‘Patients in a test tube’: Precision drugs heading our way, thanks to gene editing

Drug development is a risky business. More than half of candidate drugs that look promising in the research lab will ultimately fail. More than a quarter of drugs that reach the clinical trial stage will be rejected as ineffective. However, the wealth of genomic information now available through public databases - in particular, the rapidly growing number of known associations between diseases and specific genes - may significantly improve the drug-development success rate. At least, success rates will improve if drug developers let genomics guide their choice of molecular targets for research, according to a team of pharmaceutical industry and academic scientists.
[Gene editing] has spurred a wave of activity in the biotech sector, with several companies now offering a suite of complex gene editing services that big pharmaceutical companies are more than happy to pay for.

“It has transformed from something that is fantastically difficult to carry out into a day-to-day laboratory technology,” says Dr. Mike Mitchell, an analyst at Panmure Gordon. “It is now vital in both drug discovery and diagnostics and it’s oncology and precision medicine that are driving this.”


One such company is Horizon Discovery. … Horizon sells extremely complex genetically engineered cell lines to researchers around the world, helping them find cures for diseases like cancer.

These cells can either be made bespoke or bought off the shelf from Horizon’s portfolio. They are effectively “patients in a test tube”, explains Disley. They can be engineered to have certain characteristics, like mutations, to represent healthy or diseased patients.

This allows scientists to use the cell lines to prove a hypothesis or to test a drug to see what happens. They are also being used in diagnostic tools and genetic screening, to determine which subset of patients might benefit from a particular treatment.


“In personalized medicine now, we are narrowing it down to the genetics of small patient groups because your body is the most complex non-linear organism there is,” says Darrin Disley, [CEO of Horizon Discovery].

One day, says Disley, there will be a toolbox of drugs targeted and developed for specific types of patients.

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post: How gene editing is revolutionising the pharmaceuticals industry

Outbreak Daily Digest
Biotech Facts & Fallacies
GLP Podcasts
Infographic: Here’s where GM crops are grown around the world today

Infographic: Here’s where GM crops are grown around the world today

Do you know where biotech crops are grown in the world? This updated ISAAA infographics show where biotech crops were ...
News on human & agricultural genetics and biotechnology delivered to your inbox.
glp menu logo outlined

Newsletter Subscription

* indicates required
Email Lists
Send this to a friend