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 recently announced that it has entered a new clinical manufacturing services agreement with PCT Cell Therapy Services, LLC, 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.

Stewart Craig, Ph.D., 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 program for the treatment of children afflicted with ADA-SCID.”

Robert Preti, Ph.D., Chief Executive Officer and President of PCT said, “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 program."

"The clinical results are cause for hope among this patient population and we look forward to helping advance this important new therapeutic towards commercialization.”, he added