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Nothing in the paper on how they plan to overcome NK cell rejection.
Even more preclinical data https://www.biorxiv.org/content/10.1101/2023.08.03.551705v1
How about treating someone?!
Here is your little slice of absurdity for Saturday morning. $edit even with all their CEO changes, CMO changes CFO changes and everything else STILL was able to treat sickle cell patients and report engraftment quicker than the time it will take $beam to treat their first…
— Arnold Emily (@crisprtalk) July 15, 2023
Nice one month move off the double bottom…
https://www.the-scientist.com/news-opinion/first-person-dosed-in-novel-gene-editing-clinical-trial-70223
"Verve Therapeutics announced today (July 12) that it has dosed the first participant in a clinical trial assessing the safety and tolerability of the gene editing platform VERVE-101, which uses a precise kind of gene editing called base editing. By changing a single DNA nucleotide, VERVE-101 can inactivate a gene expressed in the liver that is linked to high cholesterol in people with a hereditary and potentially life-threatening subtype of cardiovascular disease called heterozygous familial hypercholesterolemia (HeFH).
“The dosing of the first human with such an investigational base editing medicine represents a significant achievement by our team and for the field of gene editing,” Sekar Kathiresan, the cofounder and chief executive officer of Verve Therapeutics, says in a press release. “Preclinical data suggest that VERVE-101 has the potential to offer people with HeFH a game-changing treatment option, transforming the traditional chronic care model to a single-course, life-long treatment solution.”
Prior to the trial, HeFH and other conditions that manifest in high levels of LDL cholesterol (so-called bad cholesterol) were treated using dietary and lifestyle changes combined with cholesterol-lowering drugs that have to be administered one or two times a month. This regimen can be difficult for patients to adhere to, Gary Balady, the director of preventive cardiology at Boston Medical Center, tells The Boston Globe, and only about 20 percent of patients are able to achieve their goals. Balady, who was not involved in the trial, adds that “having a one-time treatment has the potential to save lots of lives.”
VERVE-101 edits a single nucleotide within the genetic code for PCSK9, a gene expressed in the liver. PCSK9 encodes an enzyme that regulates the amount of cholesterol in the plasma by binding to LDL receptors on liver cell membranes. Certain heritable forms of HeFH are caused by mutations in the genes; by substituting an adenine base for a guanine, the base editing tool induces a new, intentional mutation that renders the defective gene inactive, lowering the level of LDL in a person’s body and, consequently, reducing the risk of developing cardiovascular diseases such as HeFH.
In preclinical studies using nonhuman primates, VERVE-101 lowered levels of LDL cholesterol by roughly 70 percent after two weeks, and the levels remained low for at least two years, Kathiresan tells the Globe.
“This seems like a bigger effect than I would have predicted,” Sarah de Ferranti, the chief of ambulatory cardiology at Boston Children’s Hospital, who was not involved in the work, tells the Globe. She adds that even if the treatment were only half as effective in humans, “I still think that would be a huge game changer.”
The human trial, which received regulatory approval in May, includes roughly 40 adults with HeFH who will receive one of three dosing regimens. The first person to receive a dose lives in New Zealand, where the trial was approved, but Verve Therapeutics also plans to seek permission from regulators this year to expand it into the UK and the US, Biopharma Dive reports. Preliminary trial results are expected in 2023.
ABOVE:
© ISTOCK.COM, VCHAL
Verve Therapeutics announced today (July 12) that it has dosed the first participant in a clinical trial assessing the safety and tolerability of the gene editing platform VERVE-101, which uses a precise kind of gene editing called base editing. By changing a single DNA nucleotide, VERVE-101 can inactivate a gene expressed in the liver that is linked to high cholesterol in people with a hereditary and potentially life-threatening subtype of cardiovascular disease called heterozygous familial hypercholesterolemia (HeFH).
