Latest news with #Beam
Yahoo
2 days ago
- Business
- Yahoo
Beam Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD)
CAMBRIDGE, Mass., May 29, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted orphan drug designation to BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation of a guide RNA and an mRNA encoding a base editor designed to correct the disease-causing mutation in patients with alpha-1 antitrypsin deficiency (AATD). AATD is an inherited genetic disorder that affects the lungs and/or liver, leading to early onset emphysema and liver disease, and for which there is significant unmet need for effective therapies that can treat the entire spectrum of disease. 'Receiving orphan drug designation for BEAM-302 is an important milestone in our efforts to bring a transformative therapy to people living with AATD, many of whom currently lack effective long-term treatment options,' said Giuseppe Ciaramella, Ph.D., president of Beam Therapeutics. 'This recognition by the FDA, following the receipt of RMAT designation from the FDA just weeks ago, highlights the urgency of addressing this serious genetic disease and the potential of BEAM-302 to directly correct the DNA mutation, the underlying root cause of this illness. We are encouraged by the FDA's continued support of this program and are committed to its advancement with the goal of delivering a one-time, potentially curative treatment to patients as quickly and safely as possible.' The FDA's orphan drug designation is designed to support the development and evaluation of treatments for rare diseases affecting fewer than 200,000 people in the U.S. The designation comes with potential benefits for the sponsor company, including tax credits for qualified clinical trials, exemption from user fees, and a potential seven years of market exclusivity after approval. Positive initial safety and efficacy data from the ongoing Phase 1/2 trial of BEAM-302, previously reported in March, established clinical proof of concept as a potential treatment for AATD and in vivo base editing. Preliminary results from the first three single-ascending dose cohorts in Part A of the study demonstrated that BEAM-302 was well tolerated, with single doses of BEAM-302 leading to durable, dose-dependent correction of the disease-causing mutation and total AAT protein levels above the therapeutic threshold in the 60 mg dose cohort. Beam has initiated dosing in the fourth cohort of Part A, evaluating 75 mg of BEAM-302, and expects to report updated data at a medical conference in the second half of 2025. Additionally, the company plans to dose the first patient in Part B, which will include AATD patients with mild to moderate liver disease, in the second half of 2025. Beam previously announced the clearance of the U.S. investigational new drug (IND) application for BEAM-302 for the treatment of AATD in March 2025, as well as the granting of Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA to BEAM-302 in May 2025. About BEAM-302BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to correct the PiZ mutation. Patients homozygous for this mutation (PiZZ) represent the majority of patients living with severe AATD disease. A one-time A-to-G correction of the PiZ mutation with Beam's adenine base editor has the potential to simultaneously reduce the aggregation of mutant, misfolded AAT protein that causes toxicity to the liver (Z-AAT), generate therapeutic levels of corrected protein (M-AAT), and increase total and functional AAT in circulation, thereby addressing the underlying pathophysiology of both the liver and lung disease. In addition, the reduction in circulating PiZ aggregates (i.e., polymers) has the potential to further minimize lung inflammation and dysfunction. Importantly, because the native AAT gene would be corrected in its normal genetic location, AAT levels are anticipated to increase physiologically in response to inflammation or infection. This is a critical aspect of AAT's normal function to regulate the body's inflammatory response, which does not occur with currently approved protein replacement therapies. Correction of the PiZ mutation is expected to be durable based on preclinical and clinical evidence. About Alpha-1 Antitrypsin Deficiency (AATD)AATD is an inherited genetic disorder that can cause early onset emphysema and liver disease. The most severe form of AATD arises when a patient has a point mutation in both copies of the SERPINA1 gene at amino acid 342 position (E342K, also known as the PiZ mutation or the 'Z' allele). This point mutation causes alpha-1 antitrypsin, or AAT, to misfold, accumulating inside liver cells rather than being secreted, resulting in very low levels (10%-15%) of circulating AAT. In addition to resulting in lower levels, the PiZ AAT protein variant is also less enzymatically effective compared to wildtype AAT protein. As a consequence, the lung is left unprotected from neutrophil elastase, resulting in progressive, destructive changes in the lung, such as emphysema, which can result in the need for lung transplants. The mutant AAT protein also accumulates in the liver, causing liver inflammation and cirrhosis, which can ultimately cause liver failure or cancer requiring patients to undergo a liver transplant. It is estimated that approximately 100,000 individuals in the U.S. have two copies of the Z allele, known as the PiZZ genotype, although only about 10% of all patients are thought to have been diagnosed. There are currently no curative treatments approved for patients with AATD, and the only approved therapy in the U.S., intravenous AAT protein replacement, has not been shown to prevent ongoing lung function decline and destruction in patients. About Beam TherapeuticsBeam Therapeutics (Nasdaq: BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform with integrated gene editing, delivery and internal manufacturing capabilities. Beam's suite of gene editing technologies is anchored by base editing, a proprietary technology that is designed to enable precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This has the potential to enable a wide range of therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases. Cautionary Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on these forward-looking statements, including, but not limited to, statements related to: the therapeutic applications and potential of our technology, including with respect to AATD; our plans, and anticipated timing, to advance our BEAM-302 program, including the clinical trial designs and expectations for BEAM-302; our plans to present data at upcoming medical conferences; expectations regarding potential benefits of the orphan drug designation for BEAM-302; and our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the uncertainty that our product candidates will receive regulatory approval necessary to advance human clinical trials; that preclinical testing of our product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that initiation and enrollment of, and anticipated timing to advance, our clinical trials may take longer than expected; that our product candidates or the delivery modalities we rely on to administer them may cause serious adverse events; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; our ability to recognize the potential benefits conferred by the orphan drug designation for BEAM-302; and the other risks and uncertainties identified under the headings 'Risk Factors Summary' and 'Risk Factors' in our Annual Report on Form 10-K for the year ended December 31, 2024, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law. Contacts: Investors:Holly ManningBeam Therapeuticshmanning@ Media:Josie Butler1ABjosie@ in to access your portfolio
Scottish Sun
2 days ago
- Entertainment
- Scottish Sun
Amad Diallo and Alejandro Garnacho show middle finger to fans during Man Utd's post-season tour in Malaysia
Click to share on X/Twitter (Opens in new window) Click to share on Facebook (Opens in new window) AMAD DIALLO and Alejandro Garnacho raised eyebrows after they were seen showing the middle finger towards Manchester United fans. The gestures came during the Malaysia leg of the club's so-far disastrous post-season tour of Asia. Sign up for Scottish Sun newsletter Sign up 3 Amad Diallo was seen gesturing a middle finger towards Man Utd fans while on the club's post-season tour Credit: Reddit 3 Amad was later seen doing it again with Alejandro Garnacho, suggesting it is an inside joke Credit: Reddit After producing the club's worst-ever Premier League campaign and losing the Europa League final, Ruben Amorim's side suffered the ignominy of losing a friendly 1-0 to ASEAN All-Stars, a team made up of players from Southeast Asian leagues such as Malaysia and Thailand. Before the game, Man Utd flops were seen touring the streets of Kuala Lumpur on an open-top bus parade. And despite their latest flop on the pitch, fans were still desperate to catch a glimpse of the Red Devils before they jet off to Hong Kong for the next leg of their tour. One fan shared a video of some of the players walking through a