Congenital Disorders

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

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

SANTA BARBARA, Calif., May 5, 2025 /PRNewswire/ -- Cottage Health has partnered with Clearway Health, a specialty pharmacy services company, to develop a specialty pharmacy program that enhances patient access to medications with concierge-level service at a lower cost. This collaboration brings personalized pharmacy support to Cottage Health's specialty outpatient clinics, enhancing patient access to medications. Continue Reading Cottage Health partners with Clearway Health to develop a specialty pharmacy program, enhancing access to medications. Post this “Integrating pharmacy services directly into our clinics allows us to address the unique challenges patients face with specialty medications,” said Lisa Moore, executive vice president and chief operating officer at Cottage Health. “This ensures our patients receive the therapies they need while reducing barriers and improving outcomes.” The program is designed to simplify the prescription process by assisting with insurance approvals, financial assistance, medication delivery, and ongoing support, making it easier for patients to manage their treatment without delays or added stress. As part of the partnership, pharmacists and patient liaisons are integrated directly within Cottage Health's specialty clinics, working closely with patients to navigate insurance coverage, secure financial assistance and manage prior authorizations—alleviating administrative burdens for both patients and providers. "Integrating pharmacy services directly into our clinics allows us to address the unique challenges patients face with specialty medications," said Lisa Moore, executive vice president and chief operating officer at Cottage Health. "This ensures our patients receive the therapies they need while reducing barriers and improving outcomes." The program has already launched at the Cottage Cystic Fibrosis Clinic and pediatric clinics, where patient liaisons are assisting patients with accessing both specialty and non-specialty medications. Specialty medications typically require special handling, administration and monitoring and are used to treat chronic conditions such as multiple sclerosis, cystic fibrosis, cancer and inflammatory conditions. "Clearway Health is pleased to work alongside Cottage Health to provide their patients and community with transformative specialty pharmacy services," said Allison Arant, senior vice president, client development and marketing, Clearway Health. "Cottage Health's patient-first approach and commitment to helping patients get back to living their lives directly coincides with our ability to improve access to care and manage the complex medical needs of patients. Every patient deserves the care we provide. We celebrate and congratulate Cottage Health for choosing to build this program and offer specialty pharmacy services to their patients." Future plans include expanding specialty pharmacy services to other Cottage Health clinics and opening an outpatient retail and specialty pharmacy, further enhancing medication access and convenience for patients. About Clearway Health Clearway Health's specialty pharmacy services create sustainable assets and build lasting legacies for hospitals and health systems, providing a service that every patient deserves. Our team began at Boston Medical Center Health System, improving access to care and managing the complex medication needs of vulnerable patient populations. Equipped with this unique experience to solve challenges, Clearway Health partners with other hospitals and health systems to provide transformative specialty pharmacy services that put patients first. Our strategic partnerships build and strengthen the infrastructure for specialty pharmacy programs through improving operational workflows, implementing clinical programs, hiring and embedding skilled pharmacists and patient liaisons into the clinical team, providing proprietary software technology, unlocking drug access and leading accreditation processes. Clearway Health delivers a clinically driven service that eliminates barriers to medication access, improves the health of patients and communities, grows revenue at healthy margins for health systems and reduces costs for patients. To learn more, visit . About Cottage Health | CottageHealth.org Cottage Health is a not-for-profit health system providing advanced medical care on California's Central Coast. In the past year, its hospitals in Goleta, Santa Barbara and Santa Ynez Valley cared for 20,733 inpatient admissions, 91,649 emergency department visits and 2,005 newborn deliveries. With more than 700 physicians, including specialists typically found only at university medical centers, Cottage offers expert, comprehensive care. As a teaching institution, it trains physicians through residency programs in medical, surgical, pediatric and radiology specialties. Cottage Health is home to the Central Coast's only Level I adult and Level II pediatric trauma center and an advanced comprehensive stroke center. Its specialty services include Cottage Children's Medical Center, Cottage Rehabilitation Hospital, the Santa Barbara Neuroscience Institute, Cottage Heart & Vascular Center and Cottage Center for Orthopedics. Beyond its hospitals, Cottage expands care through primary and specialty clinics, urgent care centers and 24/7 provider access via Cottage Virtual Care. SOURCE Clearway Health WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED