Dolly's parent a Californian

May 14, 1999

The Roslin Institute's new US backing will boost gene therapy research. Olga Wojtas reports.

Last year, the Roslin Institute, home of Dolly the sheep, set up a commercial subsidiary, Roslin Bio-Med, to capitalise on its pioneering technology. Last week, Roslin Bio-Med was taken over by a major American biopharmaceutical company in a deal which wins the institute Pounds 12.5 million in research funding over the next six years.

"We're not losing a daughter, we're gaining a son," said the Roslin Institute's director, Grahame Bulfield. "There will be a huge expansion of research in this institute."

Scientists at Roslin and the Geron Corporation of California hope that their complementary pioneering technologies will lead to new treatments for chronic degenerative diseases through human cell therapy.

While Roslin's Professor Ian Wilmut was developing the nuclear transfer technology that produced Dolly, Geron's researchers were working on cultivating embryonic human stem cells. Problems arise because the transplanted stem cells can be rejected, but combining Geron's research with Roslin's offers the hope of overcoming this, and creating transplantable, tissue-matched cells. Professor Bulfield said these could be used in a wide range of conditions where cells go out of control, such as diabetes, Alzheimer's and Parkinson's diseases, and some leukaemias.

"We are delighted that this agreement ensures a key role for United Kingdom scientists in the development of human stem cell therapies, potentially the most exciting application of the nuclear transfer technology that produced Dolly,'' he said.

Roslin is one of the world's leading centres for research on the genetics of farm animals, and Pounds 2.5 million of the applied research funding from Geron will support the institute's animal genome research programme. But the bulk, Pounds 10 million, will boost the development of nuclear transfer technologies, headed by Professor Wilmut and John Clark, a world expert on transgenic technology.

"The major challenge in making human cell therapy a reality is to understand the mechanisms involved in cellular reprogramming. This will be the focus of Geron-funded research over the next six years,'' Professor Wilmut said. "The long-term aim is to be able to reprogramme human cells without using eggs or creating embryos."

The Biotechnology and Biological Sciences Research Council provides around a quarter of Roslin's funding. The rest comes from contracts from a range of sources, including the Office of Science and Technology, the Ministry of Agriculture, Fisheries and Food, the European Union and industry.

Ray Baker, chief executive of the BBSRC said: "UK science is often criticised for not capitalising on its inventions. This agreement ensures the technology behind one of the great breakthroughs in biological science is going to be effectively applied to develop radical new treatments for disease."

Professor Wilmut's nuclear transfer discovery was spectacular, but it was not straightforward to take forward into commercial application, Professor Baker said. This was a complex process, not least because of issues of public acceptability, and how the public would respond to scientific developments in this area.

But Geron's discoveries were complementary to the nuclear transfer patent, and the intellectual property of the two organisations made them the ideal match to enable progress to be made. Human reproductive cloning is specifically excluded from the applications of the technology.

Professor Baker admitted that it was "a matter of mild regret" that Roslin's link was not with a UK company. "But I don't think the regret lasts very long. Science is a worldwide occupation. Since the match is so specific, it was always unlikely that it would be found easily in the UK. Geron is probably the only company in the world to provide it."

Professor Bulfield said the chances of finding a partner in the United States had been high, since 80 per cent of biotechnological research was carried out there. But Roslin, on the outskirts of Edinburgh, benefited from the broadest amount of biological research of any city in the UK.

"It's extremely important that the scientific atmosphere in Edinburgh is so vibrant. There are 1,000 biology graduates a year from the three universities, and it's a city people want to stay in when they graduate."

Just over a decade ago, PPL Therapeutics, an entirely independent company, was formed to commercialise Professor Clark's research into transgenic animals which would secrete human proteins in their milk. At that point public sector organisations were not allowed to take equity in spin-off companies.

Roslin still has a close relationship with PPL, and has licensed the nuclear transfer technology to it for the production of therapeutic proteins. But all other applications of the technology have been licensed to Geron. Under new rules, Roslin Bio-Med gave the researchers the capacity to own shares, trade them, and feed the proceeds back into the institute.

The shareholders in Roslin Bio-Med are the Roslin Institute and investment capital company 3i, which produced its initial backing. They will exchange their shares for 2.1 million shares in Geron, with Roslin Bio-Med becoming a wholly owned subsidiary of Geron. The Californian company is anxious to develop links with academic and commercial partners following the new links between Geron's and Roslin's technologies.

Roslin Institute is not a venture capital organisation, said Professor Bulfield, but has signed the Geron deal because it wants to release equity to support more research - which in turn is expected to generate more funds to support more research.

"I call it a virtuous circle. This is the first deal we've done, but you can expect to see more in the longer term."

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