Latest news with #AATD
Time Business News
27-06-2025
- Business
- Time Business News
Pioneering Technologies in Regenerative Medicine
Regenerative medicine is a branch of medical science that aims to repair, replace, and regenerate damaged tissues or organs to bring them back to normal function. Stem cell research and regenerative technologies are creating new opportunities for treatment and accelerating growth in the regenerative medicine market. The growing prevalence of chronic disease, increasing funding, and technology advances in gene therapy, an increasing aging population, and more favorable regulations are major contributors to the growing share and speed of development of the regenerative medicine. Key Growth Drivers and Opportunities Rising Number of Aging Population: With the global aging phenomenon, there will be increased incidences of chronic and degenerative diseases such as osteoarthritis, cardiovascular ailments, neurodegenerations, and musculo-skeletal problems, very much in demand for advanced treatment options. Regenerative medicine comprising stem cell therapy, gene therapy, and tissue engineering, among others, presents viable and sustainable alternatives in contrast to conventional treatments such as medication and surgery, which provide temporary relief. Given that regenerative medicine addresses the actual cause of tissue and organ damage, it significantly enhances the quality of life and decreases long-term care burdens, serving as the prime growth driver for the regenerative medicine. Challenges The regenerative medicine is limited by high treatment costs, difficult regulatory approvals, not enough long-term clinical data, and problems in big manufacturing and making everything the same which can block easy acceptance and availability. Innovation and Expansion Beam Therapeutics' BEAM-302 has granted RMAT status by the FDA for Alpha-1 Antitrypsin Deficiency In May 2025, The U.S. Food and Drug Administration (FDA) has designated BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation of a guide RNA and an mRNA encoding a base editor, as Regenerative Medicine Advanced Therapy (RMAT). This designation is intended to correct the disease-causing mutation in patients with alpha-1 antitrypsin deficiency (AATD). Beam Therapeutics Inc. is a biotechnology company that develops precision genetic medicines through base editing. There is a substantial unmet need for efficient treatments that may address the whole range of symptoms for AATD, an inherited genetic illness that affects the lungs and/or liver and causes early onset emphysema and liver disease. The FDA Gives BrainChild Bio's CAR T Therapy for Pediatric Brain Tumors RMAT Status In May 2025, The U.S. Food and Drug Administration (FDA) has designated the investigational B7-H3 targeting autologous CAR T-cell therapy as a Regenerative Medicine Advanced Therapy (RMAT) for the treatment of diffuse intrinsic pontine glioma (DIPG), an incurable pediatric brain tumor. This news was released by BrainChild Bio, Inc., a clinical-stage biotechnology company that is developing CAR T-cell therapies to treat tumors in the central nervous system (CNS). The FDA Designates MeiraGTx's AAV-GAD Gene Therapy as an RMAT for Parkinson's disease In May 2025, AAV-GAD has been designated by the U.S. Food and Drug Administration (FDA) as Regenerative Medicine Advanced Therapy (RMAT) for the treatment of Parkinson's disease that is not sufficiently controlled with anti-Parkinsonian drugs. This announcement was made today by MeiraGTx Holdings plc, a vertically integrated, clinical-stage genetic medicines company. This RMAT was granted after the FDA received favorable results from three clinical trials showing the advantages of AAV-GAD when given as a single stereotactic infusion to the brain's subthalamic nucleus. Inventive Sparks, Expanding Markets Key players in the regenerative medicine market are R&D Systems, Inc., Moderna, Novartis AG and others. These key players are focusing on increasing the research and development of advanced therapies and innovative strategies for 3D bioprinting advancement, and more acquisitions to enhance product offerings through collaborations will be projected to drive the target market. About Author: Prophecy is a specialized market research, analytics, marketing and business strategy, and solutions company that offer strategic and tactical support to clients for making well-informed business decisions and to identify and achieve high value opportunities in the target business area. Also, we help our client to address business challenges and provide best possible solutions to overcome them and transform their business. TIME BUSINESS NEWS
