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Back in 2012, researchers Jennifer Doudna and Emmanuelle Charpentier unveiled to the world CRISPR-Cas9 – a unique gene editing tool that has since been described as the scientific breakthrough of the century.
Thus far, it has lived up to the hype. CRISPR-Cas9 systems have ushered in a new era of genetic engineering, which has recently coupled with artificial intelligence to create a new generation of highly-effective gene-edited medicines that are currently in clinical trials.
We are, quite literally, a few quarters – if not months – away from gene editing turning into a disruptive reality.
But… (yes, there’s always a “but”)
Did you know that – for all its wonders – CRISPR-Cas9 actually has one major shortcoming as a gene editing tool?
Long story short, CRISPR-Cas9 requires cutting DNA strands open to edit the DNA sequence. But DNA is highly complex and super inter-connected… so, when you cut it open at a target location, other unexpected “things” tend to happen throughout the DNA sequence.
Those other “things” are called off-target effects – and they’re a big deal.
After all, you’re messing with DNA here… so an off-target effect could involve a change in DNA structure, which could fundamentally alter the genetic makeup of the genome.
Needless to say, off-target effects are a “must avoid” in CRISPR-Cas9 systems.
Yet, they’re always going to happen in these systems because you are physically cutting open the DNA, and there is simply no way to isolate the impact of that cut to the targeted location.
If only there were a way to edit the human genome without performing any cuts…
It was invented in 2016. It’s highly effective. It substantially limits off-target effects. And one small company is using this breakthrough genetic editing process to make what could end up being the world’s safest gene-edited medicines.
Today, we will tell you all about this highly differentiated gene editor, and show you why it could emerge as the biggest winner of the Genomic Revolution in the 2020s.
Pioneering a Gene Editing Breakthrough to Make Safer Genomic Medicines
Readers, meet Beam Therapeutics (NASDAQ:BEAM) – a highly differentiated, $6 billion gene editor that is pioneering a new evolution of gene editing techniques that could be the key to making safer genomic medicines.
Here’s the story.
Back in 2016, a team of researchers at Harvard led by David Liu worked endlessly to create a highly effective gene editing tool like CRISPR-Cas9 that didn’t require cutting, and therefore, didn’t have as many off-target effects.
Their work led them to inventing base editing.
Base editing is a hyper-simple form of gene editing that allows researchers to change individual letters of DNA. In practice, it converts one base pair in a DNA sequence to another base pair, thereby editing the genomic makeup, without doing any cuts, insertions, or deletions.
The result is a highly specific gene editing tool with substantially lower risk of off-target effects than CRISPR-Cas9 systems.
Following in the footsteps of the creators of CRISPR-Cas9 – both of whom went on to start their own CRISPR-Cas9 gene editing companies – Liu launched his own base editing company in 2018.
The name of that that company?
You guessed it. Beam Therapeutics.
Today, Beam Therapeutics features a robust drug pipeline that spans everything from treating sickle cell disease (with BEAM-101) to treating Acute Lymphoblastic Leukemia (BEAM-102) to potentially attacking the ocular and liver disease markets with a slew of gene-edited medicines in the research phase.
None of these drugs are in clinical trials yet.
But that’s not really all that important.
Sure, it means Beam is technically “behind the curve” when it comes to commercializing gene-edited medicines. But that’s to be expected, because Beam was founded less than three years ago, whereas the big gene editors are all 5+ years old.
Timing also isn’t what the Beam growth narrative is about…
Beam isn’t competing directly with CRISPR Therapeutics, or Editas Medicine, or Intellia Therapeutics. The biotech company is taking a fundamentally different approach with base editing that – if it works – will produce far safer and far more tolerable gene-edited medicines than any of those companies.
In other words, Beam has an opportunity to be the world’s leading gene editing platform company.
That’s a big deal, because there are over 6,000 genetic disorders in the world. They effect about 1 out of every 50 people. The treatment market for genetic disorders measures in excess of $50 billion.
Beam Therapeutics could be the leader in treating all of that.
And, to that end, Beam could one day be worth a whole lot more than its current $6 billion market cap.
All of that is just a long-winded way of saying that gene editing bulls should strongly consider taking a position in Beam stock today.
On the date of publication, Luke Lango did not have (either directly or indirectly) any positions in the securities mentioned in this article.
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