Orchard Therapeutics, a clinical-stage biotechnology company, has entered a new clinical manufacturing services agreement with PCT Cell Therapy Services, a Hitachi Group Company.
PCT is a provider of contract services for the development and manufacture (CDMO) of cell-based therapeutic and regenerative medicine products.
The agreement
PCT will provide GMP-compliant manufacturing services for Orchard’s lead product, OTL-101, an autologous ex-vivo gene therapy for the treatment of adenosine deaminase deficiency severe combined immunodeficiency (ADA-SCID).
This agreement represents another key step in Orchard’s strategy to establish a global supply chain to deliver autologous ex-vivo gene therapy medicinal products to patients with devastating genetic diseases.
ADA-SCID
ADA-SCID is a rare inherited disorder of the immune system 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, more than 40 ADA-SCID patients have been treated with Orchard’s autologous ex-vivo lentiviral gene therapy at University of California Los Angeles (UCLA), US; 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.
About Orchard
Orchard is dedicated to bringing transformative ex-vivo gene therapies to patients with rare diseases of high unmet medical need.
Orchard’s clinical development pipeline includes novel treatments for primary immune deficiency disorders and inherited metabolic disorders, including ADA-SCID as a lead programme and MPS-IIIA (Mucopolysaccharidosis IIIA, also called Sanfilippo syndrome type A), as well as other undisclosed late and early stage development programmes in other indications.