A word about the gene-editing technology, called Crispr-Cas9 – WSJ


There has been a lot of buzz going around about the Crispr-Cas9 gene-editing technology

But what it is? and what impact could it have on drug development, innovation and new treatments?

What it is?

A fascinating short video created by the McGovern Institute for Brain Research at MIT explains the key characteristics of the technology.

Why is it so exciting?

A Wall Street Journal article reveals what this technology could allow regarding the development of potential new treatments and why it is so exciting.

This new technology will enable the “editing” of defective genes and their replacement by healthy (or normal) genes. Pharmaceutical companies are just starting to explore how to use it as drug discovery would probably be revolutionized. Diseases like cancer, cystic fibrosis,… caused by a mutated gene will eventually find a solution.

Another key advantage is the ease of use. Unless other technologies, Crispr-Cas9 gene-editing technology is much easier to handle than prior methods.

Is it already used?

Yes, it is selected to create genetically engineered research animals for the time being but there is more to come…

Is it dangerous?

It could be, if it is not used correctly and ethically. In April 2015, Chinese scientists used this method to manipulate the DNA of human embryos. This raised fears that it could be used to tune babies up before birth. Manipulating life has to be ethically monitored in order to protect life.

In another article that was then retracted, it seems that Crispr technology caused nearly 2,000 unexpected mutations — 10 times what was previously observed (in two mice that it cured of blindness). It is not the “death” of the technology but it proved that we don’t know yet how it really works.

Studies, published in Nature Medicine, revealed that edited cells might cause cancer.

What are the technical hurdles to overcome before creating a drug treatment based on this technology?

Today we do not know how to deliver a drug that could target the right DNA in the right cells. Inactivated viruses or nanoparticles could lead be used as potential carriers.

What’s next?

Overcoming drug delivery hurdles, perfecting the technology, translating it into drugs able to cure genetic disorders will be a fantastic improvement in health sciences. It will bring hope to many patients suffering from these diseases without any cure today.

As the technology is very promising, scientists and universities are currently fighting on the legal battlefield to know who will be the owner of the patents and who will make money from the technology and its application. More details

Additional Articles

As it moves out of the lab, CRISPR brings tough questions into the clinic – STAT – October 2018

A flawed study shows how little we understand CRISPR’s effects – Wired – April 2018

Biotechnology: At the heart of gene edits in human embryos – Nature – August 2017

Redesigning Life: How Genome Editing Will Transform the World – London School of Economics Book Review – September 2016

Beyond CRISPR: A guide to the many other ways to edit a genome – Nature – August 2016

Do CRISPR enthusiasts have their head in the sand about the safety of gene editing? – STAT – July 2016

Federal panel approves first test of CRISPR editing in humans – Washington Post – June 2016

Riding The Gene Editing Wave: Reflections On CRISPR/Cas9’s Impressive Trajectory – LifeSciVC – May 2016

Scientists solve CRISPR’s “Energizer bunny” problem – STAT – April 2016

CRISP biotech Intellia strikes licensing deal with Regeneron, readies for IPO – BioPharmaDive – April 2016

Bill Gates, together with other well-known investors, recently invested in Editas Medicine as part of CRISP-Cas9 frenzy.

Medical specialists urge more debate on gene-editing technology – Reuters – September 2015

CRISPR player Intellia looks IPO-ready after $70M round – FierceBiotech – September 2015

Editing humanity – The Economist – August 2015

Eight Things You Might Not Have Known Until Editas Filed Its S-1 – Xconomy – January 2016

Historic CRISPR Patent Fight Primed To Become Head-To-Head Battle – Xconomy – January 2016

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