Orchard’s efforts bear fruit

U.K.-based company receives $20 million for clinical trial of ADA-SCID

Kelsey Kaustinen
LONDON—Clinical-stage biotechnology company Orchard Therapeutics Ltd. got a boost this quarter as it, along with researchers from the University of California, Los Angeles (UCLA) Broad Stem Cell Research, received an award of $20 million from the California Institute for Regenerative Medicine’s (CIRM) governing board. This grant will be used to fund a new clinical trial for severe combined immunodeficiency caused by adenosine deaminase deficiency, also known as ADA-SCID, or “bubble baby” disease.
ADA-SCID is a rare, inherited immune disorder. The disease is caused by mutations in the gene that codes for the adenosine deaminase enzyme, and these mutations lead to a severe deficiency in white blood cells. As noted by the National Institutes of Health’s Genetics Home Reference website, “People with SCID lack virtually all immune protection from bacteria, viruses and fungi. They are prone to repeated and persistent infections that can be very serious or life-threatening. These infections are often caused by ‘opportunistic’ organisms that ordinarily do not cause illness in people with a normal immune system. The main symptoms of ADA deficiency are pneumonia, chronic diarrhea and widespread skin rashes. Affected children also grow much more slowly than healthy children and some have developmental delay.”
Without treatment, ADA-SCID is fatal within the first months of life, and it is estimated that this disease occurs in between one in every 375,000 to 660,000 live births, according to various literature sources.
Orchard scientific advisory board member Dr. Donald Kohn is leading this work as principal investigator, and commented that, “We are very grateful for this funding and for all the patients who have already gone through our clinical trials.” Kohn is a professor of pediatrics in the UCLA David Geffen School of Medicine, professor of microbiology, immunology and molecular genetics at UCLA and a member of the UCLA Children’s Discovery and Innovation Institute at Mattel Children’s Hospital.
Thus far, more than 40 patients with ADA-SCID have been treated in clinical trials with autologous ex-vivo lentiviral gene therapy at UCLA and the Great Ormond Street Hospital (GOSH) in London. A patient’s own stem cells are withdrawn, modified with a functioning copy of the missing or faulty gene and then transplanted back into the patient. The treatment has resulted in a 100-percent overall survival rate and has proven capable of restoring patients’ immune function with a favorable safety profile.
“Funding from CIRM will allow UCLA and Orchard Therapeutics to accelerate the development program with ex-vivo autologous lentiviral gene therapy, which could potentially greatly benefit patients,” Dr. Alexander Pasteur, Orchard’s interim CEO, said in a press release.
This is good news for a young company, as Orchard just launched in May 2016 with a £21-million Series A financing led by F-Prime Capital. Along with its launch, it announced formal partnerships with University College London, Great Ormond Street Hospital for Children NHS Foundation Trust, the University of Manchester, UCLA and Boston Children’s Hospital to develop transformative gene therapies for serious and life-threatening orphan diseases.
At the time, Bobby Gaspar, Orchard’s chief scientific officer and professor of pediatrics and immunology at the UCL Institute of Child Health and GOSH, remarked that, “Orchard’s founding scientists, also including Professors Adrian Thrasher and Waseem Qasim from UCL and GOSH, have been pioneering ex-vivo autologous hematopoietic stem cell gene therapy for the last 20 years. We have seen promising effects in several different diseases and are hopeful that this technology will change the lives of many children with life-threatening conditions in the future.”

Kelsey Kaustinen

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