BOSTON--(BUSINESS WIRE)--Dec 8, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced longer-term data for CASGEVYTM (exagamglogene autotemcel) from global clinical trials in people with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). CASGEVY is the first and only approved CRISPR/Cas9 gene-edited therapy. The results, presented at the American Society of Hematology (ASH) Annual Meeting and Exposition, continue to demonstrate the transformative, durable clinical benefits of CASGEVY. The longest follow up for both SCD and TDT patients now extends more than 5 years, with a median of 33.2 months and 38.1 months, respectively. “These comprehensive data provide additional evidence of the benefits of eradicating transfusion requirements for people with transfusion-dependent beta thalassemia and vaso-occlusive crises for those with sickle cell disease,” said Franco Locatelli, M.D., Ph.D., Professor of Pediatrics at the Catholic University of the Sacred Heart of Rome, Director of the Department of Pediatric Hematology and Oncology at Bambino Gesù Children’s Hospital, Chair of Vertex’s TDT Program Steering Committee, and Presenting Author of the CASGEVY clinical data at ASH. “With median follow-up around three years there is strong evidence for the durability of these beneficial effects following treatment with CASGEVY.” “CASGEVY is changing the outlook for people living with sickle cell disease and beta thalassemia, with these data reinforcing the immense clinical value a durable one-time therapy can provide to patients,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “We have a strong commitment to build on our progress in bringing CASGEVY to patients around the world.” New long-term follow-up data presented from the CASGEVY trials Vertex had seven abstracts accepted at the ASH annual meeting as outlined below: Progress in bringing CASGEVY to patients around the world CASGEVY is approved for both SCD and TDT in the U.S., the European Union, Great Britain, Canada, Switzerland, Bahrain and the Kingdom of Saudi Arabia, and Vertex plans to make submissions in the United Arab Emirates and Kuwait. More than 45 authorized treatment centers have been activated globally to support the delivery of CASGEVY, and more than 40 patients have had a first cell collection. Vertex is continuing to work with reimbursement authorities to secure sustainable access for patients. Through this work, Vertex has agreements to provide CASGEVY in multiple countries, including the U.S., England (TDT), Austria, Bahrain and the Kingdom of Saudi Arabia, and continues to make strong progress in others, including positive Health Technology Assessments (HTAs) in Canada for both diseases and advancing access negotiations for SCD patients in England. In the U.S., Vertex recently secured an industry-first, voluntary agreement with the Centers for Medicare & Medicaid Services (CMS) on a single outcomes-based arrangement available to all state Medicaid programs to ensure broad and equitable access to CASGEVY. To support this progress on patient access and growing patient demand, Vertex has received approval for a third manufacturing facility for CASGEVY with our partner Lonza. About Sickle Cell Disease (SCD) SCD is a debilitating, progressive and life-shortening disease. SCD patients report health-related quality of life scores well below the general population, and the lifetime health care costs in the U.S. of managing SCD for patients with recurrent VOCs is estimated between $4 and $6 million. SCD is an inherited blood disorder that affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. SCD causes severe pain, organ damage and shortened life span due to misshapen or “sickled” red blood cells. The clinical hallmark of SCD is VOCs, which are caused by blockages of blood vessels by sickled red blood cells and result in severe and debilitating pain that can happen anywhere in the body at any time. SCD requires a lifetime of treatment and results in a reduced life expectancy. In the U.S., the median age of death for patients living with SCD is approximately 45 years. A cure for SCD today is a stem cell transplant from a matched donor, but this option is only available to a small fraction of patients living with SCD because of the lack of available donors. About Transfusion-Dependent Beta Thalassemia (TDT) TDT is a serious, life-threatening genetic disease. TDT patients report health-related quality of life scores below the general population and the lifetime health care costs in the U.S. of managing TDT are estimated between $5 and $5.7 million. TDT requires frequent blood transfusions and iron chelation therapy throughout a person’s life. Due to anemia, patients living with TDT may experience fatigue and shortness of breath, and infants may develop failure to thrive, jaundice and feeding problems. Complications of TDT can also include an enlarged spleen, liver and/or heart, misshapen bones and delayed puberty. TDT requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. In the U.S., the median age of death for patients living with TDT is 37 years. Stem cell transplant from a matched donor is a curative option but is only available to a small fraction of people living with TDT because of the lack of available donors. About CASGEVYTM (exagamglogene autotemcel [exa-cel]) CASGEVYTM is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world. About the CLIMB Trials The ongoing Phase 1/2/3 open-label trials, CLIMB-111 and CLIMB-121, are designed to assess the safety and efficacy of a single dose of CASGEVY in patients ages 12 to 35 years with TDT or with SCD and recurrent VOCs. The trials are closed for enrollment. Patients will be followed for approximately two years after CASGEVY infusion in these trials. Each patient will be asked to participate in the ongoing long-term, open-label trial, CLIMB-131. CLIMB-131 is designed to evaluate the long-term safety and efficacy of CASGEVY in patients who received CASGEVY, including those in other CLIMB trials. The trial is designed to follow patients for up to 15 years after CASGEVY infusion. U.S. INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR CASGEVY (exagamglogene autotemcel) WHAT IS CASGEVY? CASGEVY is a one-time therapy used to treat people aged 12 years and older with: • sickle cell disease (SCD) who have frequent vaso-occlusive crises or VOCs • beta thalassemia (β-thalassemia) who need regular blood transfusions CASGEVY is made specifically for each patient, using the patient’s own edited blood stem cells, and increases the production of a special type of hemoglobin called hemoglobin F (fetal hemoglobin or HbF). Having more HbF increases overall hemoglobin levels and has been shown to improve the production and function of red blood cells. This can eliminate VOCs in people with sickle cell disease and eliminate the need for regular blood transfusions in people with beta thalassemia. IMPORTANT SAFETY INFORMATION What is the most important information I should know about CASGEVY? After treatment with CASGEVY, you will have fewer blood cells for a while until CASGEVY takes hold (engrafts) into your bone marrow. This includes low levels of platelets (cells that usually help the blood to clot) and white blood cells (cells that usually fight infections). Your doctor will monitor this and give you treatment as required. The doctor will tell you when blood cell levels return to safe levels. You may experience side effects associated with other medicines administered as part of the treatment regimen for CASGEVY. Talk to your physician regarding those possible side effects. Your healthcare provider may give you other medicines to treat your side effects. How will I receive CASGEVY? Your healthcare provider will give you other medicines, including a conditioning medicine, as part of your treatment with CASGEVY. It’s important to talk to your healthcare provider about the risks and benefits of all medicines involved in your treatment. After receiving the conditioning medicine, it may not be possible for you to become pregnant or father a child. You should discuss options for fertility preservation with your healthcare provider before treatment. STEP 1: Before CASGEVY treatment, a doctor will give you mobilization medicine(s). This medicine moves blood stem cells from your bone marrow into the blood stream. The blood stem cells are then collected in a machine that separates the different blood cells (this is called apheresis). This entire process may happen more than once. Each time, it can take up to one week. During this step rescue cells are also collected and stored at the hospital. These are your existing blood stem cells and are kept untreated just in case there is a problem in the treatment process. If CASGEVY cannot be given after the conditioning medicine, or if the modified blood stem cells do not take hold (engraft) in the body, these rescue cells will be given back to you. If you are given rescue cells, you will not have any treatment benefit from CASGEVY. STEP 2: After they are collected, your blood stem cells will be sent to the manufacturing site where they are used to make CASGEVY. It may take up to 6 months from the time your cells are collected to manufacture and test CASGEVY before it is sent back to your healthcare provider. STEP 3: Shortly before your stem cell transplant, your healthcare provider will give you a conditioning medicine for a few days in hospital. This will prepare you for treatment by clearing cells from the bone marrow, so they can be replaced with the modified cells in CASGEVY. After you are given this medicine, your blood cell levels will fall to very low levels. You will stay in the hospital for this step and remain in the hospital until after the infusion with CASGEVY. STEP 4: One or more vials of CASGEVY will be given into a vein (intravenous infusion) over a short period of time. After the CASGEVY infusion, you will stay in hospital so that your healthcare provider can closely monitor your recovery. This can take 4-6 weeks, but times can vary. Your healthcare provider will decide when you can go home. What should I avoid after receiving CASGEVY? What are the possible or reasonably likely side effects of CASGEVY? The most common side effects of CASGEVY include: Your healthcare provider will test your blood to check for low levels of blood cells (including platelets and white blood cells). Tell your healthcare provider right away if you get any of the following symptoms: These are not all the possible side effects of CASGEVY. