Gene and cell therapy
Gene and cell therapy has the potential to transform medicine, providing long term and potentially curative treatment options for a wide range of diseases. Several highly promising cell and gene therapies have been launched recently, including GSK's StrimvelisTM and Novartis’s KymriahTM. We are a crucial partner to Novartis as we provide the lentiviral vectors used in reprogramming patients T-cells with Novartis's CTL019 therapy.
At Oxford BioMedica our goal is to build a sustainable and profitable biopharmaceutical company for our shareholders through the successful development and commercialisation of breakthrough gene and cell-based medicines that improve the lives of patients. We are doing this by exploiting our LentiVector® platform to develop our own products and to build partnerships with other companies working with lentiviral vector product.
The LentiVector® platform is an advanced lentiviral-based gene delivery system designed to overcome the safety and delivery challenges associated with earlier generation vectors.
LentiVector® Platform advantages over other types
– Large therapeutic payloads (up to 9kb)
– Permanent modification of dividing and non-dividing cells
– No pre-existing immunity
The platform is applicable in many therapeutic areas, and has a number of specific advantages. Lentiviral vectors can genetically modify dividing cells, such as T-cells, as well as non- or rarely dividing cells, such as neurons or early progenitor/stem cells, making it a delivery system of choice in gene and cell therapy. The platform can also integrate genes into non-dividing cells, including in the brain and retina, with ground-breaking long-term studies suggesting gene expression may be maintained indefinitely, offering the prospect of permanent therapeutic benefit following a single administration. The LentiVector® platform is also used as valuable research tool, with applications in transgenesis, stem cell manipulation, somatic disease models, target validation and gene discovery.
CAR-T therapy involves extracting patients’ T cells and genetically modifying them via a lentiviral vector to express artificial T-cell receptors (known as chimeric antigen receptors – CARs) that are specific for particular antigens on cancer cells.
The cells are expanded until the population numbers billions and then reinfused back into the patient, where guided by the engineered receptors they recognise and kill the cancerous cells. This highly promising approach has led to dramatic response rates in a number of studies.
As gene and cell therapy pioneers we have built an enviable intellectual property position that protects our technology and products as well as our partners’, providing us with upfront fees and royalties on future sales.
Oxford BioMedica actively manages its intellectual property estate to provide robust protection for its products and platform technologies and to identify and protect new inventions. We have comprehensive know-how across its product pipeline particularly in the area of lentiviral vector manufacture and analytics, regulatory aspects of gene and cell therapy, and the pre-clinical and clinical development of gene and cell therapy products.
We are committed to advancing the wider development of gene and cell therapy approaches by offering licences under its intellectual property, provided these do not overlap with products the Group is developing itself.