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Gene editing breakthrough to power next biotech boom

By Marek Poszepczynski and Ailsa Craig, co-investment managers of the International Biotechnology Trust. 

Gene editing has made headlines in recent weeks, as the revolutionary technology has made breakthrough progress in treating serious genetic diseases and shown promising signs of blocking viral infections – such as Covid-19.

For example, US biotech firm Intellia Therapeutics announced the first successful treatment of human patients suffering from a serious genetic disease using CRISPR gene therapy, while Australian researchers reported using the same technology to stop the transmission of Covid-19 in infected human cells.

CRISPR technology is based upon a naturally occurring ancient form of protection against viruses found in bacteria. This gene editing technology has only recently been leveraged by science, but the potential lifesaving applications are myriad. Gene editing could notably help treat some of the world’s most complex genetic disorders that are currently without cure, and we believe the latest discoveries are only the first in a series of many more to come.

Gene editing will bring about a step change in drug development and open the doors to a new world of investment opportunities, which will continue to grow as the technology is refined and gains in prominence. We expect these developments to trigger a flurry of activity in the biotechnology sector, which investors will not want to miss.

What is CRISPR?

The technology in question is called CRISPR and stands for ‘clustered regularly interspaced short palindromic repeats’ – a family of DNA sequences found in bacteria. These bacterial genes act as a defence mechanism against invading viruses, attacking genetic sequences carried by a virus, which they recognise as threatening. The CRISPR genes produce a protein called Cas9, which acts as a ‘molecular scissor’ able to cut the invading genetic material at a precise point, silencing the virus and blocking replications.

Since discovering the existence of CRISPR, scientists have found a way of exploiting it to fight debilitating diseases caused by genetic malfunctions. The disease targeted by Intellia Therapeutics is amyloidosis, where a toxic protein called TTR builds up in a patient causing heart and neurological damage. Current approved treatments attempt to silence, correct or destroy this protein. Instead, the gene-editing approach stops the formation of the protein altogether, using these natural scissors discovered in microbes.

However, CRISPR treatments remain in the early stages of testing. Intellia only just released landmark data from clinical trials in humans. While the trial showed a dramatic reduction in the toxic protein levels in humans with minimal side effects, it will be crucial to continue collecting data on this treatment to ensure the approach is safe and effective over the long term – as gene modifications are non-reversable.

Poised for take-off

While drug development is a long and rigorous process, and we are still years away from administering an approved CRISPR drug to patients, there is already a large and growing market of companies looking to tap the unexploited potential of CRISPR.

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