Latest news with #CASGEVY
Associated Press
03-07-2025
- Business
- Associated Press
Vertex Presents Longer-Term Data at the 2025 European Hematology Association (EHA) Congress Demonstrating Durability of CASGEVY® and Provides Update on Expanding Global Access to CASGEVY
- Data from longer-term follow-up of patients in ongoing clinical trials further demonstrate durability of the clinical benefits of CASGEVY® - - Multiple reimbursement agreements secured, expanding access to CASGEVY to more patients around the world - TORONTO, July 3, 2025 /CNW/ - Vertex Pharmaceuticals (Nasdaq: VRTX) recently announced positive longer-term data for PrCASGEVY® (exagamglogene autotemcel) from global ongoing pivotal clinical trials in people with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The results, presented at the European Hematology Association (EHA) Congress, continue to demonstrate the durable clinical benefits of CASGEVY. The longest follow up in SCD patients now extends more than 5.5 years and in TDT patients more than 6 years, with a mean of 39.4 months and 43.5 months, respectively. CASGEVY is the first authorized CRISPR/Cas9 gene-edited therapy. 'This longer-term data reinforces CASGEVY's durable clinical benefits for eligible people living with sickle cell disease or transfusion-dependent beta thalassemia,' said Kevin Kuo, M.D., Hematologist and Associate Professor in the Division of Hematology, University of Toronto, Clinician Investigator in the Red Blood Cell Disorders Clinic at University Health Network, and Principal Investigator for the CLIMB-131 clinical program. 'These results are a reminder of what science can achieve, especially for patients and communities with significant unmet need.' New longer-term follow-up data presented from the CASGEVY trials Progress in bringing CASGEVY to patients Through reimbursement agreements, Vertex has secured access for eligible SCD or TDT patients in multiple countries including Austria, Bahrain, England, Denmark, the Kingdom of Saudi Arabia, Northern Ireland, Scotland, the United Arab Emirates, the United States and Wales. In Canada, CASGEVY received positive recommendations for reimbursement from both Canadian health technology agencies between December 2024 and January 2025; however, a Letter of Engagement from the pan-Canadian Pharmaceutical Alliance (pCPA) is pending. Vertex is continuing to work with government and reimbursement authorities globally to secure sustainable access for additional eligible patients. 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. 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. About PrCASGEVY® (exagamglogene autotemcel) PrCASGEVY® is an autologous genome edited hematopoietic stem cell-based 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 vaso-occlusive crises (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. 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 in select regions 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 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 Kevin Kuo, M.D., in this press release, and statements regarding expectations for the anticipated durable clinical benefits of CASGEVY, expectations for the safety profile of CASGEVY, expectations for the Letter of Engagement from the pCPA, plans to continue working with government and reimbursement authorities globally to secure sustainable access for patients, 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 and available through the company's website at 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. Vertex Pharmaceuticals Incorporated Investors: [email protected] Media: Canada: +1 647-790-1600 or U.S.: 617-341-6992 SOURCE Vertex Pharmaceuticals (Canada) Inc.
