OXFORD, U.K.—At the beginning of December, Oxford Genetics, a synthetic biology company, announced a new agreement with clinical-stage gene therapy company MeiraGTx. The collaboration will enable Oxford Genetics and MeiraGTx to develop novel adeno-associated virus (AAV) vectors, as well as packaging and producer cell lines. The companies aim to create a fully scalable AAV production system that can satisfy the requirements for increased viral vector yields, process robustness and product efficacy.
“We are delighted to be working alongside the MeiraGTx team on such an exciting project, which will take us another step forward in our vision to deliver breakthrough technologies to the biotherapeutic industry,” said Dr. Ryan Cawood, CEO at Oxford Genetics. “By creating advanced AAV production systems, we aim to power the expansion of gene therapy as it moves towards addressing the more common disease indications that present technologies cannot satisfy.”
Dr. Paul Brooks, chief commercial officer at Oxford Genetics, adds, “The agreement further demonstrates that our business model for early access to our technologies is resonating with the biotherapeutic industry. Through such partnerships we will ultimately deliver real benefits to patients by helping our partners reduce therapeutic development time and achieving earlier market approval.”
According to Brooks, MeiraGTx and Oxford Genetics met at an Innovate UK (governmental) meeting, and from there began their collaboration. “Oxford Genetics was already committed to the process of developing improved AAV vectors, packaging and producer cell lines for the gene therapy industry, of which MeiraGTx is part. This collaboration and licensing agreement allows both companies to pool experience and expertise in this field and accelerate the development of these systems.”
When asked how long it may take to create a fully scalable AAV production system that can satisfy requirements for increased viral vector yields, process robustness and product efficacy, Brooks says the research and development of the AAV vectors, packaging and producer cell lines is expected to take between 12 and 18 months.
He also notes that, “AAV vectors are developed from single-stranded DNA viruses that belong to the Parvoviridae family. This virus is capable of infecting a broad range of host cells, including both dividing and non-dividing cells. In addition, it is a non-pathogenic virus that does not generate an immune response in most patients.
“Over the last few years, AAV vectors have emerged as an extremely useful and promising mode of gene delivery. This is owing to the properties of these vectors. AAVs are small, non-enveloped and have only two native genes. Thus [they] can be easily manipulated to develop vectors for different gene therapies. AAV particles are not easily degraded by shear forces, enzymes or solvents. This facilitates easy purification and final formulation of these viral vectors.”
“AAVs are non-pathogenic and have a low immunogenicity. The use of these vectors further reduces the risk of adverse inflammatory reactions. Unlike other viral vectors, such as lentivirus, herpes virus and adenovirus, AAVs are harmless and are not responsible for causing any human disease,” Brooks tells DDNews. “Genetic sequences up to 4000 bp (~4 kb) can be delivered into a patient using AAV vectors.”
As part of the agreement, MeiraGTx gains exclusive, worldwide research and manufacturing rights to novel serotype specific AAV vectors, along with associated packaging and producer cell lines. “We are very excited to begin this collaboration with Oxford Genetics. It’s one of many vital steps that we are taking to meet the demand for a broadening range of gene therapies. We hope that our approach will lead to quicker development of effective new therapies for patients,” commented Dr. Alexandria Forbes, president and CEO of MeiraGTx.
“Both companies see this initial collaboration as the first step in a wider and ongoing collaboration in the development and application of viral vectors and viral production cell lines for gene therapy,” Brooks finishes. “The excitement for us lies in bringing together tremendous talent from both organizations to tackle the challenges facing the efficient production of AAV for use in gene therapy.”