Metabolic Diseases

MB-111 is a potential first-in-class in vivo ultracompact CRISPR therapy for Familial Chylomicronemia Syndrome and Severe Hypertriglyceridemia Dr. Brown brings deep expertise in nucleic acid drug development as Mammoth advances its lead program, MB-111, to IND-enabling stage and continues to build a leading company in genetic medicines BRISBANE, CA, USA I May 05, 2025 I Mammoth Biosciences, Inc. , a biotechnology company harnessing its proprietary next-generation CRISPR gene editing platform to create potential one-time curative therapies, today announced the nomination of its first clinical development candidate, MB-111, and the appointment of biotechnology industry veteran, Bob D. Brown, Ph.D., to its Board of Directors. MB-111 utilizes CasPhi — an ultracompact CRISPR in vivo gene editing system that is less than half the size of first-generation, Cas9-based systems — encapsulated in a lipid nanoparticle for delivery to the liver after IV administration. Share MB-111 utilizes CasPhi — an ultracompact CRISPR in vivo gene editing system that is less than half the size of first-generation, Cas9-based systems — encapsulated in a lipid nanoparticle for delivery to the liver after IV administration. MB-111 has the potential to be a first-in-class, one-time treatment for patients with very high triglycerides including familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG). MB-111 is designed to permanently disrupt the expression of the APOC3 gene in the liver, and thus to reduce expression of ApoC-III protein. ApoC-III is a critical driver of lipid metabolism and disrupting its production has been shown to reduce plasma triglycerides in patients with pathologically elevated levels, including those with FCS and SHTG. These patients suffer from recurrent episodes of acute pancreatitis, leading to frequent hospitalizations, as well as an increased risk of cardiovascular disease. Mammoth Biosciences is on track to initiate IND-enabling studies this year. “The nomination of our lead program as a development candidate is a major milestone for Mammoth and the gene editing field, as well as the first proof point of the therapeutic potential of our novel ultracompact CRISPR systems and gene editing technologies,” said Trevor Martin, Ph.D., co-founder and Chief Executive Officer of Mammoth Biosciences. “The preclinical data on MB-111 is compelling, and we’re excited about its potential to be a first-in-class genetic cure for debilitating diseases such as FCS and SHTG. We believe Dr. Brown’s extensive drug discovery and development experience, and track record of building leading companies in siRNA and antisense oligonucleotide therapeutics across rare and chronic diseases will be invaluable to our goal of increasing the number of patients who can derive benefit from Mammoth Biosciences’ genetic medicines.” Dr. Bob D. Brown brings over 30 years of experience in multidisciplinary biotechnology research and drug development, including in the US and ex-US jurisdictions. Most recently, Dr. Brown served as Chief Scientific Officer and Executive Vice President of R&D at Dicerna Pharmaceuticals, an RNAi-focused therapeutics company that was acquired by Novo Nordisk. At Novo, he served as President and Head of the Dicerna Transformation Research Unit and SVP. During his time at Dicerna, he led the discovery and early clinical development of numerous genetic medicines, including nedosiran for the treatment of primary hyperoxaluria, RG6346 for the treatment of chronic hepatitis B, belcesiran for the treatment of alpha-1 antitrypsin deficiency, and several other drug candidates now in the clinical development pipelines of large pharmaceutical companies. Prior to Dicerna, Dr. Brown held various positions at Genta, a clinical-stage antisense oligonucleotide therapeutics company, most recently as its Vice President of Research and Technology. Previously, he was a co-founder and Vice President of R&D of Oasis Biosciences, which was acquired by Gen-Probe. Dr. Brown is an inventor or co-inventor of more than 85 issued US patents, and earned a Ph.D. in molecular biology from the University of California, Berkeley and B.S. degrees in chemistry and biology from the University of Washington, Seattle. “I’m honored to join Mammoth Biosciences at such a pivotal moment, as CRISPR technology stands ready to transform healthcare and redefine the future of medicine,” said Dr. Brown. “I look forward to working with the exceptional Mammoth Biosciences team to harness the power of their groundbreaking platform to tackle some of the most pressing medical challenges and improve patients’ lives. Drawing on my experiences in drug discovery and clinical development, I aim to help advance Mammoth Biosciences’ ultracompact CRISPR gene editing therapies—starting with MB-111—toward clinical application.” About Mammoth Biosciences Mammoth Biosciences is a biotechnology company focused on leveraging its proprietary ultracompact CRISPR systems to develop potential long-term curative therapies for