Rare Diseases

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

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…