaminase deficiency (ADA-SCID). Resulting in a severely compromised immune system, it is also called 'bubble baby' syndrome as any child with the disorder is often enclosed in a sterile environment to be protected from infection. ADA-SCID occurs in less than one in 100,000 live births worldwide.