patients with life-threatening and debilitating diseases. Founded by CRISPR pioneer and Nobel laureate Jennifer Doudna and Trevor Martin, Janice Chen, and Lucas Harrington, the company’s ultracompact systems are designed to be more specific and enable in vivo gene editing in difficult to reach tissues utilizing both nuclease applications and new editing modalities beyond double stranded breaks, including base editing, reverse transcriptase editing, and epigenetic editing. The company is building out its wholly owned pipeline of potential in vivo gene editing therapeutics and capabilities and has partnerships with leading pharmaceutical and biotechnology companies to broaden the reach of its innovative and proprietary technology platform. Mammoth Biosciences’ deep science and industry experience, along with a robust and differentiated intellectual property portfolio, have enabled the company to further its mission to transform the lives of patients and deliver on the promise of CRISPR technologies. SOURCE: Mammoth Biosciences

Equity Insider News Commentary Issued on behalf of Avant Technologies Inc. VANCOUVER, BC, May 5, 2025 /PRNewswire/ --The integration of artificial intelligence (AI) into healthcare is here to stay, and adoption is accelerating. According to research from PYMNTS Intelligence which polled C-suite executives at healthcare companies generating at least $1 billion in annual revenue, 90% already see positive ROI from investments in generative AI (GenAI). Analysts are projecting that by 2035 the gross value added by AI to the healthcare industry will be $461 billion on top of a baseline $2.26 trillion. Behind the scenes, several new AI healthcare tech developments are taking place, with updates recently coming from Avant Technologies, Inc. (OTCQB: AVAI), Tempus AI, Inc. (NASDAQ: TEM), Tevogen Bio Holdings Inc. (NASDAQ: TVGN), GE HealthCare Technologies Inc. (NASDAQ: GEHC), and Schrödinger, Inc. (NASDAQ: SDGR). At the regulatory level, the World Health Organization (WHO) recently announced a major global initiative to set unified standards for AI in healthcare, the US Food and Drug Administration (FDA) is working to catch up on approving new AI tech in the sector, with the latest being a cardioverter defibrillator. Avant Technologies, Inc. (OTCQB: AVAI), an emerging leader in AI-driven healthcare innovation, continues to accelerate its push into AI-powered healthcare diagnostics through its joint venture with Ainnova Tech, operating through Ai-nova Acquisition Corp. (AAC), a jointly controlled entity that holds global licensing rights to VisionAI and its supporting hardware. The two companies recently announced that the FDA has officially received their request for a pre-submission meeting for VisionAI, the joint venture's flagship screening platform. This marked a pivotal moment in Avant's trajectory. VisionAI uses a combination of retinal imaging, patient vitals, and advanced algorithms to identify early warning signs of diabetic retinopathy, cardiovascular disease, liver fibrosis, kidney disease, and type 2 diabetes—often before symptoms are visible. Studies show that AI tools can detect diabetic retinopathy and other chronic conditions with sensitivities exceeding 90%, often identifying issues before symptoms appear—according to NIH. "This milestone reflects our two-tiered strategy, rapid deployment in low-regulation markets where VisionAI operates as a screening tool, and simultaneous progress toward FDA clearance for the U.S. market," said Vinicio Vargas, CEO at Ainnova and a member of AAC's Board of Directors. "Entering the U.S. will unlock significant commercial potential, and early engagement with regulators ensures we do so with speed, credibility, and a validated product." The upcoming FDA meeting will help finalize the clinical trial design and confirm the regulatory pathway for a 510(k) submission in the U.S., setting the stage for broader deployment. What sets AVAI apart from many AI-in-healthcare names is that its technology is already in the field. VisionAI has been rolled out through commercial pilot programs across Latin America, including Chile, the Dominican Republic, Mexico, and Brazil. These early deployments are generating real-world data while demonstrating how early diagnostics can reduce system-wide costs and improve outcomes in primary care environments. In recent months, AAC expanded its portfolio with four newly licensed diagnostic algorithms from one of Asia's largest healthcare networks, validated on more than 2.3 million clinical cases. These models are now integrated into VisionAI to enhance its detection capabilities across multiple chronic disease categories. On the clinical development front, Avant's partner Ainnova has brought on global contract research organization Fortrea to help guide its FDA strategy. With both emerging-market traction and U.S. regulatory progress advancing in parallel, the company appears to be executing on a dual-pronged strategy: building early market access where regulatory barriers are lower, while