British Biotech Takes Over Development of Gene Therapy for Rare Immune Disease

18/12/2017 - 2 minutes

Orchard Therapeutics has licensed a gene therapy for the rare genetic disorder X-linked chronic granulomatous disease (X-CGD) from the non-profit Généthon. 

London-based Orchard Therapeutics is adding a new program to its pipeline of gene therapies. The company has obtained an exclusive license to a lentiviral vector to treat X-linked chronic granulomatous disease and the clinical trial data generated by its owner, the French non-profit Généthon.

X-CGD is an immune disorder caused by a mutation in the gene encoding for the gp91-phox protein — the catalytic subunit of the NADPH oxidase enzyme that neutrophils use to kill pathogenic bacteria and fungi. The mutation leaves children at risk of life-threatening infections followed by excessive inflammation. These episodes affect the quality of life and life expectancy of the patients, though routine prophylactic therapies help most survive at least until they’re 40.


The gene therapy at the center of the deal employs the lentiviral vector G1XCGD to engineer the patient’s own stem cells out of the body and correct the genetic defect before they’re infused back. The lentiviral batches Orchard will use as it takes over development will be manufactured by Yposkesi, an industrial gene and therapy platform created by Généthon, AFM-Telethon and Bpifrance’s Sociétés de Projets Industriels (SPI).

G1XCGD is currently being tested in a clinical trial, with 3 patients having received it so far. The program is being run with EU funding and has received orphan drug designation from the EMA.

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