Date: 2016-11-23
Type of information: Grant
Company: Orchard Therapeutics (UK) University of California, Los Angeles (USA - CA)
Investors: California’s stem cell agency (USA - CA)
Amount: $20 million
Funding type: grant
Planned used:
- The grant will fund a new clinical trial for Severe Combined Immunodeficiency caused by adenosine deaminase deficiency, commonly known as ADA-SCID or “bubble baby” disease. 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 months of life.
- To date, over 40 ADA-SCID patients have been treated in clinical trials with autologous ex-vivo lentiviral gene therapy at UCLA, Los Angeles and at the Great Ormond Street Hospital (GOSH) in London, UK. All patients have survived (100% overall survival) and the treatment has been shown to restore patients’ immune function, with a favourable safety profile.
- The treatment uses the patient’s own stem cells which are modified with a functioning copy of the missing or faulty gene before being transplanted back into the patient’s body. The use of the patient’s own cells (autologous) removes the need to search for a matching stem cell donor, which can take months or even years.
Others:
- • On October 26, 2016, Orchard Therapeutics and researchers from the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los Angeles, led by Orchard Scientific Advisory Board member Dr. Donald Kohn have been awarded $20 million by the California Institute for Regenerative Medicine’s governing board (CIRM).
Therapeutic area: Rare diseases - Genetic diseases
Is general: Yes
