BioWorld International Correspondent
LONDON - The UK government announced £4 million (US$7.3 million) in grants to develop gene therapies for hemophilia A, muscular dystrophy and inherited childhood blindness, all diseases that are caused by single gene defects.
The money is part of a £50 million program announced in June to expand the use of genetics in the National Health System (NHS), with the aim of making the UK a leader in genetics-based health care.
The largest grant, of £1.6 million, was awarded to a consortium developing a "molecular patch" of antisense oligonucleotides to override the gene defect that occurs in 60 percent of muscular dystrophy cases, restoring production of the muscle protein dystrophin.
The consortium, led by Francesco Muntoni of Imperial College, London, said the money should enable it to move the treatment to Phase I trials. Jenny Versnel, head of research at the Muscular Dystrophy Campaign, who brought the consortium together, said, "It is hoped that the severity of muscular dystrophy could be reduced to a much milder form, improving quality and length of life." There are 1,800 boys with the fatal muscle-wasting disease in the UK at present.
In mouse models of the disease, dystrophin production has been initiated by injecting the antisense oligonucleotides directly into muscles, but the researchers aim to develop a delivery system to target all muscles.
A second grant of £900,000 was awarded to the Institute of Ophthalmology, University College London, to fund a gene therapy trial to treat a form of retinitis pigmentosa caused by a single gene defect. The researchers have shown that gene replacement in mice leads to the restoration of the photoreceptors destroyed by the disease.
The third award of £500,000 is to Oxford BioMedica plc, of Oxford, for further development of its Requinate treatment for hemophilia A. Requinate comprises the company's LentiVector gene-delivery system carrying a version of the human Factor VIII gene, modified to enhance production of the blood-clotting protein.
Although Oxford BioMedica has solved several problems encountered by others attempting gene therapy for hemophilia since it started the program three years ago, funding constraints forced it to mothball Requinate in favor of its oncology and neurotherapy products. The money will fund the first trial in the UK seeking to use gene therapy to treat hemophilia.
In addition, a total of £1 million was awarded to four projects looking at the long-term safety of gene therapy, including £450,000 for work on retroviral vectors.
Since the first gene therapy trial in 1993 there have been 90 clinical trials in the UK, involving more than 700 patients.
The £50 million three-year program to increase the use of genetics in the NHS also included £2.5 million for cystic fibrosis research, £4 million to fund pharmacogenomic research on existing medicines, £18 million to update genetic testing laboratories and money to pilot the introduction of large-scale genetic screening programs, and integrate gene testing into clinical specialties in hospitals and into primary care.