Cision Canada
03-07-2025
- Business
- Cision Canada
Vertex Presents Longer-Term Data at the 2025 European Hematology Association (EHA) Congress Demonstrating Durability of CASGEVY® and Provides Update on Expanding Global Access to CASGEVY Français
- Data from longer-term follow-up of patients in ongoing clinical trials further demonstrate durability of the clinical benefits of CASGEVY ® - - Multiple reimbursement agreements secured, expanding access to CASGEVY to more patients around the world - TORONTO, July 3, 2025 /CNW/ - Vertex Pharmaceuticals (Nasdaq: VRTX) recently announced positive longer-term data for Pr CASGEVY ® (exagamglogene autotemcel) from global ongoing pivotal clinical trials in people with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The results, presented at the European Hematology Association (EHA) Congress, continue to demonstrate the durable clinical benefits of CASGEVY. The longest follow up in SCD patients now extends more than 5.5 years and in TDT patients more than 6 years, with a mean of 39.4 months and 43.5 months, respectively. CASGEVY is the first authorized CRISPR/Cas9 gene-edited therapy. "This longer-term data reinforces CASGEVY's durable clinical benefits for eligible people living with sickle cell disease or transfusion-dependent beta thalassemia," said Kevin Kuo, M.D., Hematologist and Associate Professor in the Division of Hematology, University of Toronto, Clinician Investigator in the Red Blood Cell Disorders Clinic at University Health Network, and Principal Investigator for the CLIMB-131 clinical program. "These results are a reminder of what science can achieve, especially for patients and communities with significant unmet need." New longer-term follow-up data presented from the CASGEVY trials In SCD, 43/45 (95.6%) evaluable patients (those with at least 16 months of follow-up) were free from vaso-occlusive crises (VOCs) for at least 12 consecutive months (VF12) in CLIMB-121 and CLIMB-131 combined (95% CI: 84.9%, 99.5%). The mean duration of VOC-free was 35.0 months (range 14.4 to 66.2 months). All evaluable patients (45/45 [100%]) achieved freedom from in-patient hospitalization for severe VOCs for at least 12 consecutive months (HF12) in CLIMB-121 and CLIMB-131 combined (95% CI: 92.1%, 100%), with a mean hospitalization-free of 36.1 months (range 14.5 to 66.2 months). In TDT, 54/55 (98.2%) evaluable patients (those with at least 16 months of follow-up) achieved transfusion-independence for at least 12 consecutive months with a weighted average hemoglobin (Hb) of at least 9 g/dL (TI12) in CLIMB-111 and CLIMB-131 combined (95% CI: 90.3%, 100%). The mean duration of transfusion independence was 40.5 months (range 13.6 to 70.8 months). The one evaluable patient who did not achieve TI12 has been transfusion free for 14.8 months. Iron removal therapy has been stopped for more than 6 months in 39/56 (69.6%) treated patients following infusion with CASGEVY, with sustained improvement in ferritin and liver iron content, suggesting that CASGEVY has the potential to correct ineffective erythropoiesis. Patients continue to demonstrate stable levels of fetal hemoglobin (HbF) and allelic editing. The safety profile of CASGEVY continues to be generally consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant. Progress in bringing CASGEVY to patients Through reimbursement agreements, Vertex has secured access for eligible SCD or TDT patients in multiple countries including Austria, Bahrain, England, Denmark, the Kingdom of Saudi Arabia, Northern Ireland, Scotland, the United Arab Emirates, the United States and Wales. In Canada, CASGEVY received positive recommendations for reimbursement from both Canadian health technology agencies between December 2024 and January 2025; however, a Letter of Engagement from the pan-Canadian Pharmaceutical Alliance (pCPA) is pending. Vertex is continuing to work with government and reimbursement authorities globally to secure sustainable access for additional eligible patients. 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. 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. About Pr CASGEVY ® (exagamglogene autotemcel) Pr CASGEVY ® is an autologous genome edited hematopoietic stem cell-based 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 vaso-occlusive crises (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. 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 in select regions 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 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 Kevin Kuo, M.D., in this press release, and statements regarding expectations for the anticipated durable clinical benefits of CASGEVY, expectations for the safety profile of CASGEVY, expectations for the Letter of Engagement from the pCPA, plans to continue working with government and reimbursement authorities globally to secure sustainable access for patients, 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 and available through the company's website at 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. Vertex Pharmaceuticals Incorporated Media: [email protected] orCanada: +1 647-790-1600orU.S.: 617-341-6992 SOURCE Vertex Pharmaceuticals (Canada) Inc.
Yahoo
24-06-2025
- Business
- Yahoo
CRISPR Therapeutics AG (CRSP): A Bull Case Theory
We came across a bullish thesis on CRISPR Therapeutics AG (CRSP) on Two Natural Cap's Substack. In this article, we will summarize the bulls' thesis on CRSP. CRISPR Therapeutics AG (CRSP)'s share was trading at $41.52 as of 11th June. CRSP's trailing and forward P/E were 22.55 and 23.36 respectively according to Yahoo Finance. A scientist peering into a microscope, researching the next gene therapy breakthrough. CRISPR has re-entered the spotlight thanks to a groundbreaking case where the technology was used to treat Baby KJ, born with a severe genetic liver disorder caused by a CPS1 enzyme deficiency. With only a 50% survival rate in infancy, traditional treatment included heavy medication and a strict low-protein diet. But researchers rapidly developed a personalized CRISPR therapy that KJ has now received three times, allowing him to eat a regular diet and significantly reduce medication use. This marks a major milestone not only for KJ but for the broader gene-editing landscape. At its core, CRISPR-Cas9 was originally a bacterial defense mechanism that evolved to recognize and cut viral DNA. Scientists have since repurposed this system, using modified guide RNAs and engineered Cas9 proteins to target human DNA with remarkable precision. In most therapeutic uses, CRISPR creates double-stranded breaks that are imperfectly repaired by the body—helpful for knocking out malfunctioning genes, as seen with CASGEVY, the FDA-approved therapy for sickle cell disease and beta thalassemia. KJ's case went further, employing 'base editing' to fix a single-letter DNA error using a deaminase-modified CRISPR complex that swapped an adenine for a guanine, correcting the mutation without a DNA break. This was delivered via lipid nanoparticles (LNPs), offering dosing flexibility over traditional viral vectors. The success not only validates the science behind base editing but also uplifts companies like Beam, CRISPR Therapeutics, and Intellia after years of market declines. It also spotlights Danaher's Aldevron and Acuitas Therapeutics, whose platforms were instrumental in the therapy's development, underscoring the growing viability of personalized gene medicine. We previously covered a bullish thesis on CRISPR Therapeutics (CRSP) from wallstreetbets by MADD-Scientis, emphasizing Casgevy's revenue potential, strong cash runway, and optionality in oncology and cardiology. Since the coverage, the stock price has appreciated by roughly 1.3%. Two Natural Cap's thesis adds depth by spotlighting CRSP's role in a groundbreaking base-editing therapy, reinforcing the platform's adaptability. Both cases point to CRSP's leadership in curative gene editing. CRISPR Therapeutics AG (CRSP) is not on our list of the 30 Most Popular Stocks Among Hedge Funds. As per our database, 29 hedge fund portfolios held CRSP at the end of the first quarter which was 27 in the previous quarter. While we acknowledge the risk and potential of CRSP as an investment, 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 extremely cheap AI stock that is also a major beneficiary of Trump tariffs and onshoring, see our free report on the best short-term AI stock. READ NEXT: 8 Best Wide Moat Stocks to Buy Now and 30 Most Important AI Stocks According to BlackRock. Disclosure: None. This article was originally published at Insider Monkey. 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
Al Etihad
19-06-2025
- Health
- Al Etihad
Department of Health - Abu Dhabi highlights sickle cell awareness efforts
19 June 2025 14:30 ABU DHABI (WAM)Executive Director of the Health Life Sciences Sector at the Department of Health – Abu Dhabi (DoH), Dr. Asma Al Mannaei, affirmed that World Sickle Cell Day, marked annually on 19th June, is a vital occasion to raise awareness about the disease, support patients and their families, and shed light on efforts aimed at reducing its health and social Al Mannaei told WAM that Sickle cell disease remains a significant public health concern globally, affecting the quality of life for patients and placing long-term pressure on families. "At the Department of Health – Abu Dhabi, we are committed to reshaping that reality through innovation, early detection and personalised care. Our mission is to foster a healthy population, deliver best-in-class care, and drive continuous resilience and innovation."Dr. Al Mannaei