It is a technology hailed as one of the most important breakthroughs in human history – and it could be about to change everything it touches.
CRISPR (pronounced ‘crisper’) is a revolutionary gene-editing tool that allows scientists to make precise changes to the DNA sequence of living organisms – including human cells.
Emerging from a laboratory a little over 10 years ago, the genome editing tool has the potential to have a significant impact on the fruit on your supermarket shelves, tackling human disease, drug development or making crops immune to failure.
What is CRISPR?
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. It refers to a particular sequence found in the DNA of certain bacteria.
CRISPR technology enables scientists to target and modify specific genes within an organism’s DNA, similar to using a pair of molecular scissors.
It consists of two main components: the Cas9 protein, which acts as the scissors, and a small RNA molecule, which acts as a guide to direct the Cas9 to the desired target DNA.
Such has been its success and potential for genome editing so significant, its use has been swiftly adopted by many offering a broad range of opportunities for CRISPR investing.
For example, in Japan, startup Sanatech Seed launched genetically modified tomatoes to consumers at the end of 2021.
By using the CRISPR gene editing technology, it was able to produce the fruit with high levels of Gamma-AminoButyric Acid (GABA), an amino acid said to aid relaxation and help lower blood pressure.
It is likely to be only the tip of a very significant iceberg which CRISPR is fuelling.
According to a report published earlier this year by the Business Research Company, the CRISPR technology market, globally, will be worth USD1.65 billion this year and rise to USD3.7 billion by 2027 – exhibiting a CAGR of 22.9%.
But it is not without its challenges. And key in the vast majority of key markets will be regulatory approval.
The same report says:
“Stringent government regulations are expected to retard the growth of the CRISPR technology market during the period.
“There is no existence of internationally agreed regulatory framework for gene editing products and countries are in the process of evaluating whether and to what extent current regulations are adequate for…products related to gene editing.
“The Court of Justice of the European Union ruled it would treat gene-edited crops as genetically modified organisms (GMO); subject to stringent regulation.”
But that may not be the start and the end of the complications it faces.
While the potential to make huge steps forward remain clear, there are also the so-far unknown consequences of editing DNA which have yet to emerge.
For example, if you edit crops could it trigger allergies in people previously unseen?
There are, in short, lots of questions to be answered, but, for investors, enormous gains if it can leap the hurdles which lie before it in the years ahead.
CRISPR Industry Innovators
San Francisco-based Arsenal Biosciences is a privately held programmable cell therapy company discovering and developing a pipeline of “next-generation autologous T cell therapies to defeat cancer”.
It is “enabled by precise and specific CRISPR insertion of large synthetic DNA sequences”.
Last September, it closed a USD220 million Series B financing round. The funding came just ahead of clinical trials for a treatment for ovarian cancer.
US-based Intellia Therapeutics is less than 10 years old and was co-founded by Jennifer Doudna. It was she, along with colleague Emmanuelle Charpentier, who made the breakthrough with CRISPR – with the pair winning the 2020 Nobel Prize for Chemistry for their efforts.
Its researchers are working to harness CRISPR-based genome editing technologies for human therapeutic use.
It went public in 2016 and currently has a market capitalisation of USD3.7 billion. It has raised USD1.3 billion.
Swiss-American biopharma company CRISPR Technologies is focused on creating transformative gene-based medicines for serious diseases.
It is currently tackling the likes of sickle cell disease, various cancers, diabetes and cardiovascular diseases.
It was founded by Emmanuelle Charpentier in 2014 and saw an IPO two years later. Through a number of funding rounds it has raised more than USD125 million.
Its CEO, Samarth Kulkarni, said earlier this year:
“We and our partner Vertex have now completed regulatory submissions for exa-cel in the US, EU and UK. Which would position exa-cel to potentially become the first approved CRISPR-based therapy in the world – a remarkable pace of progress considering the discovery of the CRISPR platform took place a little more than a decade ago.”
Exa-cel stands for exagamglogene autotemcel – a treatment for sickle cell disease.
Beam Therapeutics is another biotechnology company engaged in creating genetic medicines based on its base editing technology. Formed just seven years ago, it has already raised USD689 million in post-IPO equity.
Locus Biosciences is a clinical-stage biotechnology company developing a new class of precision engineered bacteriophage treatments for a diverse set of bacterial diseases.
Last year, it completed a $35 million financing which included a Series B equity financing and conversion of an earlier convertible note. Notable investors participating in the round included Artis Ventures, Tencent Holdings, Viking Global Investors, Johnson and Johnson Innovation and Discovery Innovations.
Don’t be surprised to see the big established pharma companies keen to get onboard.
There is enormous, life-changing potential with the technology behind CRISPR and, potentially, untold riches to be made for the canny investor. Yet the regulatory hurdles it will have to leap could mean any returns are only visible as specks on the horizon rather than near term. And, of course, while regulations around the world adapt to the new technology, so money is burnt by all those seeking to emerge as the significant player. All of which means investors need to study the sector and understand the barriers to the progress this remarkable breakthrough offers.
Companies to Watch
Sanatech Seed, Arsenal Biosciences, Intellia Therapeutics, CRISPR Technologies, Beam Therapeutics, Locus Biosciences