crowd. READ MORE IN FOOTBALL RED WITH ANGER Fuming Amorim says Man Utd 'choke in EVERY game' after ASEAN All-Stars loss In the clip, 22-year-old Amad can be seen heading past supporters should his name. He then extends his right arm with his middle finger raised in a gesture seemingly directed in the direction of fans. Meanwhile, in a separate snap, a group of Man Utd players, including Amad and Garnacho, are posing for a selfie with a pair of fans. The two wingers can be seen with sombre expressions on their faces while also lifting their middle fingers up towards one another while standing in the group. Join SUN CLUB for the Man Utd Files every Thursday plus in-depth coverage and exclusives from Old Trafford It is not clear what the meaning behind the pose is, though the consistent use of it suggests it might be an inside joke in the Man Utd camp. SunSport have gone to Man Utd for comment. Glum Man Utd stars take part in humiliating 'open-top bus parade' with no trophy and hardly any fans Reacting to the gestures on social media, one fan said: "Everyone knows what the middle finger means and those fans still looked pretty happy. Maybe they got asked to do it?" A second said: "Honestly could just be them trynna pose for a photo like guys do, one of those guy poses to give the middle finger, but it would be very weird as a professional to do that right after a loss, it looks bad." A third added: "Is this a trend or something?" Another said: "Glad to see United's utterly unnecessary micro tour is going well." A separate fan interaction had seen Amad, Garnacho and Ayden Heaven asking locals to help them pay for some Beam e-scooters to take in the city. While Garnacho, 20 - who has been told to find a new club by Amorim - cast a glum look at a shirt signing event. MAN UTD TRANSFER NEWS LIVE: All the latest deals and rumours from Old Trafford After losing to ASEAN All-Stars in front of 70,000 people, Amorim once again slammed his team, saying: "Yes, a disappointing result, but no injuries and a tough pitch. "We don't have it in us not to choke in every exercise, in every game, that is what happened. "We should win these kind of games, no matter what. We need to bring that inside of us in every game. "The important thing is no injuries with this weather and to arrive here in this day. It is not an excuse, we should perform better." Amorim went on to praise the club's youngsters, saying: "I liked the young kids, they try. That's enough for me in the moment. "They want to play more but we have to rotate everybody to play. They did well." Man Utd will now face the Hong Kong national team tomorrow at 1pm UK time.
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New Straits Times
2 days ago
- Entertainment
- New Straits Times
#NSTviral: Scooters and selfies: Man Utd stars embrace KL like big kids on tour [WATCH]
KUALA LUMPUR: In the quiet early hours of the city, Manchester United's Amad Diallo, Alejandro Garnacho, and rising academy star Ayden Heaven were wide awake and ready for some fun. They were spotted wandering the streets near their hotel in full United gear, attempting to rent Beam e-scooters with the help from amused locals. Though the scooters didn't quite cooperate, they laughed, posed for selfies with surprised fans, and soaked up the calm charm of the city in the wee hours, unbothered by the absence of security or paparazzi. In a video shared by SimplyUtd on X (formerly Twitter), several Manchester United players in their 20s were seen approaching a group of locals, asking for help to rent electric scooters. While a few fans were willing to assist, the scooters appeared to malfunction, with one of the men heard saying, "It's not working" and "Very unlucky." The clip was captioned: "When Amad casually asked my brother to pay for an electric scooter for him. Same with Garnacho and Heaven." @lICHA_1806 said: "I think out of everyone in the squad, Amad is the one who could ask any United fan and they'd do it." @cindymills75 said: "It looks like they're using their phones. They're just getting help with the transaction, and if not, it's no big deal. Kind people." Less delightful, however, was what followed. United fell 0-1 to the Asean All-Stars at the National Stadium in Bukit Jalil, with Myanmar's Maung Maung Lwin netting the winner in the 71st minute last night. But for that short slice of the night before, Amad, Garnacho and Ayden reminded everyone that behind the bright lights of football stardom, they're still just big kids — curious, hungry, and loving every bit of a night out in KL.