added, "Supported by powerful Artificial Intelligence tools, driven by data and genomic sciences, we are building a system that predicts, prevents and acts.""Our efforts begin with prevention. Through the Premarital Genetic Screening Programme, we empower couples to make informed decisions that help reduce the prevalence of genetic conditions such as sickle cell in future generations."She continued, 'We then move to prediction, where we are shifting from a reactive approach to proactive and personalised care approach for sickle cell, by utilising platforms such as the Emirati Genome Programme. By integrating sickle cell disease in the newly launched Newborn Genetic Screening Programme, we can ensure early detection, timely intervention and healthier outcomes for families across the Emirate.'Dr. Al Mannaei also praised the introduction of innovative treatments. In collaboration with the Abu Dhabi Stem Cells Centre and global partner Vertex Pharmaceuticals, the Department has introduced CASGEVY, a CRISPR/Cas9-based gene-editing therapy for patients with sickle cell disease and transfusion-dependent beta-thalassaemia."This marks the first time this cutting-edge treatment is available in the UAE," she added that, beyond treatment, investment in scientific research remains a top priority. In partnership with the Authority of Social Contribution – Ma'an, and through the Research and Innovation Fund, the Department is supporting research that addresses key healthcare priorities. One of the grant awardees received funding for research focused on anaemia and sickle cell disease, examining their impact on healthspan.
Associated Press
09-05-2025
- Business
- Associated Press
Biomay Launches FDA-Grade CRISPR/Cas9 Nuclease for Off-the-Shelf Purchase
Biomay, a leading manufacturer of recombinant proteins, today announced the commercial availability of its CRISPR/Cas9 nuclease, marking a significant addition to its off-the-shelf product portfolio for genome-editing applications. Clients purchasing Biomay´s Cas9 will benefit from the company´s unparalleled track record and expertise as a market-registered GMP-manufacturer of the nuclease. Biomay is the FDA-approved manufacturer of recombinant Cas9 as the essential component of CASGEVY®, the very first CRISPR genome editing product on the market. Biomay's Cas9 (internal code 'BMC9') is based on the classical wild-type Cas9 nuclease fromStreptococcus pyogenes. The Cas9 manufacturing process has beende novodeveloped, GMP-implemented and PPQ-validated by Biomay. GMP and RUO manufacturing is performed by fermentation withE. coliand by purification with chromatographic methods. By quality control with a comprehensive set of validated analytical assays, the consistent integrity, purity and potency of the product is secured. 'The addition of Cas9 to Biomay's off-the-shelf portfolio aligns with our mission to provide high-quality and scalable solutions for emerging therapeutic modalities,'said Dr. Hans Huber, CEO of Biomay.'With this launch, we are expanding access to a critical component of gene-editing workflows, backed by our proven manufacturing expertise. Biomay´s off-the-shelf distributed Cas9, in combination with our made-to-order GMP services, will guarantee full scalability and GMP compliance throughout the whole product lifecycle. Biomay´s latest addition further strengthens the company's position as a key supplier in the field of gene and cell therapies. The CRISPR/Cas system, a transformative gene-editing technology, whose discovery was honored with the 2020 Nobel Prize in Chemistry, enables precise and efficient modification of genomic sequences. About Biomay: Biomay AG is a fully integrated Contract Development and Manufacturing Organization (CDMO) based in Vienna, Austria. Founded in 1984, the expression of recombinant proteins inE. colihas been Biomay's business focus yet from its beginning. Today, Biomay offers cGMP services for manufacturing of messenger RNA (mRNA), circular plasmid DNA, linear IVT-template DNA and therapeutic recombinant proteins. Biomay operates a dedicated mRNA Competence Center for cGMP manufacturing and QC testing of mRNA drug substance and drug product (clinical, commercial). The company's scope of services comprises process and analytical development, cell banking, R&D material supply, cGMP manufacturing, lipid nanoparticle / LNP formulation and aseptic filling. Biomay's facilities are inspected by the US FDA.
