Astra Zeneca drive to develop drugs from genome project

By Pallab Ghosh
Science correspondent, BBC News

image copyrightThinkstock

Astra Zeneca has announced a research programme to develop a generation of medicines to treat the genetic causes of many debilitating diseases.

It will be the first concerted use of an emerging technique called Crispr to "snip out" specific disease genes in order to discover drugs.

The technique is cheaper, faster and more accurate than current methods.

The research will be carried out with four leading academic and industrial gene-research centres across the world.

AZ vice-president Dr Lorenz Mayr hopes it will speed the development of medicines for illnesses including diabetes, heart disease and many cancers

"You can't stop the science," he told BBC News. "It is one of the biggest developments in the area of biotechnology in decades."

Many serious illnesses occur when genes in the human body go wrong.

The human genome project determined that humans had about 24,000 genes. These are found along the DNA double helix in every cell in the body.

The decoding of the human genome 15 years ago led to the hope that doctors would eventually identify faulty genes responsible for specific diseases and eventually develop medicines to treat them.

The principle is simple - drug companies would "snip out" the gene responsible for the disease from the patient's DNA, then use it to test drugs to see if they could fix the problem.

image captionHuman DNA contains about 24,000 genes, and diseases emerge when some of them go wrong

At the time, US President Bill Clinton said: "Our children's children will only know cancer as a constellation of stars," and hailed the completion of the project after a 10-year race that cost billions.

And Tony Blair, then UK Prime Minister, who joined the Mr Clinton by satellite from Downing Street, added: "Every so often in the history of human endeavour, there comes a breakthrough that takes mankind across the frontier and into a new era."

media captionScientists have created a new technique to "snip out" specific disease genes in order to develop drugs

Fifteen years on, one could wonder: "What new era?" There are only a handful of new medicines based on the human genome project, and, although Mr Clinton may eventually be proved right, cancer is still known as "cancer".

Progress has been hampered by two main factors. First, researches soon began to realise that most common illnesses were caused by any combination of tens of genes.

Second, the genetic techniques to snip out specific genes are expensive and take a long time. Researchers have to make what are in effect "genetic scissors" tailor-made to the gene they want to snip out. This process can take months for each and every gene.

But in recent years, scientists have developed a set of genetic scissors that can be quickly and cheaply tailored to cut out a specific gene.

And this technique, called Crispr, will be the focus of the research programme.

Dr Kosuke Yusa, of the Wellcome Trust Sanger Institute, one of the academic organisations involved in the project, said "this powerful technology" would enable "researchers to target genes with incredible specificity".

Also involved are the Innovative Genomics Initiative, a joint venture between the University of California, Berkeley, and University of California in San Francisco, the Broad Institute/Whitehead Institute and Thermo Fisher Scientific, which are both based in Massachusetts.

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