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. General information about the safe and effective use of CASGEVY Talk to your healthcare provider about any health concerns. Please see full Prescribing Information including Patient Information for CASGEVY. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on LinkedIn , Facebook , Instagram , YouTube and X . (VRTX-GEN) Vertex Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the statements by Franco Locatelli, M.D., Ph.D. and Carmen Bozic, M.D., in this press release, and statements regarding expectations for the anticipated transformative, durable clinical benefits of CASGEVY, plans to continue working with reimbursement authorities to secure sustainable access for patients, including our expectations for progress in Canada and England, and our plans for and design of the CLIMB studies. While we believe the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that eligible patient access to CASGEVY may not be achieved on the anticipated timeline, or at all, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy, and other reasons, and other risks listed under the heading “Risk Factors” in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company's website at www.vrtx.com . You should not place undue reliance on these statements, or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. View source version on businesswire.com : https://www.businesswire.com/news/home/20241206536804/en/ CONTACT: Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@vrtx.comMedia : mediainfo@vrtx.com or International: +44 20 3204 5275 or U.S.: 617-341-6992 or Heather Nichols: +1 617-839-3607 KEYWORD: MASSACHUSETTS EUROPE UNITED STATES NORTH AMERICA INDUSTRY KEYWORD: BIOTECHNOLOGY PHARMACEUTICAL HEALTH CLINICAL TRIALS SOURCE: Vertex Pharmaceuticals Incorporated Copyright Business Wire 2024. PUB: 12/08/2024 12:52 PM/DISC: 12/08/2024 12:50 PM http://www.businesswire.com/news/home/20241206536804/en
Inside Alistair Johnston’s crazy career from live TV transfers to Dolly Parton’s hometown & signing new Celtic contractFor the second time in four seasons, the Montana State football team will face UT Martin at home in the second round of the FCS playoffs. Unseeded UT Martin (9-4) cruised to a 41-10 win at No. 16-seeded New Hampshire (8-5) on Saturday to set up next week’s second-round game against top-seeded MSU (12-0).
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Voice search has revolutionized how users interact with technology and this will increasingly be the case in the years to come. By 2025, more than half of all Internet searches will be voice based. Now that voice search is on the rise this has to become a part of any digital marketing strategy, trying to optimize website for voice search. Unlike text based searches, voice queries tend to be more conversational, localized, and just in the moment. In this article, we cover all basic things that are required for essential voice search optimization tips for 2025, including natural language use, local SEO and mobile optimization. Devices like smart speakers, mobile assistants, and voice activated apps have all made enable us to enjoy voice search as an integral part of our daily lives. Nowadays, users expect fast, precise and convenient answers to a query, without needing to look at a screen. In 2025, voice search will play a key role in shaping SEO practices . Websites optimized for voice search are more likely to rank higher, attract more organic traffic, and deliver a better user experience. However, as businesses continue to evolve in the Digital Age this is growing in importance and as such businesses must evolve their SEO strategies to meet the unique demands of voice based interactions. The main difference between a voice search and a text search is how the users phrase their queries. Voice search tends to be more conversational, relying on natural language patterns. People often use longer, question-based phrases when speaking to voice assistants, asking questions such as “What’s the best Italian restaurant near me?” instead of typing “Italian restaurant nearby.” To optimize for voice search, using conversational keywords becomes crucial. Instead of short, generic keywords, focus on longer-tail phrases that reflect how people naturally speak. These long-tail keywords better match the structure of voice search queries. Integrating question-based formats like “who,” “what,” “where,” “why,” and “how” can also help capture relevant traffic. Understandably, voice search optimization is important for performing well in local search, since your users frequently perform voice queries trying to find location-related stuff. Voice searches usually include phrases such as “near me” or “closest”, which means that businesses have to put more attention to the local SEO. It can help improve visibility by ensuring that business information is consistent across platforms like Google My Business, Bing and Local directories. However, it is important to get your business profile setup on business profiles by having all the accurate location details, opening hours, contact information and user friendly descriptions about your business for your local SEO to be good. It also encourages customer reviews, as many voice search assistants pull review data when suggesting local businesses. Since there are more voice searches coming from nearby locations “near me” searches, optimizing a business for “near me” searches and localized content can greatly increase a business’s chances of showing up in localized voice searches results. The fact is that voice assistants commonly draw their answers from the featured snippets, which are also referred to as position zero on Google’s search results page. These snippets are concise summaries of answers to a user’s query and are crucial for voice search because the assistant often reads them aloud as the answer. To optimize for featured snippets, create clear, concise, and structured content that answers specific questions. Using bulleted lists, short paragraphs, and direct answers improves the chances of securing a snippet spot. Focusing on question-based search queries also increases the likelihood of being selected as the featured snippet. The technical performance of a website plays a vital role in voice search rankings. Websites must load quickly and be mobile-friendly, as voice search users often conduct queries on mobile devices. A slow website can lead to lower rankings, as search engines prioritize fast-loading pages for voice search results. Optimize site speed by compressing images, using a reliable hosting service, and minimizing unnecessary code. Make sure that websites are responsive, adapting smoothly to various screen sizes and devices. Mobile optimization and site speed improvements enhance the overall user experience, which is critical for ranking well in voice searches. As voice search continues to evolve, optimizing for it will become increasingly important for businesses looking to stay competitive in the digital landscape. Focusing on conversational keywords, improving local SEO, targeting featured snippets, and ensuring website speed and mobile responsiveness are essential steps for voice search optimization in 2025. 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Copy link Copied Copy link Copied Subscribe to gift this article Gift 5 articles to anyone you choose each month when you subscribe. Already a subscriber? Login One of the Nine Network’s highest-profile reporters is suing the broadcaster amid the fallout of a major sexual harassment scandal and a workplace cultural review that has led to the departure of senior executives. Airlie Walsh filed a claim against the company in the Federal Court this week. Her lawyers declined to comment, citing court rules. Copy link Copied Copy link Copied Subscribe to gift this article Gift 5 articles to anyone you choose each month when you subscribe. Already a subscriber? Login Introducing your Newsfeed Follow the topics, people and companies that matter to you. Latest In Media & marketing Fetching latest articles Most Viewed In Companies
Ousted Syrian leader Assad flees to Moscow after fall of Damascus, Russian state media sayMELBOURNE, Australia -- The Australian Senate on Thursday began considering a ban on children younger than 16 years old from social media after the House of Representatives overwhelmingly supported the age restriction. The world-first bill that would make platforms including TikTok, Facebook, Snapchat, Reddit, X and Instagram liable for fines of up to 50 million Australian dollars ($33 million) for systemic failures to prevent young children from holding accounts is likely to be passed by the Senate on Thursday, the Parliament’s final session for the year and potentially the last before elections, which are due within months. The major parties’ support for the ban all but guarantees the legislation will become law. But many child welfare and mental health advocates are concerned about unintended consequences. Unaligned Sen. Jacqui Lambie complained about the limited amount of time the government gave the Senate to debate the age restriction, which she described as “undercooked.” “I thought this was a good idea. A lot of people out there thought it was a good idea until we looked at the detail and, let's be honest, there's no detail,” Lambie told the Senate. The House of Representatives on Wednesday overwhelmingly carried the bill 102 votes to 13. Once the legislation becomes law, the platforms would have one year to work out how they could implement the ban before penalties are enforced. The platforms complained that the law would be unworkable, and urged the Senate to delay the vote until at least June next year when a government-commissioned evaluation of age assurance technologies made its report on how young children could be excluded. Critics argue the government is attempting to convince parents it is protecting their children ahead of general elections due by May. The government hopes that voters will reward it for responding to parents' concerns about their children's addiction to social media. Some argue the legislation could cause more harm than it prevents. Criticisms include that the legislation was rushed through Parliament without adequate scrutiny, is ineffective, poses privacy risks for all users, and undermines parental authority to make decisions for their children. Opponents of the bill also argue the ban would isolate children, deprive them of the positive aspects of social media, drive them to the dark web, discourage children too young for social media to report harm and reduce incentives for platforms to improve online safety.