The UK Department of Health has awarded Oxford BioMedica £0.5 million to develop its proprietary LentiVector technology for use in the treatment of single gene inherited disorders
The focus of the work is to be haemophilia A, a condition caused by a defective gene for Factor VIII, a key component of the mechanism for forming blood clots.
The LentiVector technology, developed primarily for the company's neurotherapy pipeline, can be used in many areas outside the field of neurobiology.
It is ideally suited to the treatment of single gene disorders where therapy is based on functional correction of a defective gene in patients.
In this situation, there is a need for long-term, stable and predictable gene activity and this can be achieved with the LentiVector technology.
In 2001 the company initiated a programme to develop a product known as Requinate.
Requinate comprises a LentiVector gene delivery system carrying a modified version of the human Factor VIII gene.
During this exploratory programme the company solved several problems that had been encountered by others attempting gene therapy for haemophilia.
However, since haemophilia falls outside of the Company's therapeutic focus of cancer and neurotherapy, the programme has received minimal internal resources.
The new money from the Department of Health will enable the company to progress the Requinate haemophilia programme without compromising progress of its cancer and neurotherapy products.
The current market for Factor VIII treatment is in excess of $1.0 billion.
Commenting on the award, Oxford BioMedica's chief excutive, Alan Kingsman said "We are pleased to see the Department of Health implementing the government's decision to place gene-based medicine in the mainstream of healthcare in the UK.
"We are also delighted to be the only company selected to contribute to this initiative and to aid in the process of bringing products to the market that will directly benefit patients with genetic diseases.
"Oxford BioMedica is currently evaluating how it can expand the application of the LentiVector technology for other inherited disorders, in addition to haemophilia, while retaining its essential focus on bringing its current product pipeline in cancer and neurotherapy to the market as quickly as possible".