Yahoo
10-06-2025
- Health
- Yahoo
Alpha-1 Antitrypsin Deficiency Market Research 2025-2035: Rising Demand for AATD Treatments Fuels Expansion with North America Leading
The global alpha-1 antitrypsin deficiency (AATD) market is expanding, driven by advances in diagnosis, treatment, and growing awareness. Key players like Grifols, CSL Behring, and Pfizer lead innovation. Market growth is fueled by rising disease prevalence and improved therapies but faces high costs. Dublin, June 10, 2025 (GLOBE NEWSWIRE) -- The "Alpha-1 Antitrypsin Deficiency Market - A Global and Regional Analysis: Focus on Treatment, Distribution Channel, and Regional Analysis - Analysis and Forecast, 2025-2035" report has been added to global alpha-1 antitrypsin deficiency (AATD) market is experiencing growth, fueled by advances in diagnosis, treatment options, and increasing awareness of the condition. AATD is a genetic disorder where the body produces insufficient alpha-1 antitrypsin, a protein that protects the lungs and liver. Its deficiency can lead to chronic obstructive pulmonary disease (COPD), emphysema, and liver cirrhosis. The condition often remains underdiagnosed, which presents an opportunity for early detection and primary treatment for AATD is augmentation therapy, which involves the infusion of AAT protein to restore deficient levels in the bloodstream. This therapy is designed to protect the lungs and slow the progression of emphysema. Other treatment options include symptomatic management, such as bronchodilators and corticosteroids. Research continues to explore gene therapies and novel treatments to better address the underlying genetic causes of market is expanding due to rising awareness about AATD, improvements in diagnostic tools, and innovations in treatment options. Key drivers include the increasing prevalence of the disease, particularly in older populations, and the growing availability of specialized therapies. However, challenges such as high treatment costs and the need for long-term care remain significant factors impacting the global alpha-1 antitrypsin deficiency market growth. The North America alpha-1 antitrypsin deficiency market is expected to lead globally due to advanced healthcare infrastructure, high awareness, and significant research investments. The market is primarily driven by augmentation therapy, which involves the administration of alpha-1 proteinase inhibitors to manage disease progression. Enhanced genetic testing and early diagnosis have improved treatment outcomes. Hospitals and specialty clinics serve as key treatment settings, offering specialized care. The region's favorable reimbursement policies and commitment to research and development further support market growth, positioning North America as a dominant force in the global alpha-1 antitrypsin deficiency treatment summary, the global alpha-1 antitrypsin deficiency market is poised for growth, with a strong focus on improving diagnostic accuracy and providing effective treatments, offering significant opportunities for stakeholders in the healthcare and pharmaceutical industries. How Can This Report Add Value to an Organization?Product/Innovation Strategy: Product launches and innovations in the global alpha-1 antitrypsin deficiency market are focused on advancing treatment options to improve patient care. These innovations aim to enhance the efficacy of therapies and streamline the detection and management of the disease. Key players in the global alpha-1 antitrypsin deficiency market, such as Grifols, CSL Behring, Takeda, GlaxoSmithKline plc, and Pfizer Inc., have been involved in offering of therapies for alpha-1 antitrypsin Strategy: Enterprises led by market leaders in the global alpha-1 antitrypsin deficiency market are continuously working on updating their product portfolios with innovative treatments to maintain competitiveness. A detailed competitive benchmarking of the key players in the global alpha-1 antitrypsin