Abstract

Analytical Testing Opportunities to Support Autologous Gene Therapy

Chaminda Salgado, Cell & Gene Therapy Medicinal Process Delivery Leader, GlaxoSmithKline

Autologous ex-vivo gene therapies are at the cutting edge of personalised medicines. However with such personalisation comes a huge supply chain challenge to ensure that the drug is manufactured, tested, and released in time to intercept the disease progression in the patient. It is a unique challenge never before seen in the small molecule or biopharmaceutical sectors. This presentation highlights the challenges involved. Following the successful launch of Strimvelis, the presentation explains GSKs current supply chain capability vs future ambitions in order to reach the patient populations affected by more common indications. Until now analytical testing meant that the pharmaceutical industry could afford to use extensive laboratory facilities, employing teams of staff, and spending months to test the products to ensure safe and timely release to treat millions of patients. Such a model is not possible for the future ambitions for autologous ex-vivo gene therapies in terms of efficiency, speed, and cost, and this creates a potential new market for Point of Care diagnostic type technologies.