“The dosing of the first human with such an investigational base editing medicine represents a significant achievement by our team and for the field of gene editing,” Sekar Kathiresan, the cofounder and chief executive officer of Verve Therapeutics, says in a press release. “Preclinical data suggest that VERVE-101 has the potential to offer people with HeFH a game-changing treatment option, transforming the traditional chronic care model to a single-course, life-long treatment solution.”
Prior to the trial, HeFH and other conditions that manifest in high levels of LDL cholesterol (so-called bad cholesterol) were treated using dietary and lifestyle changes combined with cholesterol-lowering drugs that have to be administered one or two times a month. This regimen can be difficult for patients to adhere to, Gary Balady, the director of preventive cardiology at Boston Medical Center, tells The Boston Globe, and only about 20 percent of patients are able to achieve their goals. Balady, who was not involved in the trial, adds that “having a one-time treatment has the potential to save lots of lives.”
VERVE-101 edits a single nucleotide within the genetic code for PCSK9, a gene expressed in the liver. PCSK9 encodes an enzyme that regulates the amount of cholesterol in the plasma by binding to LDL receptors on liver cell membranes. Certain heritable forms of HeFH are caused by mutations in the genes; by substituting an adenine base for a guanine, the base editing tool induces a new, intentional mutation that renders the defective gene inactive, lowering the level of LDL in a person’s body and, consequently, reducing the risk of developing cardiovascular diseases such as HeFH.
In preclinical studies using nonhuman primates, VERVE-101 lowered levels of LDL cholesterol by roughly 70 percent after two weeks, and the levels remained low for at least two years, Kathiresan tells the Globe.
“This seems like a bigger effect than I would have predicted,” Sarah de Ferranti, the chief of ambulatory cardiology at Boston Children’s Hospital, who was not involved in the work, tells the Globe. She adds that even if the treatment were only half as effective in humans, “I still think that would be a huge game changer.”
The human trial, which received regulatory approval in May, includes roughly 40 adults with HeFH who will receive one of three dosing regimens. The first person to receive a dose lives in New Zealand, where the trial was approved, but Verve Therapeutics also plans to seek permission from regulators this year to expand it into the UK and the US, Biopharma Dive reports. Preliminary trial results are expected in 2023.
Base editing technology has only recently made its way to human trials, and VERVE-101 is not the first. This year, researchers at University College London launched a clinical trial to treat leukemia using base editing tools, and the Boston-based biotech company Beam Therapeutics, which licensed some of its technology to Verve Therapeutics, will launch a trial later this year using a base editor to treat people with blood disorders such as sickle cell disease and beta thalassemia, the Globe reports. But these trials involve modifying patients’ cells outside of their body before infusing them back in; Verve Therapeutics joins a short list of companies that have developed treatments meant to edit genes directly in the body.
David Liu, the Broad Institute of MIT and Harvard University researcher who first developed base editing in 2016, tells the Globe that he hopes to see many more platforms in the coming year, noting that “to be able to take control of our genomes, to me, is one of the most human things we can do” to alleviate suffering.
But Jennifer Doudna of the University of California, Berkeley, one of the researchers credited with coinventing CRISPR gene editing in 2012, notes that even base editing—generally thought to be safer than other CRISPR techniques because it manipulates a smaller chunk of the genome—is not without risk, and she doesn’t yet think CRISPR has the precision to correct point mutations. There can be off-target effects, she tells the Globe, meaning that you “end up usually getting more editing than you might want.”
See “Base Editors Cause Off-Target Mutations in RNA”
The repercussions of these effects are not yet known, but John Evans, the chief executive of Beam Therapeutics, tells the Globe that these off-target edits are somewhat predictable, and potentially something that can be avoided. “You can kind of engineer around it,” he says.
c
Biotechs have been hot recently. No worries about tax loss selling this year.
LOL
Blue sky breakout!
$BEAM
Weeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeeee
How about them apples!
$BEAM
Beam me up Scotty lol lmao
Beasting out!
$BEAM
Revenue 24k........ 0.00% revenue per... Never seen an Operating Margin of -1,269,908.4% negative 1 million & a quarter percent
Profitability
Profit Margin 0.00%
Operating Margin (ttm) -1,269,908.40%
Management Effectiveness
Return on Assets (ttm) -37.50%
Return on Equity (ttm) -106.96%
Income Statement
Revenue (ttm) 24k
Revenue Per Share (ttm) 0.00
Quarterly Revenue Growth (yoy) 0.00%
Gross Profit (ttm) 24k
EBITDA -299.74M
Net Income Avi to Common (ttm) -365.69M
Diluted EPS (ttm) -6.76
Balance Sheet
Total Cash (mrq) 503.48M
Total Cash Per Share (mrq) 8.23
Total Debt (mrq) 136.59M
Total Debt/Equity (mrq) 32.33
Current Ratio (mrq) 5.04
Book Value Per Share (mrq) 6.91
Cash Flow Statement
Operating Cash Flow (ttm) -106.93M
Levered Free Cash Flow (ttm) -126.99M
Kaboomage
$BEAM
Reloaded $69.00-$71.00 will ride it to new highs.
$BEAM
Starting buying at $15.00 Will add if it dips into the low 80.00s again.
I have no problem adding the dips and buying quality stocks for the long term.
$BEAM
What a beast!
$BEAM
Short Shrift for Short-Sighted Short Sellers
Desmond Wheatley CEO Beam Global
Dear friends, shareholders, employees and supporters,
The United States has the most efficient capital markets in the world. It’s part of what makes this nation great. During the last hundred years or so, this efficient use of capital has enabled the greatest period of innovation in human history. The stock market provides companies access to the capital they need to succeed and investors the opportunity to share in that success. An efficiently functioning stock market is critical to a healthy economy.
There are many ways in which investors can participate in the stock market. One, which is receiving a great deal of attention at the moment, is short selling. As a public company CEO and an issuer, I have mixed feelings about short selling. I recognize that it can be a useful contributor to a healthy and efficient stock market because it can help in identifying and correcting overvalued securities. An honest and informed short seller that analyzes a company’s business plan, operations, finances, leadership and prospects, and finds that company to be overvalued, can profit by coming to that conclusion before the rest of the market inevitably identifies the same weaknesses. I’m in favor of that. One should not pity the target of that kind of short selling, particularly if the overvaluation is driven by unrealistic or even dishonest projections on the part of the issuer.
But what if the issuer is not dishonest and the projections are reasonable? What if certain short sellers create a narrative just to support their short position? They know that if they can be convincing enough, in an already opaque and complicated world, that the seeds of doubt they sow can lead to selling and price depreciation. They know that they can profit from the losses of the shareholders who believe, correctly, in the issuer. That’s not efficient, that’s dishonest. That kind of behavior does not support innovation and entrepreneurialism. It destroys the opportunities created by the inventiveness and hard work of the entrepreneur, and it destroys the value of the position of the honest investor. It undermines the essential ingredient in the investor’s arsenal – reliable information.
I get a lot of advice in my capacity as CEO of Beam Global. One of the most common is that I should not take negative comments about the company personally. Well, I do take them personally. Like everything else to do with the company, I take them very personally indeed. I love this company and I am very proud of the team, the products, our customers and our shareholders. It’s insulting to our hard-working team, our discerning (often repeat) customers and our loyal supporters and shareholders to suggest that we are anything other than what we say we are.
Almost all public comments about Beam are resoundingly positive. We have regular corporate and government customer endorsements in our press releases (I’m told that that is unusual by our Public Relations company). I personally spend a great deal of time on the phone, in video meetings or face to face with customers, shareholders, vendors and other interested parties. I am always willing to listen to constructive criticism during those interactions and the overwhelming majority of feedback I get is positive – very positive. We have never had a product returned or any customer that regrets driving on sunshine.
But I know that from time to time, particularly when our share price has increased in value, there will be those who will attempt to profit from sowing the seeds of doubt about our products, our plan, our team and our leadership.