preparing for the commercial scale and reimbursement potential of the U.S. healthcare system. For investors tracking small-cap companies operating at the intersection of AI, diagnostics, and chronic disease management, Avant Technologies may be worth a closer look. As the FDA process moves forward and pilot programs scale, AVAI sits in a rare position—commercially active, globally licensed, and now formally in dialogue with U.S. regulators. Tempus AI, Inc. (NASDAQ: TEM) recently expanded its support for phase I clinical trials by launching the TIME Precision Network, a group of investigators across over 40 research centers focused on rapid activation and enrollment. "The integration of phase I clinical trial sites adds a critical capability to the TIME Network,"said Ezra Cohen, MD, Chief Medical Officer, Oncology at Tempus. "We are now able to activate and efficiently execute these early studies that are fundamental to drug development and such a valuable offering to patients. The reality is that these studies require specific and coordinated infrastructure to be successful, and I am proud that Tempus can now work with these sites and our life science partners to enroll patients into these cutting-edge clinical trials." Recent examples include first-patient-in enrollments within weeks at multiple cancer centers, showcasing the network's efficiency in matching patients to studies. By integrating community oncology sites and streamlining site activation, Tempus is lowering access barriers to early-stage trials across underserved regions. Tevogen Bio Holdings Inc. (NASDAQ: TVGN) is advancing its AI-driven precision medicine capabilities through a new partnership with Databricks, focused on accelerating the development of its PredicTcell platform. The collaboration will enhance Tevogen's ability to model immunologically active peptide complexes and predict T cell receptor (TCR) engagement. This builds on the company's ExacTcell™ platform and complements other strategic alliances under the Tevogen.AI umbrella, including support from Microsoft Research. Together, these efforts aim to integrate clinical immunology with cutting-edge AI to fast-track the discovery and development of next-generation T cell therapies. GE HealthCare Technologies Inc. (NASDAQ: GEHC) is deepening its commitment to AI-driven oncology solutions with the launch of MR Contour DL, a new FDA-cleared model for organ segmentation in radiation therapy planning. "We're committed to advancing MR imaging capabilities in radiation therapy to deliver more accurate and precise care," said Kelly Londy, President and CEO, MR, GE HealthCare. "The anticipated addition of Spectronic's software offering to our portfolio would support our vision for an MR-based radiation oncology workflow and would help enable high-precision treatment planning for radiotherapy patients across the globe." The company also announced its intent to integrate Spectronic Medical's MRI Planner software to support MR-only workflows across global radiotherapy centers. These tools are being folded into the expanded Intelligent Radiation Therapy (iRT) solution, which now supports third-party AI applications and accelerates the treatment timeline. By combining MR imaging, automation, and deep learning, GE HealthCare aims to raise the precision and accessibility of radiotherapy worldwide. Schrödinger, Inc. (NASDAQ: SDGR) recently presented new preclinical data at AACR 2025 on two targeted cancer therapies developed using its proprietary computational platform. Among the findings, a machine learning model predicted synergistic drug combinations for its Wee1 inhibitor SGR-3515, enhancing its potential use across multiple tumor types. "The preclinical data for SGR-3515 and SGR-4174 further demonstrate that molecules discovered and developed by Schrödinger have favorably differentiated molecular profiles compared to existing development-stage molecules," said Karen Akinsanya, Ph.D, President of R&D therapeutics at Schrödinger. "The preclinical profiles of these development candidates reinforce the power of our computationally-driven approach to designing molecules that meet challenging target product profiles and have the potential for meaningful benefit to patients." The company's AI-driven discovery approach continues to yield differentiated molecules with promising therapeutic profiles. As AI continues to evolve from concept to clinical application, these companies are quietly shaping the future of medicine at the infrastructure level. With regulatory momentum building and real-world deployments expanding, investors may want to pay attention as this shift begins to accelerate. CONTACT: Equity Insider [email protected] (604) 265-2873 DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. We are not licensed under securities laws to address your particular financial situation. No communication by our employees to you should be deemed as personalized financial advice. 