BCL11A

LONDON--(BUSINESS WIRE)--Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with the Italian Medicines Agency (AIFA) for eligible transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY® (exagamglogene autotemcel). “Today is a turning point for eligible people in Italy living with transfusion-dependent beta thalassemia and sickle cell disease, two life-shortening diseases with limited treatment options,” said Ludovic Fenaux, Senior Vice President, Vertex International. “Italy has the largest TDT population in Europe, which underscores the importance of this agreement. We appreciate the collaboration with AIFA to recognize the value a one-time transformative treatment provides to patients, families and the health care system.” In Italy, there are approximately 5,000 people 12 years and older living with TDT and around 2,300 with SCD. Italy joins a number of countries that have signed reimbursement agreements for CASGEVY including Austria, Bahrain, Denmark, England, the Kingdom of Saudi Arabia and the United Arab Emirates. 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 significant health care resource utilization. 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 Europe, the mean age of death for patients living with TDT is 50-55 years. About Sickle Cell Disease (SCD) SCD is a debilitating, progressive, life-shortening genetic disease. SCD patients report health-related quality of life scores well below the general population and significant health care resource utilization. SCD 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 vaso-occlusive crises (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 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 Europe, the mean age of death for patients living with SCD is around 40 years. About CASGEVY® (exagamglogene autotemcel) CASGEVY® 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. In the European Union, CASGEVY is approved for patients 12 years of age and older with either severe SCD with recurrent VOCs or TDT, for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available. For complete product information, please see the Summary of Product Characteristics (SmPC) at www.ema.europa.eu. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. 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 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/en-global. 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 Ludovic Fenaux, in this press release, and statements regarding Vertex’s expectations for and the anticipated benefits of CASGEVY, expectations for access to CASGEVY for eligible TDT and SCD patients in Italy, and expectations for the population of people living with TDT and SCD in Europe. 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 data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy, and other reasons, anticipated patient populations may be different than expected, 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. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN)

- CASGEVY is the first, and only, gene editing therapy approved for the treatment of transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD) in Europe - - Italy has the largest population of people living with TDT in Europe - LONDON--(BUSINESS WIRE)--Sep. 18, 2025-- Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with the Italian Medicines Agency (AIFA) for eligible transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY® (exagamglogene autotemcel). “Today is a turning point for eligible people in Italy living with transfusion-dependent beta thalassemia and sickle cell disease, two life-shortening diseases with limited treatment options,” said Ludovic Fenaux, Senior Vice President, Vertex International. “Italy has the largest TDT population in Europe, which underscores the importance of this agreement. We appreciate the collaboration with AIFA to recognize the value a one-time transformative treatment provides to patients, families and the health care system.” In Italy, there are approximately 5,000 people 12 years and older living with TDT and around 2,300 with SCD. Italy joins a number of countries that have signed reimbursement agreements for CASGEVY including Austria, Bahrain, Denmark, England, the Kingdom of Saudi Arabia and the United Arab Emirates. 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 significant health care resource utilization. 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 Europe, the mean age of death for patients living with TDT is 50-55 years. About Sickle Cell Disease (SCD) SCD is a debilitating, progressive, life-shortening genetic disease. SCD patients report health-related quality of life scores well below the general population and significant health care resource utilization. SCD 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 vaso-occlusive crises (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 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 Europe, the mean age of death for patients living with SCD is around 40 years. About CASGEVY® (exagamglogene autotemcel) CASGEVY® 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. In the European Union, CASGEVY is approved for patients 12 years of age and older with either severe SCD with recurrent VOCs or TDT, for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available. For complete product information, please see the Summary of Product Characteristics (SmPC) at www.ema.europa.eu. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. 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 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/en-global. 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 Ludovic Fenaux, in this press release, and statements regarding Vertex’s expectations for and the anticipated benefits of CASGEVY, expectations for access to CASGEVY for eligible TDT and SCD patients in Italy, and expectations for the population of people living with TDT and SCD in Europe. 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 data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy, and other reasons, anticipated patient populations may be different than expected, 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. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN) View source version on businesswire.com: https://www.businesswire.com/news/home/20250916726929/en/ Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@vrtx.com Media: mediainfo@vrtx.com or International: +44 20 3204 5275 or U.S.: 617-341-6992 Source: Vertex Pharmaceuticals

