iHub News
3月前
Beam Therapeutics Shares Climb After Positive BEAM-302 Trial UpdateMarch 25, 2026 10:04 AM
IH Market News
Shares of Beam Therapeutics Inc. (NASDAQ:BEAM) gained about 8% on Wednesday after the company released updated results from its ongoing Phase 1/2 clinical study of BEAM-302, a treatment being developed for alpha-1 antitrypsin deficiency (AATD).According to the company, patients receiving a 60 mg dose of BEAM-302 achieved a mean steady-state total AAT level of 16.1 µM. All treated patients maintained levels consistently above the protective threshold of 11 µM, with follow-up data extending up to 12 months. Corrected M-AAT represented 94% of total AAT, while levels of the mutant Z-AAT protein were reduced by 84% following treatment with the 60 mg dose.BEAM-302 is a liver-targeted lipid nanoparticle therapy designed to correct the genetic mutation responsible for the severe form of AATD using base editing technology. The treatment is currently being studied in a Phase 1/2 open-label trial assessing its safety, tolerability, pharmacokinetics, pharmacodynamics, and overall effectiveness.As of the February 10 data cutoff, a total of 29 patients had received BEAM-302, with follow-up periods reaching up to 18 months. Among 26 patients treated with a single dose, the therapy demonstrated a favorable safety profile at doses up to 75 mg. Reported adverse events were generally mild to moderate, with no serious adverse events or dose-limiting toxicities recorded by the cutoff date.Based on the observed safety and efficacy across the single-dose cohorts, the company selected 60 mg as the optimal biological dose. Beam said it plans to launch a global pivotal cohort in the second half of 2026 to support a potential accelerated regulatory approval.Researchers also observed a dynamic increase in AAT expression in one patient in the 60 mg cohort during a respiratory infection. In that case, total AAT levels rose from a steady-state level of 15.9 µM to 29.5 µM, while the AAT composition remained stable at 95% M-AAT.In Part B of the trial, which includes patients with AATD-related liver disease, single doses of 30 mg and 60 mg of BEAM-302 produced efficacy results consistent with those seen in Part A participants who did not have liver disease.Beam Therapeutics stock price
Original: Beam Therapeutics Shares Climb After Positive BEAM-302 Trial Update
abew4me
3年前
BEAM Focuses on Developing Gene Drug for Growth
9:06 am ET October 10, 2023
Beam Therapeutics’ BEAM proprietary base-editing technology potentially enables the development of a differentiated class of precision genetic medicines that target a single base in the genome without making a double-stranded break in the DNA, thereby minimizing errors.
The company is pursuing two primary approaches depending on tissue type in vivo programs that use intravenously administered CRISPR as the therapy and ex vivo programs that use CRISPR to create the therapy using engineered human cells.
BEAM is evaluating its leading ex-vivo genome-editing candidate, BEAM-101, in the phase I/II BEACON study for sickle cell disease (SCD). It has successfully consented enough patients projected to both fill the sentinel cohort and initiate the expansion cohort of the early-stage study.
Apart from BEAM-101, the company is evaluating BEAM-201, a therapy designed to create allogeneic CD7, targeting CAR-T cells resistant to fratricide and immunosuppression. BEAM-201 is actively enrolling patients at multiple clinical sites, in a phase I/II study for treating patients with T-cell leukemia and T-cell lymphoblastic lymphoma, a severe disease affecting children and adults, and potentially other CD7+ malignancies.
The company also has two pre-clinical candidates —BEAM-301 and BEAM-302 — in its portfolio for genetic diseases.
BEAM has initiated IND-enabling studies for both candidates and plans to submit a regulatory application to begin clinical studies in the first half of 2024.
While the company is yet to have an approved marketable product in its pipeline, its collaboration deals help access capabilities and resources to support the ongoing therapeutic programs.
BEAM collaborated with Pfizer PFE in 2021, focusing on using its gene-editing technology to create treatments for rare genetic diseases that affect the liver, muscles and the central nervous system. It is conducting the research work for three specific gene-editing programs. If these programs prove effective in clinical studies, Pfizer can choose to help develop and sell them globally, with both companies sharing the costs and profits.
north40000
4年前
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.