Most businesses are fairly complex. Beam is one of the simpler and easier to understand but even so, it’s not difficult to selectively identify historical data points, magnify insignificancies and cast half-truths, opinions and falsehoods as supporting facts to a negative narrative. That has happened and it will happen again. Where there’s profit there’s motive and where there’s motive there’s often action.
Fortunately for Beam, there is a lot of good and honest research available for the discerning investor. Professional, hard-working Wall Street analysts who have spent months researching our company and the industries in which we operate, have released detailed and well researched reports. Reports for which Beam paid nothing. Reports over which Beam had no influence or input other than to answer the questions of the analysts and to open our factory, products and business practices to their scrutiny. Their conclusions are unanimously positive.
Those who have come to the opposite conclusion have never contacted us, never seen our operations, never tested our products and never shown the slightest inclination to do so. They have created the negative narrative but without the hard work required to know the truth. I will leave it up to the investment community to conclude what their motivations might be.
Beam Global is in the best position in our history. The (global) political, consumer, market and environmental conditions are firmly in our favor. Our ability to execute, be financially responsible and grow have been proven in fact over the last many years. If you read or hear something about Beam which makes you doubt us, take a look at the published respected analysts’ reports about us or all the very positive comments made by our customers. If you don’t get the answers you are looking for there, then contact me. My direct line and direct email are at the bottom of this letter. Remember, I have a company to run and I owe it to our customers, employees and shareholders not to spend all my time responding to nefarious commentary, but I’ll say it again, contact me. Ask me your questions, no matter how tough you think them. If disclosure rules allow, I’ll answer them in the only way I know how – honestly and with pride in what we are doing.
We have fantastic opportunities ahead of us. I could not be more enthusiastic about what I see coming in 2021 and beyond for Beam. I believe that concentrating on the nexus of clean energy and transportation will continue to place us in one of the most exciting transitions in history. We will vigorously pursue profits doing something we can all be very proud of. Thank you for being involved and let us all ensure that facts and truth guide us, and not allow those who know nothing, and care even less, about our fundamentals to slow us down, even for a second.
Desmond Wheatley
President, CEO and Chairman of the Board
Added a 1000 shares today.
$BEAM
Beam investors i was in early here in the teens. Next ARKG gene editing stock that is going to run hard is EVGN
$EVGN
$BEAM
BEAM Sold half like i said at $100.00 riding the rest for free now. Parlayed more into HGEN.
BEAM
https://newsrts.com/2020/12/29/why-beam-therapeutics-inc-beam-stock-could-see-much-higher-prices-ahead/
HGEN
https://www.globenewswire.com/news-release/2019/07/02/1877284/0/en/Humanigen-Secures-Exclusive-Worldwide-License-to-Gene-Editing-Technology-from-Mayo-Clinic-to-Improve-CAR-T.html
BEAM 5.5 billion market cap
HGEN 800 million market cap phase 3 CV-19 deep pipeline partnered with Gilead & KITE & MAYO & NIH BARDA
$BEAM
will start selling part of my holdings at $100.00 love this company!
500%+ since summer!
$BEAM
my $15.00's looking good!
always early.
https://gfycat.com/infantilemammothboar-goodwillhunty-caseyaffleck-drama
$BEAM
News: $BEAM Here's Why Shares of Editas Medicine and Beam Therapeutics Are Soaring Today
Shares of Editas Medicine (NASDAQ: EDIT) and Beam Therapeutics (NASDAQ: BEAM) rose as much as 23% and 29%, respectively, today after the pair were rumored to be considering a merger. Although investors shouldn't invest based on speculation, a merger would make sense on multiple fronts. ...
Got this from BEAM - Here's Why Shares of Editas Medicine and Beam Therapeutics Are Soaring Today
This is making news all over. Just heard it on the FM as well.
RIP ticker $BEAM me up Scotty
Wow, there goes another nice vehicle.
wish I got those 700 shares @ 70.13 when news hit
BEAM
Very nice.. Watch close I guess.. Some stocks breaking higher and many look to turn lower. Strange times.
October 2011 ;)
Go Jimmy, Go!!!