Orchard announces an alliance with PharmaCell B.V. (“PharmaCell”), a leading Contract Manufacturing Organization (CMO) for Cell and Gene Therapies and Regenerative Medicine.
Under the terms of the alliance, PharmaCell will provide GMP-compliant manufacturing services to support clinical trials and commercialization of Orchard’s ex-vivo autologous gene therapy products.
This agreement represents another important milestone for Orchard’s strategy to establish a global supply chain to deliver ex-vivo autologous gene therapy medicines to patients with devastating genetic diseases. Stewart Craig, Ph.D. Orchard’s Chief Manufacturing Officer commented: “We are delighted to partner with PharmaCell, a world-leading CMO with a proven track record in the manufacture and supply of cell-based products for both clinical trials and commercial markets. We are excited about the prospect for this partnership to accelerate our plans to make medicines available on a global basis.”
Orchard’s clinical development pipeline includes novel treatments for primary immune deficiency disorders and inherited metabolic disorders, including ADA-SCID (adenosine deaminase deficiency severe combined immunodeficiency) and MPS-IIIA (Mucopolysaccharidosis IIIA or Sanfilippo syndrome type A) as well as other indications.
Alexander Vos, Chief Executive Officer of PharmaCell, commented: “We are very excited to have the opportunity to collaborate with Orchard Therapeutics on their ground-breaking development pipeline for severely debilitating genetic disorders. PharmaCell will bring its extensive resources and experience to bear to support the global commercialization of these innovative therapies like we have been and are doing for an increasing portfolio of late-stage clinical programs for leading industry players.”
About adenosine deaminase deficiency severe combined immunodeficiency (“ADA-SCID”)
ADA-SCID is a rare inherited disorder of the immune system. The incidence of ADA-SCID is estimated between 1 in every 375,000 to 660,000 live births, according to literature sources. ADA-SCID is 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. Despite currently available treatment options, there remains significant need for therapies that reduce the mortality, morbidity and burden of disease on patients and families.