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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
conix
5年前
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
IPO$
14年前
Pucker™ Vodka Expands Boldly-Flavored Vodka Portfolio with the Launch of Two New Expressions
Date : 02/21/2012 @ 8:00AM
Source : PR Newswire
Stock : Beam Inc. Common Stock (BEAM)
Quote : 55.11 -0.91 (-1.62%) @ 2:44PM
Pucker™ Vodka Expands Boldly-Flavored Vodka Portfolio with the Launch of Two New Expressions
PrintAlert
Beam Inc. Common Stock (NYSE:BEAM)
Intraday Stock Chart
Today : Tuesday 21 February 2012
Pucker™ Vodka Expands Boldly-Flavored Vodka Portfolio with the Launch of Two New Expressions Pucker™ Lemonade Lust Vodka and Pucker™ Raspberry Rave Vodka Join the Fast-Growing Flavored Vodka Line
PR Newswire
DEERFIELD, Ill., Feb. 21, 2012
DEERFIELD, Ill., Feb. 21, 2012 /PRNewswire/ -- Pucker™ Vodka continues to surprise flavored vodka fans with its distinct and unexpected flavors. Now, one year after bursting on the scene, Pucker Vodka is introducing two bold, intense flavors – Pucker™ Lemonade Lust Vodka and Pucker™ Raspberry Rave Vodka – to the family.
All six Pucker Vodka flavors are perfect for those looking to break up with their boring vodka and to match up with an exciting and new intensely flavored vodka.
"Last year, we launched Pucker Vodka to help flavored vodka drinkers express their 'live out loud' personalities, and they certainly responded well to our original flavors," said Kim Washington, Senior Director, Vodkas for Beam Inc. "This year, we chose to expand our line to include two of the most popular flavors – lemonade and raspberry – to help liven up old, boring, vodka cocktails."
Pucker Lemonade Lust Vodka provides bright bursts of crisp lemon flavor and a hint of lime, while Pucker Raspberry Rave Vodka offers an intense raspberry flavor and a touch of floral notes. Both new products are four-times distilled, 70-proof, and packed with intense flavor. Each of the new flavors provides a taste that is balanced and confident enough to enjoy with a simple mixer, served straight-up or in a playful cocktail creation.
To demonstrate its stand-out and unique new flavors, Pucker Vodka recommends these cocktails:
Lusty Lemon 1 1/2 parts Pucker™ Lemonade Lust Vodka 2 parts Fresh Lemon Sour 4 parts Iced Tea Lemon Wedge Combine ingredients and serve over ice in a tall highball glass. Garnish with a lemon wedge.
Raspberry Breeze 1 1/2 parts Pucker™ Raspberry Rave Vodka 2 parts Grapefruit Juice 2 parts Cranberry Juice Lemon Wedge Combine ingredients in order and serve over ice in a tall highball glass. Garnish with a lemon wedge.
Lemonade Lust and Raspberry Rave are available nationwide in February at a suggested retail price of $15.99 for a 750ml bottle. For more information, please go to www.puckervodka.com or www.facebook.com/puckervodka.
About Pucker™ Vodka
Pucker™ Vodka is made from natural flavors, combined with a vodka that's distilled four times to deliver full flavor that can be enjoyed straight or mixed in any cocktail. Pucker Vodka creates a unique sensory vodka experience unlike any other flavored vodka on the market today. Flavors include Sour Apple Sass, Grape Gone Wild, Cherry Tease, Citrus Squeeze, Lemonade Lust and Raspberry Rave.
About Beam Inc.
As one of the world's leading premium spirits companies, Beam is Crafting the Spirits that Stir the World. Consumers from all corners of the globe call for the company's brands, including Jim Beam Bourbon, Maker's Mark Bourbon, Sauza Tequila, Canadian Club Whisky, Courvoisier Cognac, Teacher's Scotch Whisky, Kilbeggan Irish Whiskey, Laphroaig Scotch Whisky, Cruzan Rum, Hornitos Tequila, Knob Creek Bourbon, EFFEN Vodka, Pucker Flavored Vodka, Larios Gin, Whisky DYC, DeKuyper Cordials, and Skinnygirl Cocktails. The Beam portfolio includes 10 of the world's top 100 premium spirits brands and some of the industry's fastest growing innovations. Beam is focused on delivering superior performance with its unique combination of scale with agility and a strategy of Creating Famous Brands, Building Winning Markets and Fueling Our Growth. Beam and its 3,200 passionate associates worldwide generated 2011 sales of $2.8 billion on volume of 34 million 9-liter cases.
Headquartered in Deerfield, Illinois, Beam is traded on the New York Stock Exchange under the ticker symbol BEAM and is included in the S&P 500 Index and the MSCI World Index. For more information on Beam, its brands, and its commitment to social responsibility, please visit www.beamglobal.com and www.drinksmart.com.
Pucker™ Flavored Vodka, 35% Alc./Vol. 100% Grain Neutral Spirits ©2012 Fielding & Jones Ltd., Clermont, KY; Frankfort, KY; Cincinnati, OH Facebook is a registered trademark of Facebook, Inc.
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