A list of the world’s pharmaceutical and biotechnology companies and their areas of research will show that only a handful are targeting eye diseases. Oxford BioMedica, based at the Oxford Science Park, bucks that trend and is well advanced on treatments for macular degeneration, Stargardt’s disease, Usher’s syndrome, corneal graft rejection and chronic glaucoma.

The company was spun out of Oxford University by Professors Alan and Susan Kingsman.

It listed on the Alternative Investment Market in 1996, achieving a full listing in 2001. Glaucoma and macular degeneration are relatively well-known diseases. The former is caused by high intra ocular pressure (IOP) and if left untreated, can damage sight permanently and lead to blindness. Macular degeneration comes in ‘dry’ and ‘wet’ forms, of which the wet is the more severe. Blood vessels grow up behind the retina, which can become detached.

Wet age-related macular degeneration (AMD) tends to strike those in their 60s and 70s, impairing or destroying normal vision. Stargardt’s disease is quite rare, caused by an inherited genetic mutation. Unless a family is aware of its genetic history and organises regular checkups for offspring, it may lie undetected until a child is at least 10 and possibly older.

Like macular degeneration, central vision is affected, accompanied by wavy vision, blurriness and difficulty in adapting to dim light. Usher’s syndrome is also the result of a genetic mutation. It affects not only sight, but hearing too and is often diagnosed early because a child complains of deafness. Both Stargardt’s and Usher’s have no known cure. The kernel of the BioMedica therapies lies in delivering genetic material to the affected areas that will create its own ‘mini-factory’ for the correct cells that halt the degeneration.

The beauty of the therapy is that trials suggest a single treatment is sufficient to have a permanent effect. For Usher’s, Biomedica’s genetic intervention improves only sight, not hearing. Transporting the curative genes to their target needs a carrier. Oxford BioMedica’s is called LentiVector and forms the platform for a range of therapies. Lentivector is derived from a lentivirus, which has a long incubation period and is one of the best methods of gene delivery.

There are a number of lentiviruses. BioMedica uses a ‘stripped-down’ version of the equine infectious anaemia virus (EIAV). Chief scientific Officer Dr Stuart Naylor explained: “We strip out all the nasty infectious parts of the virus so it becomes a shell, then load it with the genetic material. “We have a great team of molecular biologists who can remove DNA and replace it with good genes.” While virus delivery systems are often engineered to pass harmlessly through the body and hit their exact target, the company’s approach is far simpler. For their ocular therapies, the treatment is injected directly into the target, the back of the eye. This is a major advance in patient care for those with wet AMD.

The current – and very expensive – treatment, Lucentis, means a needle in the eye around once a month. For a patient who may need the drug for 20 years or more, that is not good news. Nonetheless, Lucentis generates £2.3bn of revenue each year, so the outlook for RetinoStat is very strong. The strategy is to partner with major pharmaceutical companies.

The major collaboration is with Sanofi and covers RetinoStat for AMD, StarGen for Stargardt’s, UshStat for Usher’s, and EncorStat for corneal graft rejection. The first three are in clinical trials, the fourth has trials imminent. Such collaborations are revenue generating and in June 2012, Sanofi paid nearly £2m for options on StarGen and UshStat. Manufacturing the LentiVector range is a skilled and detailed operation. Every stage in the process must conform to the rigorous Good Manufacturing Protocol (GMP) standards.

Oxford BioMedica were lucky to acquire a GMP plant in Cowley for £1.9m in February 2011. Its in-house team converted the plant in 18 months for £1.5m. Certification by the UK’s Medicines and Healthcare Products Regulatory Agency came in June 2012. Building such a facility from scratch would have cost £20m. The alternative, outsourcing, involves greater risk, substantial costs and negotiating time slots with the third-party manufacturer.

Oxford BioMedica now employs 80 staff, of which 30 are engaged in manufacturing. As a PLC, the firm has access to major funding from companies such as M&G and Vulpes. Vulpes’ Martin Diggle is a non-executive director of BioMedica. The total amount raised since founding is £149m and the company is well funded until the first quarter of 2014.

Chief executive John Dawson’s track record is in growing pharma companies. He said: “We are targeting high-value, fast-growth markets. With very sound intellectual property and the GMP plant, we can offer the complete package to partners like Sanofi. “The LentiVector platform underpins a broad treatments’ pipeline and we have therapies for Parkinson’s disease and solid tumour cancers in development. “Our target markets offer multi-billon pound revenues and we aim to capitalise on them.”