Scientific split - the human genome breakthrough dividing former colleagues

Two groups are racing to establish scientific and commercial priority over the discovery of the decade

Science Editor

The money men have moved in on a new technique for editing the human genome that promises to revolutionise the way many human diseases will be treated in the future. But Big Money has in the process divided a scientific community into two competing camps.

Some might see it has healthy scientific rivalry spilling over into commercial competition which could ultimately speed up medical innovation. But behind closed doors there is a desperate race to establish scientific and commercial priority over what could be the scientific discovery – and intellectual property – of the decade.

On the one side is a consortium of world-class researchers led by French-born Professor Emmanuelle Charpentier who made a key discovery behind the Crispr gene editing technique and has been promised $25m (£16m) by a group of venture capitalists to commercialise her invention for medical use.

On the other side is her former colleague and the co-discoverer of the gene-editing process, Professor Jennifer Doudna of the University of California, Berkeley, who has joined a rival consortium of researchers with $43m in venture capital to advance the Crispr technique into the clinic.

Each group has recruited a formidable panel of senior scientists as advisers. The Charpentier team, called Crispr Therapeutics, includes Nobel Laureate Craig Mello, the co-discoverer of a gene-silencing technique known as RNAi, and Daniel Anderson of the Massachusetts Institute of Technology, who was the first person to show that Crispr can cure a genetic disease in an adult animal.

Meanwhile the Doudna team, known as Editas Medicine, includes the Harvard geneticist George Church, a pioneer in synthetic biology, and Feng Zhang of MIT and the Broad Institute, who successfully managed to get Crispr to work in human cells and was this month awarded the first US patent on the technique – much to the dismay of Professor Charpentier.

“I have to be careful what I say here. It is very surprising. But the fundamental discovery comes from my laboratory and no-one has told me that they have scooped me,” Professor Charpentier told The Independent.

“Be certain that this discovery did not happen only by chance. I have been thinking, defending and carrying this study from Austria to Sweden and now Germany,” she said.

Patent attorneys are now pouring over the rival patent applications, in particular the claims relating to who has priority over a key element of the Crispr technique called Cas9, a bacterial gene for an enzyme that snips both strands of the DNA double helix at the same place – a key feature of the gene-editing process.

Professor Charpentier said that she identified Cas9, the most important fundamental discovery behind Crispr (pronounced “crisper”), when she worked at Umea University in Sweden, before she had teamed up with Professor Doudna to co-author a scientific paper on Crispr-Cas9 published in August 2012 in the journal Science.

Professor Charpentier, who is now at the Hannover Medical School in Germany, said that Cas9 was in fact described for the first time in an earlier scientific paper, published in Nature in March 2011, under its former name of Csn1, which she had isolated from the bacterium Steptococcus pyogenes.

“I was the scientist who described the technology and I kept the intellectual property when I was in Sweden….Editas does not have access to the intellectual property of the patent where I’m the co-inventor,” Professor Charpentier said.

“I made the decision to do something in Europe and I made the decision not to do something with Editas Medicine. I’m trying to remain true to myself….The aim is to use Crispr-Cas9 as a kind of genetic medicine to treat serious diseases. The point about Cas9 is that it works in every cell and in every organism tested – it’s mind blowing,” she said.

For her part, Professor Doudna said there is room for both camps to develop new medical therapies based on Crispr-Cas9.

“Emmanuelle and I are excited to see this platform employed to help patients, and there are of course many different targets and strategies to be taken, providing opportunities for multiple companies in this space,” Professor Doudna said.

“In addition to start-ups, many existing companies are also interested in using the technology for various applications that extend beyond human therapeutics. I expect that the commercial landscape will continue to evolve as the technology matures,” she said.

The main barrier to using Crispr-Cas9, however, will be its safe and efficient delivery to the cells and tissues that need the genetic therapy. Professor Mello believes that the first treatments are likely to involve the genetic manipulation of stem cells in the laboratory, before transplantation back into the affected parts of the body.

“Delivery to cells within the context of the whole body, brain or other organ is very difficult and inefficient… This is why Crispr therapies will no doubt be limited for the foreseeable future to applications where stem cells can be modified one, or a few, at a time and then reintroduced after double checking that only the intended change was made,” Professor Mello said.

WHAT IS CRISPR?

The human genome consists of a long sequence of "letters" written in the code of the genetic alphabet - the three billion base-pairs of the DNA molecule. The gene-editing technique known as Crispr-Cas9 is able for the first time to delete or swap any of these letters, right down to changing a single base pair at any designated place in the genome.

Crispr, which stands for clustered regularly interspaced short palindromic repeats, was originally discovered as a kind of immune system in bacteria to defend against invading viruses. Its potential for editing the genomes of animals, including humans, only came with the discovery of the Cas9 gene, an enzyme for cutting both strands of DNA, found in the bacterium Streptococcus pyogenes.

The commercial applications of Crispr, which could extend to treating genetic diseases and cancer, will depend to a great extent on Cas9 and who owns the intellectual property on this part of the invention. This is why patent disputes are likely to focus on who did what and when in terms of the Cas9 discovery. Emmanuelle Charpentier of Hannover Medical School in Germany and Jennifer Doudna of the University of California, Berkeley are both named as co-inventors on one patent.

