Orchard Therapeutics Limited, a clinical-stage biotechnology company dedicated to bringing transformative ex vivo gene therapies to patients with rare diseases of high unmet medical need announces that it has entered a new clinical manufacturing services agreement with  PCT Cell Therapy Services, a Hitachi Group Company.

PCT is a leading provider of contract services for the development and manufacture (CDMO) of cell-based therapeutic and regenerative medicine products. PCT has previously provided a Strategic Manufacturing Assessment (SMA) and manufacturing process development services to Orchard.

Under the terms of this new 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).

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.

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.

Stewart Craig, PhD, Orchard’s Chief Manufacturing Officer, commented: “We are very pleased to extend our relationship with PCT into a full GMP manufacturing services agreement for OTL-101. As a world-leading CDMO for cell-based therapeutic products, this is an important step in advancing our lead programme for the treatment of children afflicted with ADA-SCID.”

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.

Robert Preti, PhD, Chief Executive Officer and President of PCT, commented: “Expansion of our agreement with Orchard to now include clinical manufacturing in support of their ADA-SCID gene therapy is testament to our successful collaboration and our dedicated stewardship of this important programme. The clinical results are cause for hope among this patient population and we look forward to helping advance this important new therapeutic towards commercialisation.”

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.