Orchard Therapeutics launched to treat rare child disease
10 Jun 2016 by Evoluted New Media
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.
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.
Current treatments for this disease are either a bone marrow transplant, or lifelong therapy using replacement enzymes. Orchard Therapeutics has secured £21m worth of investment, led by F-Prime with support from University College London Business (UCLB) and participation from the UCL Technology fund.
Cengiz Tarhan, Managing Director of UCLB, said: “We are very excited to see UCL’s breakthrough treatments move forward in a commercial environment in a way that will benefit patients globally. The formation of Orchard represents the culmination of decades of research at UCL and its partners and we are delighted to be able to work with F-Prime Capital to launch Orchard.”
Phase 0 trials led by UCLB, Great Ormond Street Hospital (GOSH) and University of California Los Angeles (UCLA) showed significant immune reconstitution with 100% survival in 32 patients treated, as of March 2016.
Orchard has also announced formal partnerships with UCL, GOSH, UCLA, Manchester University and Boston Children’s Hospital for the development of these gene therapies.
