Wednesday, April 16, 2025

Beam Us Up, Biotech

By Jeff Brown, Editor, The Bleeding Edge
Managing Editor's Note: Before we get into today's TBE, I want to put something on your radar that our friend and colleague Larry Benedict has been working on…
As Jeff's been saying, there's no way around it… we can expect more volatility ahead for markets.
The good news is Larry has developed a time-tested strategy for profiting from the kind of wild swings we only see in times of extreme volatility. After all, traders like Larry thrive in these sorts of market conditions. He used this strategy to profit through the chaotic conditions of 2008, 2020, and 2022.
Next Wednesday, April 23, at 11 a.m. ET, he's sharing his blueprint for navigating this choppy market, including setting yourself up to profit from the swings and preparing for the next chaos catalyst that's coming.
You can go here to automatically sign up to attend. Now, on to today's Bleeding Edge…

One of the most painful things to watch in the markets right now has nothing to do with tariffs, trade negotiations, or bond market activity…
It's the market's treatment of the biotech industry which just can't seem to get a break.
It has been a remarkably long biotech winter which began in early 2021 when money printing became de rigueur, inflation went on a tear, and interest rates followed.
S&P Biotech Equally Weighted ETF (XBI) | Source: Bloomberg
If measured by the equally weighted biotech ETF (XBI), the industry is trading at levels that we've seen as far back as 2015. We've never seen a biotech bear market last this long.
This is ironic considering we've also never seen so much innovation and progress in life sciences and biotechnology than we have in the last three or four years.
One recent development in the last few weeks reminded me of that.
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But Larry Benedict has a time-tested system that thrives on volatility.

So far this year, he's used it to see gains as high as 97.70% in 13 days, 134.85% in 2 days, and 94.46% in less than a day.

And he's going to share it step by step on Wednesday, April 23rd, at 11 a.m. ET in an exclusive interview.

If you're even a little concerned about the markets, you need to be there.

Click to Automatically Add Your Name to Larry's Guest List >>>

Base Editing Breakthrough
Beam Therapeutics (BEAM) announced some incredible results for a genetic editing therapy that it has been developing for Alpha-1 antitrypsin deficiency (AATD).
AATD is a disease caused by a single-gene mutation in the liver that results in about 80% of patients having lung disease which includes early onset of emphysema, chronic cough, and shortness of breath.
Equally as bad, AATD often results in liver disease, which includes cirrhosis and cancer, often requiring a liver transplant. Severe cases can be life-threatening.
S&P Biotech Equally Weighted ETF (XBI) | Source: Bloomberg
The mechanics of this genetic disease are well understood. Most of us have the SERPINA1 gene in our livers which produces and secretes the healthy M-AAT protein. The M-AAT protein provides our lungs with protection from inflammatory mediators.
But, about 1 out of every 2,500 are born with an AAT deficiency. This is caused by a mutation of a single gene – the E342K mutation – which results in the Z-AAT protein (unhealthy) aggregating in the liver as opposed to the secretion of AAT to the lungs.
The decreased AAT in the lungs is what results in lung disease. And the aggregation in the liver, rather than secretion, is what results in liver damage.
And the worst part… there is no known cure and there is only one existing therapy which is very limited in its efficacy and doesn't actually do anything to treat the disorder.
The best the medical industry can do right now is a weekly injection of plasma-derived AAT given intravenously. This therapy does nothing to help the liver, and it only slows down the progression of the lung disease. And it's certainly not getting us any closer to a cure.
Beam Therapeutics BEAM-302 Therapy | Source: Beam Therapeutics
This is why Beam's recent data readout of its BEAM-302 genetic-editing therapy for AATD in its Phase 1/2 clinical trial is so exciting.
A Cure for AADT
Early this month, Beam published incredible results that demonstrated that, for its clinical trial patients, a mean of 91% of the total AAT in circulation in the lungs was the healthy M-AAT protein. There was also a mean decrease of 79% of the mutant protein – Z-AAT.
These results were recorded 28 days after treatment.
This was all achieved using CRISPR genetic-editing technology – the kind developed and patented by Editas Medicine (EDIT) – by using a CRISPR-related protein and a guide RNA to deliver the therapeutic payload to the precise location in a patient's DNA.
Beam Therapeutics uses what it calls "base editing" to make a single base edit – an enzymatic base conversion in the patient's DNA from one letter to another.
Based Editing | Source: Beam Therapeutics
In the case of AADT, it's as simple as changing an A to a G, and as we have learned from Beam's latest clinical trial results, the edit appears to be lasting and shows demonstrable proof that this may present a cure for AADT.
And the best part?
The therapy can be provided through a simple IV and done as an outpatient treatment. All it takes is just one visit. This is an in vivo treatment, the kind that Editas pioneered for genetic editing. And in this case, it doesn't require a needle precisely into the target area, just an IV into the bloodstream.
Of course, we're probably thinking that Beam's stock price went to the moon on the news. There are more than 100,000 people in the U.S. with AATD, and there is no FDA-approved cure for the disease.
BEAM-302 is the answer.
And yet, the market response was dismal.
Beam Therapeutics (BEAM) 1-Year Stock Chart
Beam is down more than 50% since its February highs. And get this, the stock is now trading below its early 2020 IPO price of $17.
And if that's not crazy enough, its current enterprise value is about $950 million, $850 million of which is cash. Absolutely nuts.
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Not Out of the Woods Yet
If the biotech market were healthy, Beam would be a screaming buy. But with interest rates as high as they are, and 10-year Treasury yields also elevated as high as they are, institutional capital is still steering clear of the early-stage biotech space.
It's frustrating to see such incredible developments like Beam's latest announcement go completely unrewarded by the markets.
But I know this won't last. The Federal Reserve will lower the Fed Funds rate at the next FOMC meeting in early May, which will be a step in the right direction.
Inflation is now at the lowest level in four years and interest rates are going to fall as things settle down in the coming weeks.
It's just going to take a little more patience before institutional capital piles into one of the greatest investment opportunities we'll see in a generation.
Jeff

Keep reading
Supercomputing is being completely redefined by the needs of artificial intelligence.
Intel's newly appointed CEO is wasting no time getting the company back on track…
Trump's long-anticipated tariffs and subsequent pause have had markets swinging hard.

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