 

Arts and Entertainment
books
Voices
Caustic she may be, but Joan Rivers is a feminist hero, whether she likes it or not
voicesShe's an inspiration, whether she likes it or not, says Ellen E Jones
Arts and Entertainment
The Doctor and the Dalek meet
tvReview: Doctor Who Into the Dalek more than compensated for last week's nonsensical offering
Sport
Diego Costa
footballEverton 3 Chelsea 6: Diego Costa double has manager purring
PROMOTED VIDEO
Life and Style
3D printed bump keys can access almost any lock
techSoftware needs photo of lock and not much more
Arts and Entertainment
The 'three chords and the truth gal' performing at the Cornbury Music Festival, Oxford, earlier this summer
music... so how did she become country music's hottest new star?
Life and Style
The spy mistress-general: A lecturer in nutritional therapy in her modern life, Heather Rosa favours a Byzantine look topped off with a squid and a schooner
fashionEurope's biggest steampunk convention heads to Lincoln
News
Dr Alice Roberts in front of a
peopleAlice Roberts talks about her new book on evolution - and why her early TV work drew flak from (mostly male) colleagues
News
i100
Arts and Entertainment
Star turns: Montacute House
tv
News
ebooksAn evocation of the conflict through the eyes of those who lived through it
News
i100Steve Carell selling chicken, Tina Fey selling saving accounts and Steve Colbert selling, um...
Arts and Entertainment
Unsettling perspective: Iraq gave Turner a subject and a voice (stock photo)
booksBrian Turner's new book goes back to the bloody battles he fought in Iraq
News
The Digicub app, for young fans
advertisingNSPCC 'extremely concerned'
News
i100
Arts and Entertainment
Some of the key words and phrases to remember
booksA user's guide to weasel words
Independent
Travel Shop
the manor
Up to 70% off luxury travel
on city breaks Find out more
santorini
Up to 70% off luxury travel
on chic beach resorts Find out more
sardina foodie
Up to 70% off luxury travel
on country retreats Find out more
Latest stories from i100
Have you tried new the Independent Digital Edition apps?
Independent Dating
and  

By clicking 'Search' you
are agreeing to our
Terms of Use.

iJobs Job Widget
iJobs General

Junior SQL DBA (SQL Server 2012, T-SQL, SSIS) London - Finance

£30000 - £33000 per annum + Benefits + Bonus: Harrington Starr: Junior SQL DBA...

C# Web Developer (ASP.NET, JavaScript, MVC-4, HTML5) London

£35000 - £45000 per annum + Benefits + Bonus: Harrington Starr: C# Web Develop...

Senior Data Scientist (Data Mining, RSPSS, R, AI, CPLEX, SQL)

£60000 - £70000 per annum + Benefits + Bonus: Harrington Starr: Senior Data Sc...

Law Costs

Highly Attractive Salary: Austen Lloyd: BRISTOL - This is a very unusual law c...

Day In a Page

The other Mugabe who is lining up for the Zimbabwean presidency

The other Mugabe who is lining up for the Zimbabwean presidency

Wife of President Robert Mugabe appears to have her sights set on succeeding her husband
The model of a gadget launch: Cultivate an atmosphere of mystery and excitement to sell stuff people didn't realise they needed

The model for a gadget launch

Cultivate an atmosphere of mystery and excitement to sell stuff people didn't realise they needed
Alice Roberts: She's done pretty well, for a boffin without a beard

She's done pretty well, for a boffin without a beard

Alice Roberts talks about her new book on evolution - and why her early TV work drew flak from (mostly male) colleagues
Get well soon, Joan Rivers - an inspiration, whether she likes it or not

Get well soon, Joan Rivers

She is awful. But she's also wonderful, not in spite of but because of the fact she's forever saying appalling things, argues Ellen E Jones
Doctor Who Into the Dalek review: A classic sci-fi adventure with all the spectacle of a blockbuster

A fresh take on an old foe

Doctor Who Into the Dalek more than compensated for last week's nonsensical offering
Fashion walks away from the celebrity runway show

Fashion walks away from the celebrity runway show

As the collections start, fashion editor Alexander Fury finds video and the internet are proving more attractive
Meet the stars of TV's Wolf Hall... and it's not the cast of the Tudor trilogy

Meet the stars of TV's Wolf Hall...

... and it's not the cast of the Tudor trilogy
Weekend at the Asylum: Europe's biggest steampunk convention heads to Lincoln

Europe's biggest steampunk convention

Jake Wallis Simons discovers how Victorian ray guns and the martial art of biscuit dunking are precisely what the 21st century needs
Don't swallow the tripe – a user's guide to weasel words

Don't swallow the tripe – a user's guide to weasel words

Lying is dangerous and unnecessary. A new book explains the strategies needed to avoid it. John Rentoul on the art of 'uncommunication'
Daddy, who was Richard Attenborough? Was the beloved thespian the last of the cross-generation stars?

Daddy, who was Richard Attenborough?

The atomisation of culture means that few of those we regard as stars are universally loved any more, says DJ Taylor
She's dark, sarcastic, and bashes life in Nowheresville ... so how did Kacey Musgraves become country music's hottest new star?

Kacey Musgraves: Nashville's hottest new star

The singer has two Grammys for her first album under her belt and her celebrity fans include Willie Nelson, Ryan Adams and Katy Perry
American soldier-poet Brian Turner reveals the enduring turmoil that inspired his memoir

Soldier-poet Brian Turner on his new memoir

James Kidd meets the prize-winning writer, whose new memoir takes him back to the bloody battles he fought in Iraq
Aston Villa vs Hull match preview: Villa were not surprised that Ron Vlaar was a World Cup star

Villa were not surprised that Vlaar was a World Cup star

Andi Weimann reveals just how good his Dutch teammate really is
Bill Granger recipes: Our chef ekes out his holiday in Italy with divine, simple salads

Bill Granger's simple Italian salads

Our chef presents his own version of Italian dishes, taking in the flavours and produce that inspired him while he was in the country
The Last Word: Tumbleweed through deserted stands and suites at Wembley

The Last Word: Tumbleweed through deserted stands and suites at Wembley

If supporters begin to close bank accounts, switch broadband suppliers or shun satellite sales, their voices will be heard. It’s time for revolution