SHANGHAI, Aug. 26, 2025 /PRNewswire/ -- CorrectSequence Therapeutics Co., Ltd. (Correctseq), a clinical-stage biotechnology company pioneering transformer Base Editing (tBE) technology for the treatment of severe diseases, today announced the successful treatment of the first sickle cell disease (SCD) patient using its high-precision base-editing therapy CS-101. Continue Reading Picture: The patient, a 21-year-old woman from Nigeria, had previously suffered from recurrent VOCs. For six months after receiving CS-101, she remains crisis-free and in good health. The breakthrough was achieved in an Investigator-Initiated Trial (IIT) conducted in collaboration with the First Affiliated Hospital of Guangxi Medical University. The patient demonstrated a significant and sustained increase in fetal hemoglobin (HbF) levels, accompanied by a marked reduction in sickle hemoglobin (HbS). Six months post-treatment, the HbF-to-HbS ratio stabilized at 6.5:3.5, with total hemoglobin consistently exceeding 120 g/L. Over the six-month follow-up period, the patient experienced no vaso-occlusive crises (VOCs) and successfully resumed normal daily activities — marking the first reported clinical treatment of sickle cell disease in China using base editing technology. Picture: The patient, a 21-year-old woman from Nigeria, had previously suffered from recurrent VOCs. For six months after receiving CS-101, she remains crisis-free and in good health. Sickle Cell Disease and the Need for Curative Therapies Hemoglobinopathies — including SCD and β-thalassemia (β-thal) — are the world's most common group of monogenic diseases, with approximately 7% of the global population carrying a pathogenic variant. Each year, around 400,000 newborns are affected. SCD, caused by mutations in the β-globin gene, leads to abnormal, sickle-shaped red blood cells, resulting in chronic anemia, recurrent pain crises, increased infection risk, and progressive organ damage. Globally, about 3.5% of the population carries the sickle mutation, with roughly 300,000 affected infants born annually, especially in Africa, the Mediterranean, the Middle East, and South Asia. Existing treatments such as symptomatic drugs and blood transfusions can alleviate symptoms but do not offer a cure. Hematopoietic stem cell transplantation (HSCT) can be curative, but the requirement for a matched donor limits accessibility. Gene-editing approaches, particularly base editing, can activate fetal hemoglobin in a patient's own blood cells — eliminating the need for a donor and offering a shorter, potentially safer treatment path. CS-101: Precision Base Editing for Hemoglobinopathies CS-101 is a gene-editing therapy based on the high-precision transformer Base Editor (tBE) platform (Wang et al., Nat Cell Biol, 2021), developed by Correctseq's scientific co-founders. The therapy targets β-hemoglobinopathies by precisely modifying a regulatory element in the promoter of the γ-globin genes (HBG1/2), mimicking naturally occurring single-nucleotide variants found in individuals with hereditary persistence of fetal hemoglobin. This reactivates γ-globin expression to produce functional HbF, which prevents sickling and reduces hemolysis. In the IIT, the patient began with a baseline hemoglobin level of 67.3 g/L. Following CS-101 treatment in February 2025, she achieved rapid hematopoietic recovery, with neutrophil engraftment in 13 days and platelet counts surpassing 50×10⁹/L in 21 days. Her HbF levels rose from 4.4% to 34.6% within one month, and since the third month post-treatment, HbF levels have remained above 60% while HbS has stayed below 40%. No VOCs or treatment-related adverse events have been reported. A Safer, More Effective Approach Compared with conventional CRISPR/Cas9-based therapies, CS-101 delivers efficient hematopoietic recovery and higher HbF-to-HbS ratio, without the risks of large DNA deletions, chromosomal rearrangements, or off-target mutations. To date, CS-101 has been used to treat nearly 20 patients with β-thalassemia or SCD in clinical trials. The first β-thalassemia patient treated has remained transfusion-free for over 22 months. This positions CS-101 as a potential best-in-class gene-editing therapy — and the world's first base editing treatment — for β-hemoglobinopathies. Global Development and Next Steps The Phase I trial of CS-101 for β-thalassemia has been completed, with all patients achieving transfusion independence. Pivotal Phase II/III trials are set to begin soon, and global recruitment for both SCD and β-thalassemia trials is now underway. Correctseq is committed to advancing CS-101 — a first-in-class, China-originated gene-editing therapy — to deliver safe, effective, and accessible treatments for patients worldwide living with severe hemoglobin disorders. About CorrectSequence Therapeutics CorrectSequence Therapeutics (Correctseq), incubated at ShanghaiTech University, is dedicated to leveraging innovative gene-editing technologies to transform the lives of people with severe diseases. The company has developed multiple state-of-the-art base-editing systems that offer exceptional precision, minimize off-target effects, and enhance in vivo editing efficiency. Correctseq's mission is to discover, develop, manufacture, and commercialize curative genetic medicines across a broad range of conditions. Its robust pipeline spans genetic disorders, metabolic diseases, and cardiovascular conditions, with several programs already advancing toward clinical development. For more information, visit . Acknowledgments: The First Affiliated Hospital of Guangxi Medical University, ShanghaiTech University, Shanghai Clinical Research and Trial Center. Media Contact: Business Cooperate: [email protected] Clinical Trial Recruitment: [email protected] Photo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED