UK-headquartered Oxford BioMedica manufactures lentiviral vectors, which are used to deliver personalised gene therapies into immune cells, for in vivo and ex vivo products.
The firm is the sole manufacturer of lentiviral vectors for Novartis’ chimeric antigen receptor (CAR) T-cell therapy, Kymriah (tisagenlecleucel), and has partnered with Bioverativ, Sanofi, and Orchard Therapeutics, among others, in the cell and gene therapy space.
Oxford BioMedica also has its own pipeline of treatment candidates across Parkinson’s disease, oncology, and ophthalmology.
With recent growth in the cell and gene therapy market – strengthened by CAR-T approvals in the US and EU – Oxford BioMedica’s chief technical officer (CTO), James Miskin, told us he predicts the lentiviral vector market to follow suit.
Biopharma-Reporter (BPR) asked Miskin (JM) about the Novartis deal, if Oxford BioMedica has the capacity to meet growing demand, and whether more could be done at a regulatory level to drive advanced therapy approvals.
BPR: What role does Oxford BioMedica play in the manufacture of Novartis’ Kymriah?
JM: Oxford BioMedica is the sole manufacturer of the lentiviral vector that encodes the CD19-directed chimeric antigen receptor (CAR) in Kymriah. Our commercial and clinical supply agreement, which was established in July 2017 and builds on earlier agreements from 2014, provides for the manufacture of lentiviral vectors used to generate Kymriah and other CAR-T products.
We are looking to work with other CAR-T companies in areas beyond those covered by our existing partnership with Novartis.
BPR: What challenges are currently facing the lentiviral space?
JM: Capacity is an issue in the industry, particularly as there are very few companies with the ability to manufacture gene therapies at commercial scale. We are able to meet the current demand with our existing facilities and, to prepare for future growth, we are expanding with the recent acquisition of a 84,000-square-foot (7,800m²) manufacturing facility.
The facility will include approximately 45,000-square-foot (4,200m²) of four good manufacturing practice (GMP) cleanroom suites for vector production, and two fill/finish suites, as well as warehousing and quality control (QC) laboratories – with space available for future expansion. Once open in 2020, the facility will more than double our bioprocessing capacity.
BPR: Is staffing an issue in the industry?
JM: The importance of people to our endeavours cannot be underestimated. Being in Oxford we are fortunate to be in a good place to attract the best talent. However, we also are working internally and with external agencies, such as the BIA, on initiatives to help grow the talent pool.
BPR: Could more be done at a regulatory level?
JM: The regulatory framework is also a significant challenge, as gene therapy is an evolving area. We work closely with the relevant regulatory bodies to bring our products through development.
Globally, there are various initiatives that agencies have put in place relevant to advanced therapies, not least Breakthrough Designation, or PRIME. We work with our partners and other organisations to provide input into developing regulatory requirements in order to help refine guidelines in various territories. By working collaboratively and sharing our expertise we hope to expedite development and help the industry deliver these important therapies to patients.
BPR: What do you predict for the future of lentiviral vector manufacturing in the UK, and beyond?
JM: We estimate that the global market for lentiviral vector manufacturing services was worth around $200m (€176m) in 2017 and will grow to approximately $800m in 2026.
We expect to capture 25-30% of that global market as we expand our partnerships and our manufacturing facilities and capabilities.
BPR: Looking now to Oxford BioMedica’s proprietary products, how is the firm contributing to the Parkinson’s disease treatment space?
JM: Oxford BioMedica has maintained a long-term interest in Parkinson’s disease gene therapy and carried out a Phase I clinical trial as early as 2008 with an early product candidate.
In 2018, we formed a partnership with Axovant Sciences, to whom we licensed one of our own gene therapy products known as AXO-Lenti-PD (previously OXB-102). AXO-Lenti-PD enables the expression of a set of three critical enzymes required for end-to-end dopamine synthesis in the brain.
In October, Axovant announced that the first patient in the clinical study had received treatment with the therapy, at the Clinical Research Facility of the University College London.
As chief technical officer of Oxford BioMedica, James Miskin oversees the company’s biomanufacturing and supply activities, as well as its process development work. Miskin is also a member of the UK BioIndustry Association (BIA) manufacturing advisory committee.