deficiency market has been conducted, providing insights into how these companies compare in terms of product offerings, market share, and innovation. This benchmarking provides readers with a clear understanding of the market landscape and the positions of the leading players. Additionally, comprehensive competitive strategies, such as partnerships, agreements, and collaborations, will help readers identify untapped revenue opportunities in the global alpha-1 antitrypsin deficiency Increasing demand for Alpha-1 Antitrypsin Deficiency therapies is anticipated to support the growth of the global alpha-1 antitrypsin deficiency market during the forecast period 2025-2035. The global alpha-1 antitrypsin deficiency market is expected to grow at a significant rate due to advancements in diagnostic technologies, the development of innovative therapies, and increasing awareness among patients and healthcare providers. Recent Developments: Regulatory Activities: In March 2025, Beam Therapeutics announced that the U.S. FDA cleared its Investigational New Drug (IND) application for BEAM-302, a base-editing therapy for AATD. This marks the first clinical advancement of a base-editing approach for AATD, with initial Phase 1/2 trial data showing promising results in correcting the PiZ mutation associated with the disease. Regulatory Activities: In February 2025, Grifols completed enrollment of the second cohort in its Phase 1/2 study for Alpha-1 15%, a subcutaneous AATD treatment. This development could provide patients with a more convenient administration option, enhancing treatment adherence and quality of life. Acquisition: In May 2024, Sanofi completed its acquisition of Inhibrx, Inc., gaining control of INBRX-101, a recombinant human AAT-Fc fusion protein under development for AATD treatment. INBRX-101 aims to normalize serum AAT levels with less frequent dosing, potentially offering a significant advancement in AATD therapy. Market Dynamics The following are the drivers for the global alpha-1 antitrypsin deficiency market: Increasing Awareness and Early Diagnosis Advancements in Treatment Options The global alpha-1 antitrypsin deficiency market is expected to face some limitations too, due to the following challenges: High Treatment Costs Limited Awareness in Emerging Markets Some of the prominent names established in this market are: Grifols CSL Behring Takeda Pharmaceuticals LFB Biotechnologies GlaxoSmithKline plc AstraZeneca Merck Pfizer Inc. Key Topics Covered: Executive SummaryScope and Definition1. Global Alpha-1 Antitrypsin Deficiency Market: Market Outlook1.1 Industry Outlook1.1.1 Market Overview and Ecosystem1.1.2 Market Trends1.1.3 Epidemiological Analysis of Alpha-1 Antitrypsin Deficiency1.1.3.1 By Region1.1.4 Clinical Trials1.1.4.1 By Phase1.1.4.2 By Sponsor Type1.1.5 Regulatory Landscape / Compliance1.1.5.1 Legal Requirement and Framework in the U.S.1.1.5.2 Legal Requirement and Framework in the E.U.1.1.5.3 Legal Requirement and Framework in Japan1.1.5.4 Legal Requirement and Framework in Rest-of-the-World1.2 Market Dynamics1.2.1 Impact Analysis1.2.2 Market Drivers1.2.3 Market Restraints1.2.4 Market Opportunities2. Global Alpha-1 Antitrypsin Deficiency Market, By Treatment, $Million, 2023-20352.1 Overview2.2 Augmentation Therapy2.3 Bronchodilators2.4 Corticosteroids2.5 Oxygen Therapy3. Global Alpha-1 Antitrypsin Deficiency Market, By Distribution Channel, $Million, 2023-20353.1 Overview3.2 Hospitals Pharmacy3.3 Retail Pharmacies3.4 Online Pharmacies4. Global Alpha-1 Antitrypsin Deficiency Market, By Region, $Million, 2023-20354.1 North America4.1.1 Key Findings4.1.2 Market Dynamics4.1.3 Market Sizing and Forecast4.1.3.1 North America Alpha-1 Antitrypsin Deficiency Market (by Distribution Channel)4.1.3.2 North America Alpha-1 Antitrypsin Deficiency Market (by Country)4.1.3.2.1 U.S.4.1.3.2.2 Canada4.2 Europe4.2.1 Key Findings4.2.2 Market Dynamics4.2.3 Market Sizing and Forecast4.2.3.1 Europe Alpha-1 Antitrypsin Deficiency Market (by Distribution Channel)4.2.3.2 Europe Alpha-1 Antitrypsin Deficiency Market (by Country)4.3 Asia-Pacific4.4 Rest-of-the-World5. Global Alpha-1 Antitrypsin Deficiency Market - Competitive Landscape and Company Profiles5.1 Competitive Landscape5.1.1 Growth Share Matrix (2024)5.1.1.1 By Treatment5.1.2 Key Strategies and Developments by Company5.1.2.1 Funding Activities5.1.2.2 Mergers and Acquisitions5.1.2.3 Regulatory Approvals5.1.2.4 Partnerships, Collaborations and Business Expansions5.1.3 Key Developments Analysis5.2 Company Profiles5.2.1 Company Overview5.2.2 Product Portfolio5.2.3 Target Customers/End Users5.2.4 Analyst View6. Research Methodology For more information about this report visit About is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. CONTACT: CONTACT: Laura Wood,Senior Press Manager press@ For E.S.T Office Hours Call 1-917-300-0470 For U.S./ CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
