Media Release: Scotland to produce treatment for haemophiliacs

Fraser Black with Richard Cruse ProFactor Pharma at BioCity Scotland

SCIENTISTS at ProFactor Pharma, a life science company moving to BioCity Scotland, are developing a novel manufacturing process for a drug to treat haemophiliacs.

Once commercial, the product is expected to create significant export income and high-grade employment opportunities in Scotland. In time it could also save healthcare providers globally millions of pounds annually in treatments for haemophiliacs.

Says a spokesperson: “Haemophilia is a bleeding problem. People with haemophilia bleed for a longer time than normal following bruising or injury, due to insufficient blood clotting factors being present in their blood.

“The bleeding can cause pain and damage to muscles and joints, in particular to knees, ankles and elbows. This can affect mobility, and in severe cases, with bleeding into internal organs, death is an outcome.

“Treatment for haemophilia today is very effective, with the injection of the missing blood factors. Bleeding is controlled once there is enough clotting factor circulating in the blood.

“With an adequate quantity of treatment products and proper care, haemophilia is no longer a life threatening condition, and haemophiliacs can live perfectly healthy lives. Without treatment, most children with severe haemophilia will die young (ref WFH).

“Haemophilia A is treated with replacement therapy of a human blood protein, Factor VIII.  Factor VIII, gram for gram, is probably the most expensive compound in the world, at some $4m per gram, and consequently an expensive treatment. There is also a real shortage of supply.”

ProFactor Pharma secured a funding round from Scottish venture capitalists, Kelvin Capital, earlier this year, enabling the company to progress development of their production process.

Richard Cruse, CEO and one of the founders of ProFactor Pharma said: “We established ProFactor Pharma to commercialise and supply recombinant blood factors for the treatment of haemophilia. We intend to supply product to countries which have been severely under-supplied up until now.

“We are confident our novel manufacturing process will enable product supply cost reductions, enabling many more patients to receive life saving treatment where few if any treatment options have existed until now.”

ProFactor Pharma was established in 2009 to commercialise and supply recombinant blood factors for the treatment of haemophilia. Its chief scientist is Dr Ian Garner, who identified the need to produce more clinical product, and believed this could be achieved in Scotland. The company recently appointed chair, Dr David Brown, who is an advisor to the Bill & Melinda Gates Foundation, which seeks to improve the health of populations in developing countries.

Fraser Black, managing director of BioCity Scotland, said: “We are delighted to welcome Richard and the ProFactor Pharma team to our facilities here at Newhouse. The work they are doing will make a significant contribution to world health and we are pleased that our facilities suit their purposes. BioCity Scotland is here to ensure everything is done to speed up the process they are beginning.”

Richard Cruse continued: “BioCity Scotland is able to provide our company with first class laboratory facilities and some of the equipment we need for our production processes on hire, saving us major capital investment and speeding up the process in which we can establish our laboratories. ”

Cruse trained as an accountant and moved into the biotech sector in 1987 eventually becoming financial director of PPL Therapeutics at Roslin, outside Edinburgh, the firm which helped produce the first cloned mammal, Dolly the Sheep. During that time, they raised over £125m of capital including floating the company on the London Stock Exchange.


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Contact: Carol Anderson
Phone: 0131 718 6022