Rakyat Post
3 days ago
- Entertainment
- Rakyat Post
Manchester United Stars Spotted Exploring Kuala Lumpur, Fans Thrilled by Surprise Encounter
Subscribe to our FREE Three Manchester United players caused a stir in the heart of Malaysia's capital this week, when they were spotted enjoying a casual evening out in Kuala Lumpur. Amad Diallo, Alejandro Garnacho, and rising youth talent Ayden Heaven were seen strolling around the city, attracting excited locals who quickly recognized the footballers. Videos of the trio quickly went viral on social media, with fans enthusiastically approaching them for selfies and autographs. The players appeared relaxed and friendly, engaging with the crowd and soaking in the lively city atmosphere. In one moment that gained particular attention online, the United players asked a group of locals if they could help them rent Beam e-scooters. Several fans happily obliged, although there seemed to be an issue with the electric scooters as a man could be heard saying 'It's not working' and 'very unlucky'. 😂 Amad Diallo asking a fan to pay for his scooter hire in Malaysia 🇲🇾 — SimplyUtd (@SimplyUtd) Folks on social media reacted to the video documenting the players' casual night stroll, noting how they were in their full team uniform and without security or papparazzi surrounding them. 'They were free to walk around like that. That means Malaysia is a safe country,' one user posted in the comments. Meanwhile, some users expressed that they feel left out from not getting the opportunity to meet the players, and others jokingly asked which mamak they're hanging out at. Their surprise appearance followed a recent visit to Malaysia for the ASEAN All-Stars match, held at Bukit Jalil National Stadium. However, the night on the town came just before a far less cheerful moment for the players – a humiliating defeat in the much-anticipated exhibition match the next day. Share your thoughts with us via TRP's . Get more stories like this to your inbox by signing up for our newsletter.
Scottish Sun
3 days ago
- Entertainment
- Scottish Sun
Man Utd star Amad Diallo ‘asks locals to pay for electric scooter' as fans joke ‘these cuts are getting ridiculous'
The 22-year-old put out a defiant message after United's horror campaign A-MAD DASH Man Utd star Amad Diallo 'asks locals to pay for electric scooter' as fans joke 'these cuts are getting ridiculous' AMAD DIALLO was having trouble with the local transport in Malaysia - just hours before another embarrassing defeat for Manchester United. The Ivorian forward is part of the Red Devils squad currently on a post-season tour of Asia. 4 Adam Diallo was filmed appearing to ask locals for help paying for his e-scooter Credit: X 4 Alejandro Garnacho and Ayden Heaven were also taking in the sights of Kuala Lumpur Credit: X 4 The Ivorian has enjoyed a strong season with the Red Devils Credit: EPA Amad came off the bench during Wednesday's dismal 1-0 loss to the ASEAN All Stars in Kuala Lumpur. Hours before, the 22-year-old joined teammates Alejandro Garnacho and Ayden Heaven for a spot of sightseeing. The trio - decked out in United training gear - hopped on some Beam e-scooters to take in the city. But they quickly ran into trouble when attempting to pay for their trip. READ MORE ON MAN UTD RIO EXIT Rio Ferdinand quitting TNT Sports after 10 years as Man Utd icon issues statement Viral footage originally shared on TikTok showed a local having to help Amad and his fellow stars put a payment through. The striker appeared to give his phone over to one fan, while others took selfies with Garnacho and Heaven. One fan laughed in response: "Ratcliffe didn't pay the players wages." A second joked: "These cuts are getting ridiculous." BEST ONLINE CASINOS - TOP SITES IN THE UK An eagle-eyed third commented: "My guy left with the hotel slippers." And a fourth clarified: "For those saying he was asking people to pay for his scooter, it's their phones they just needed help with the app." Ruben Amorim vows 'good days are coming' after 'disaster season' as Man Utd flops give him daggers during public apology Despite United suffering a dismal campaign, Diallo proved to be a shining light at Old Trafford. He suffered an ankle injury which kept him out for over tw months. But across 26 Premier League games, the Ivorian racked up 14 goal contributions. Taking to Instagram after Sunday's final Prem game, he wrote: "Thanks to all United fans for the support no matter what happened. "See you next season, Reds." 4 United's Asia tour is their first post-season trip in the club's modern era. After an embarrassing defeat to the All Stars on Wednesday, Ruben Amorim's men will head next to Hong Kong.
