Gene editing: The biotech bringing potential new investment opportunities

The coronavirus pandemic has made biotechnology front page news. The arrival of multiple vaccines in record time has starkly highlighted how innovative – and versatile – the sector can be.

Its new position under the spotlight injected a healthy dose of momentum into what can at times be a volatile market and certainly investors have understandably been honing in on the biotech and pharmaceutical industries. In 2020 the NASDAQ Biotechnology Index delivered a 26% total return while the S&P Biotechnology Index achieved 48%, versus 18% from the wider S&P 500.¹

While the present focus is rightly on inoculating the global population against coronavirus, simultaneously there is a myriad of other work taking place, not least in the gene-editing space. A world away from the typical media portrayal of genetically modified ‘Frankenstein foods’ and ‘designer babies’, gene editing is a cutting-edge technique transforming scientific research, food production and ultimately how we can tackle life-threatening illnesses.

What is gene editing?

DNA is the blueprint of everything that makes us, us. It consists of 3.2 billion bases linked together into long chains arranged in a slightly different order from person to person. Gene or genome editing is a type of genetic engineering where DNA is deleted, inserted, replaced or altered in the genome of a living organism.

The unique sequence is ordered into genes, some of which direct the body to make proteins. The Human Genome project, an international effort, sequenced the first full human genome in 2003.²

The completion of the project arguably marked the start of a new era of medical innovation in which scientists could investigate and comprehend the underlying genetic causes of disease. Rapid improvements in technology drove down the time and cost of sequencing - the first human genome took 13 years and cost $2.7bn. Today the equivalent of an entire genome is sequenced every second and can be done so for less than $1,000.³

Where are we now?

All this genetic data is rapidly improving our understanding of diseases because “all diseases have a genetic component”.⁴

While many diseases are complex, involving contributions from multiple genes and environmental stimuli, there are approximately 5,000 to 8,000 diseases caused by a single genetic mutation. This fact creates an opportunity to apply a novel technology, gene editing, with potentially curative results.⁵

There are several firms working in this field but perhaps the best-known gene editing tool is CRISPR/Cas-9. The scientists behind the discovery - Emmanuelle Charpentier and Jennifer A. Doudna - received the Nobel Prize in Chemistry last year.⁶

The tool can cut DNA in specific places with high precision. The technology is composed of two main parts; a targeting molecule known as a ‘guide’, and a cutting tool, or ‘endonuclease’ that cuts the DNA at the specific sequence.

Essentially it can help turn off a faulty gene or prevent suppression of another gene or create a space into which other genetic material can be added.

Track record

These tools are already being tested in clinical trials and showing life-changing and, potentially, curative results for two rare blood disorders, beta thalassemia and sickle cell disease - both of which prevent the production of normal haemoglobin, the protein that carries oxygen in our blood.

These are life-long illnesses for which there is currently no cure. But an ongoing clinical trial, funded by biotech company CRISPR Therapeutics in partnership with Vertex, is showing remarkable early results and providing reasons to believe a cure for these diseases may ultimately be achievable utilising novel technologies such as gene editing. In many serious cases of beta thalassemia, patients require regular blood transfusions to prevent anaemia.  Patients who enrolled in the trial required on average 15 transfusions per year before treatment, and six months post-treatment all patients were transfusion free. The trial is ongoing and new patient data is still emerging.⁷

This is just one example of the applications of gene editing - already efforts are underway to apply this novel technology to range of diseases affecting different parts of the human anatomy. For example, Editas Medicine is working on correcting gene mutations that cause rare causes of blindness and Beam Therapeutics is harnessing gene editing to treat protein deficiency disorders, and produce immune cells capable of better targeting cancer cells.

The long-term potential

Gene editing is already being used widely in scientific research to accelerate new discoveries - and it has the potential to revolutionise medicine. The technology has been known for decades, but we are still very much in the infancy of investigating its use in human disease.

Scientists from The National Human Genome Research Institute have made some bold predictions about the future of genomics. While all may not bear fruit, at least in the short term, these forecasts include, among others, that genomic testing will become commonplace in medicine, just like a standard blood test, that an individual’s complete genome sequence will be easily available on their smartphones – and that ultimately the science will help cure more genetic diseases than ever before.⁸

Of course, the science behind gene editing is deeply intricate; many cancers are caused by genetic mutations and so there remains concerns that incorrect editing could potentially cause disease. Therefore, companies and scientists have taken a stepwise approach to applying the technology to treat diseases.

As with all new technologies there are still hurdles to overcome. It takes time to move from proof of concept to broader commercial validation. Given the long-term potential, we believe this is something to keep an eye on as an investor and is an example of the tremendous innovation being conducted by scientists and biotechnology companies right now.