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 – its
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.
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