30-05-2025
- Business
- Yahoo
FDA Grants Orphan Drug Status to Beam Therapeutics Inc. (BEAM)' AATD Gene Therapy
Beam Therapeutics Inc. (NASDAQ:BEAM) has secured U.S. FDA orphan drug designation for BEAM-302, its pioneering genetic medicine targeting alpha-1 antitrypsin deficiency (AATD), a rare inherited disorder that can cause severe lung and liver disease. BEAM-302, delivered via liver-targeting lipid nanoparticles, is designed to correct the underlying DNA mutation responsible for AATD, offering hope for a one-time, potentially curative treatment. This regulatory milestone follows the recent Regenerative Medicine Advanced Therapy (RMAT) designation, underscoring the therapy's promise and the urgent need for effective AATD solutions. A biotechnologist in a lab coat discussing a therapeutic antibody with a colleague. Preliminary data from ongoing Phase 1/2 trials show BEAM-302 is well tolerated and achieves dose-dependent, durable correction of the disease-causing mutation, with protein levels exceeding therapeutic thresholds in the 60 mg dose group. Dosing in higher cohorts is underway, with more results from Beam Therapeutics Inc. (NASDAQ:BEAM) expected later in 2025. Orphan drug designation brings significant benefits, including tax credits, user fee exemptions, and up to seven years of market exclusivity post-approval, accelerating development for this first-in-class base editing therapy for AATD, a condition with no current curative treatments. While we acknowledge the potential of BEAM to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than BEAM and that has 100x upside potential, check out our report about this READ NEXT: and Disclosure: None. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
29-05-2025
- 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
Yahoo
27-05-2025
- Business
- Yahoo
Citi Downgrades Prime Medicine (PRME) to Neutral, Cuts PT to $1.50 Due to Market Uncertainty
On Tuesday, Citi downgraded Prime Medicine Inc. (NASDAQ:PRME) from a Buy to a Neutral rating while also significantly reducing its price target from $10 to $1.50. Citi said that this adjustment reflects that the company's shares are trading near cash, which shows the market's uncertainty on Prime Medicine's path to potential value inflection. A scientist examining a microchip and circuit board in a hi-tech lab. Despite implementing cost-cutting measures, which include a 25% workforce reduction and prioritizing its liver disease franchise & externally funded programs over Chronic Granulomatous Disease/CGD programs, Prime Medicine's cash reserves are projected to support operations only into H1 2026. This timeline falls short of management's guidance, which anticipates initial in-vivo clinical data for Wilson's disease and Alpha-1 antitrypsin deficiency/AATD in 2027. The company also plans to file an investigational new drug/IND application for these programs in H1 2026 and mid-2026, respectively. Prime Medicine is seeking business development deals to secure non-dilutive capital and extend its financial runway. It continues its cystic fibrosis program, supported by the Cystic Fibrosis Foundation, and collaborates with Bristol Myers Squibb on Prime Edited CAR-T products in hematology, immunology, and oncology. Prime Medicine Inc. (NASDAQ:PRME) is a biotech company that delivers genetic therapies to address the spectrum of diseases by deploying gene editing technology in the US. While we acknowledge the potential of PRME to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than PRME and that has 100x upside potential, check out our report about the cheapest AI stock. READ NEXT: and . Disclosure: None. This article is originally published at Insider Monkey. Sign in to access your portfolio