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MISSION, Kan., May 5, 2025 /PRNewswire/ -- (Family Features) Most people think of heart attacks as debilitating pain in the chest. However, that's not always the case, especially for women, and missing the signs can be a matter of life and death. Although heart disease is the leading cause of death among American women, according to the American Heart Association, symptoms are often overlooked or explained away as less worrisome conditions. Continue Reading Photo courtesy of Shutterstock "Men and women experience many aspects of life differently, and heart disease is no exception," said LeAnne Bloedon, MS, RD, vice president of clinical development, Esperion Therapeutics. "Symptoms of a heart attack aren't as obvious as many women think, and failing to recognize the danger and get help can have catastrophic results." One heart disease risk factor, for example, is high LDL cholesterol. Often referred to as a "silent killer," it doesn't always present noticeable symptoms. In fact, data suggests women with high LDL cholesterol are not diagnosed or treated as early or aggressively as men, which can put women at an increased risk for cardiovascular events. Understanding how men's and women's risk and symptoms differ may help identify and treat a serious heart health problem before it causes lasting damage. Anatomy Differences Some differences between men and women are present in the body's internal systems, including the cardiovascular system. Women generally have smaller hearts and narrower blood vessels. This can affect heart health in numerous ways, including less efficient stress responses and greater risk of widespread plaque buildup, especially in smaller vessels, called microvasculature, which can pose treatment challenges. Cholesterol Buildup Hormones influence aspects of the body's function, including cholesterol levels. Estrogen, a female sex hormone, raises HDL (good) cholesterol levels. This may be why women tend to have more HDL cholesterol and lower LDL (bad) cholesterol than men, especially before they reach menopause, though inherited high cholesterol can affect women of all ages. "It's critical to raise awareness about the importance of measuring LDL cholesterol, diagnosing high cholesterol and treating elevated LDL cholesterol per guidelines and individual patient needs," Bloedon said. Risk Factors While there are shared risk factors for heart disease among men and women (such as obesity, high blood pressure and diabetes), some risk factors disproportionately affect women. For example, uncontrolled cholesterol in women, particularly after menopause, can increase the risk of heart disease and stroke. Care and Treatment "While you may not be able to fully prevent heart disease, you can understand the risks and take proactive steps," Bloedon said. "Commit to a healthy, smoke-free lifestyle. Exercise regularly and eat a well-balanced diet with limits on processed foods, sugar, sodium and alcohol. Take any medications as directed by your health care provider." Statins, which reduce the production of cholesterol in the liver and lower cholesterol levels in the bloodstream, are the medications most often prescribed to help manage high LDL cholesterol. While generally well-tolerated, statin intolerance (the inability to take a statin at any dose or the recommended dose) does occur in some patients and is more common in women. Talk with your doctor about other steps you can take to manage elevated LDL cholesterol levels and learn more about women's heart health at goredforwomen.org. Symptoms of Heart Attacks in Women Women's experiences with heart disease may be quite different from men. For example, according to the Heart Disease Foundation, women are likely to be older when they experience a heart attack. They may also attribute symptoms to other conditions, such as diabetes or arthritis. Further complicating matters, several diseases mimic heart attacks so getting to the root of the problem can be tricky. Women are more apt than men to experience a coronary spasm, coronary dissection or broken heart syndrome. What to Watch For: Like men, women experiencing a heart attack may notice prolonged or reoccurring chest pain or pressure. In women, that pain may extend to the arms, back, neck, jaw and stomach. Some women experience shortness of breath with no chest pain at all. Other symptoms women are more likely to report can be easily mistaken for other conditions. Some of these signs include unexplained fatigue, disruptions to normal sleep patterns, lightheadedness, nausea and cold sweats. Michael French [email protected] 1-888-824-3337 editors.familyfeatures.com About Family Features Editorial Syndicate A leading source for high-quality food, lifestyle and home and garden content, Family Features provides readers with topically and seasonally relevant tips, takeaways, information, recipes, videos, infographics and more. Find additional articles and information at Culinary.net and eLivingToday.com. SOURCE Family Features Editorial Syndicate WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED