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Globe and Mail
3 days ago
- Health
- Globe and Mail
Sickle Cell Disease Drugs Market 2034: Clinical Trials, Prevalence, EMA, PDMA, FDA Approvals, Medication, Statistics, NICE Approval.Revenue and Companies by DelveInsight
Sickle Cell Disease companies working in the treatment market are Cellectis, Sana Biotechnology, Global Blood Therapeutics, Inc., CSL Behring, Pfizer, Aruvant Sciences, Graphite Bio, Novartis, Agios Pharmaceuticals, Forma therapeutics, Vertex Pharmaceuticals, Global Blood Therapeutics, Inc., Alfasigma, Novo Nordisk, bluebird bio, ExCellThera, Gamida Cell, KM Biologics/Takeda, Editas Medicine, and others. Sickle Cell Disease Market Summary In 2023, the Sickle Cell Disease (SCD) market across the 6MM was valued at approximately USD 650 million, with the United States dominating at ~USD 603 million (92% share), followed by France and the UK. SCD is a hereditary blood disorder caused by mutations in the HBB gene, resulting in abnormal hemoglobin (HbS) that distorts red blood cells into a sickle shape. These rigid cells obstruct microvasculature, triggering vaso-occlusive crises, anemia, acute chest syndrome, stroke, organ damage, and heightened infection risk, severely impacting patient quality of life and survival. Historically, management relied on NSAIDs, transfusions, chelators, and supportive care, with hydroxyurea (DROXIA) being the primary disease-modifying agent for decades. Recent approvals of ENDARI (L-glutamine), ADAKVEO (crizanlizumab), and OXBRYTA (voxelotor) have expanded options, though challenges in adherence and reimbursement persist. New approvals like CASGEVY (exa-cel) and LYFGENIA (lovo-cel) are expected to drive market growth, alongside pipeline candidates such as EDIT-301, Mitapivat, Inclacumab, and Osivelotor. Despite advances, delays in diagnosis, economic burden, and complications continue to constrain outcomes. Multidisciplinary care, genetic counseling, early intervention, and patient education are critical, while gene therapy and gene editing hold promise for transformative treatment, aiming to mitigate SCD's substantial clinical and economic impact in the coming decade. (Albany, USA) DelveInsight's ' Sickle Cell Disease Market Insights, Epidemiology, and Market Forecast-2034 ' report delivers an in-depth understanding of Sickle Cell Disease, historical and forecasted epidemiology as well as the Sickle Cell Disease market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan. The Sickle Cell Disease market report provides current treatment practices, emerging drugs, the market share of the individual therapies, and the current and forecasted Sickle Cell Disease market size from 2020 to 2034, segmented by seven major markets. The Report also covers current Sickle Cell Disease treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying potential of the Sickle Cell Disease market. Some facts of the Sickle Cell Disease Market Report are: According to DelveInsight, Sickle Cell Disease market size is expected to grow at a decent CAGR by 2034. Leading Sickle Cell Disease companies working in the market are Cellectis, Sana Biotechnology, Global Blood Therapeutics, Inc., CSL Behring, Pfizer, Aruvant Sciences, Graphite Bio, Novartis, Agios Pharmaceuticals, Forma therapeutics, Vertex Pharmaceuticals, Global Blood Therapeutics, Inc., Alfasigma, Novo Nordisk, bluebird bio, ExCellThera, Gamida Cell, KM Biologics/Takeda, Editas Medicine, and others. Key Sickle Cell Disease Therapies expected to launch in the market are BPX-501 T cells, Canakinumab, EPI01, CTX001, ADAKVEO, DROXIA, ENDARI, OXBRYTA, and many others. In May 2025, Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company specializing in precision genetic medicines via base editing, announced it will share new findings from its BEACON Phase 1/2 clinical trial of BEAM-101 at the upcoming European Hematology Association 2025 Congress (EHA2025), scheduled for June 12–15, 2025, in Milan, Italy. BEAM-101 is an investigational ex vivo genetically modified cell therapy being developed to treat sickle cell disease (SCD), specifically in patients experiencing severe vaso-occlusive crises (VOCs). In November 2024, BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a commercial-stage biopharmaceutical company focused on oncology and rare diseases, announced that an abstract featuring initial results from a Phase 1 trial of motixafortide—both as a monotherapy and in combination with natalizumab for CD34+ hematopoietic stem cell (HSC) mobilization in gene therapies for sickle cell disease (SCD)—has been accepted for oral presentation at the 66th American Society of Hematology (ASH) Annual Meeting & Exposition, taking place December 7-10, 2024, in San Diego, California. Conducted in collaboration with Washington University School of Medicine in St. Louis, this proof-of-concept study aims to explore alternative HSC mobilization approaches to enhance the treatment experience for SCD patients undergoing gene therapy. In May 2024, Afimmune announced results of an Open-label Mechanistic Study to Assess the Pharmacokinetics, Pharmacodynamics and Safety of Orally Administered Epeleuton in Patients With Sickle Cell Disease In April 2024, Pfizer announced results of an Open-label Extension Study to Evaluate the Long-term Safety of GBT021601 Administered to Participants With Sickle Cell Disease Who Have Participated in a GBT021601 Clinical Trial In April 2024, Novo Nordisk A/S (Forma Therapeutics, Inc.) announced results of an adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients With Sickle Cell Disease (HIBISCUS). In July 2023, Novartis announced results of an Open-label, Multi-center, Phase IV, Rollover Study for Patients With Sickle Cell Disease Who Have Completed a Prior Novartis-Sponsored Crizanlizumab Study. Sickle Cell Disease Overview Sickle cell disease (SCD) is a hereditary blood disorder characterized by the production of abnormal hemoglobin, known as hemoglobin S. This defect causes red blood cells to assume a rigid, sickle-like shape, which impedes their ability to flow smoothly through blood vessels. Consequently, these misshapen cells can obstruct blood flow, leading to severe pain, organ damage, and an increased risk of infection. Sickle cell disease is most prevalent among individuals of African, Mediterranean, Middle Eastern, and Indian ancestry. Symptoms typically appear in early childhood and include episodes of pain (called sickle cell crises), chronic anemia, fatigue, swelling in the hands and feet, and delayed growth. Complications can be severe, including stroke, acute chest syndrome, and organ failure. Management of Sickle cell disease involves both preventive and therapeutic strategies. Preventive measures include regular vaccinations, antibiotics to prevent infections, and hydroxyurea, a medication that reduces the frequency of pain crises and the need for blood transfusions. Pain management, blood transfusions, and bone marrow transplants are critical therapeutic options for managing acute and chronic complications. Recent advancements in gene therapy and CRISPR technology hold promise for more effective treatments and potential cures for Sickle cell disease. Ongoing research and clinical trials aim to improve the quality of life for patients and reduce the burden of this debilitating disease. Do you know what will be the Sickle Cell Disease market share in 7MM by 2034 @ Sickle Cell Disease Treatment Market Sickle Cell Disease Market The Sickle Cell Disease market outlook of the report helps to build a detailed comprehension of the historical, current, and forecasted Sickle Cell Disease market trends by analyzing the impact of current Sickle Cell Disease therapies on the market and unmet needs, and drivers, barriers, and demand for better technology. This segment gives a thorough detail of the Sickle Cell Disease market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated Sickle Cell Disease market data are presented with relevant tables and graphs to give a clear view of the market at first sight. According to DelveInsight, the Sickle Cell Disease market in 7MM is expected to witness a major change in the study period 2020-2034. Sickle Cell Disease Epidemiology The Sickle Cell Disease epidemiology section provides insights into the historical and current Sickle Cell Disease patient pool and forecasted trends for seven individual major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the Sickle Cell Disease market report also provides the diagnosed patient pool, trends, and assumptions. Interested to know how the emerging diagnostic approaches will be contributing in increased Sickle Cell Disease diagnosed prevalence pool? Download report @ Sickle Cell Disease Market Dynamics and Trends Sickle Cell Disease Drugs Uptake This section focuses on the uptake rate of the potential Sickle Cell Disease drugs recently launched in the Sickle Cell Disease market or expected to be launched in 2020-2034. The analysis covers the Sickle Cell Disease market uptake by drugs, patient uptake by therapies, and sales of each drug. Sickle Cell Disease Drugs Uptake helps in understanding the drugs with the most rapid uptake and the reasons behind the maximal use of new drugs and allows the comparison of the drugs based on Sickle Cell Disease market share and size, which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions. Sickle Cell Disease Pipeline Development Activities The Sickle Cell Disease report provides insights into different therapeutic candidates in Phase II, and Phase III stages. It also analyses Sickle Cell Disease key players involved in developing targeted therapeutics. Download report to know which TOP 3 therapies will be capturing the largest Sickle Cell Disease market share by 2034? Click here @ Sickle Cell Disease Companies and Medication Sickle Cell Disease Therapeutics Assessment Major key companies are working proactively in the Sickle Cell Disease Therapeutics market to develop novel therapies which will drive the Sickle Cell Disease treatment markets in the upcoming years are Cellectis, Sana Biotechnology, Global Blood Therapeutics, Inc., CSL Behring, Pfizer, Aruvant Sciences, Graphite Bio, Novartis, Agios Pharmaceuticals, Forma therapeutics, Vertex Pharmaceuticals, Global Blood Therapeutics, Inc., Alfasigma, Novo Nordisk, bluebird bio, ExCellThera, Gamida Cell, KM Biologics/Takeda, Editas Medicine, and others. Do you know how market launches of New drugs will be impacting the Sickle Cell Disease market CAGR? Download sample report @ Sickle Cell Disease Therapies and Clinical Trials Sickle Cell Disease Report Key Insights 1. Sickle Cell Disease Patient Population 2. Sickle Cell Disease Market Size and Trends 3. Key Cross Competition in the Sickle Cell Disease Market 4. Sickle Cell Disease Market Dynamics (Key Drivers and Barriers) 5. Sickle Cell Disease Market Opportunities 6. Sickle Cell Disease Therapeutic Approaches 7. Sickle Cell Disease Pipeline Analysis 8. Sickle Cell Disease Current Treatment Practices/Algorithm 9. Impact of Emerging Therapies on the Sickle Cell Disease Market Table of Contents 1. Key Insights 2. Executive Summary 3. Sickle Cell Disease Competitive Intelligence Analysis 4. Sickle Cell Disease Market Overview at a Glance 5. Sickle Cell Disease Disease Background and Overview 6. Sickle Cell Disease Patient Journey 7. Sickle Cell Disease Epidemiology and Patient Population 8. Sickle Cell Disease Treatment Algorithm, Current Treatment, and Medical Practices 9. Sickle Cell Disease Unmet Needs 10. Key Endpoints of Sickle Cell Disease Treatment 11. Sickle Cell Disease Marketed Products 12. Sickle Cell Disease Emerging Therapies 13. Sickle Cell Disease Seven Major Market Analysis 14. Attribute Analysis 15. Sickle Cell Disease Market Outlook (7 major markets) 16. Sickle Cell Disease Access and Reimbursement Overview 17. KOL Views on the Sickle Cell Disease Market 18. Sickle Cell Disease Market Drivers 19. Sickle Cell Disease Market Barriers 20. Appendix 21. DelveInsight Capabilities 22. Disclaimer About DelveInsight DelveInsight is a leading Life Science market research and business consulting company recognized for its off-the-shelf syndicated market research reports and customized solutions to firms in the healthcare sector. Media Contact Company Name: DelveInsight Business Research LLP Contact Person: Ankit Nigam Email: Send Email Phone: +14699457679 Address: 304 S. Jones Blvd #2432 City: Albany State: New York Country: United States Website:
Yahoo
21-05-2025
- Business
- Yahoo
Cellectis' Annual Shareholders General Meeting to be Held on June 26, 2025
NEW YORK, May 21, 2025 (GLOBE NEWSWIRE) -- Cellectis (the 'Company') (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that it will hold its annual general meeting on June 26, 2025 at 2:30 p.m. CET at the Biopark auditorium, 11 rue Watt, 4th floor, 75013 Paris, France. The notice convening the annual general meeting stating the detailed agenda and modalities of participation in the meeting and the report of the board of directors to the shareholders meeting are available on the Cellectis website: About Cellectis Cellectis is a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies. The company utilizes an allogeneic approach for CAR T immunotherapies in oncology, pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients, and a platform to develop gene therapies in other therapeutic indications. With its in-house manufacturing capabilities, Cellectis is one of the few end-to-end gene editing companies that controls the cell and gene therapy value chain from start to finish. Cellectis' headquarters are in Paris, France, with locations in New York and Raleigh, NC. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS). To find out more, visit and follow Cellectis on LinkedIn and X. For further information on Cellectis, please contact: Media contacts: Pascalyne Wilson, Director, Communications, + 33 (0)7 76 99 14 33, media@ Patricia Sosa Navarro, Chief of Staff to the CEO, +33 (0)7 76 77 46 93 Investor Relations contact: Arthur Stril, Chief Financial Officer & Chief Business Officer, investors@ 20240521_AGM 2025 announcement_ENGLISHSign in to access your portfolio
Yahoo
06-05-2025
- Business
- Yahoo
Cellectis to Report First Quarter Financial Results on May 12, 2025
Cellectis Inc. NEW YORK, May 06, 2025 (GLOBE NEWSWIRE) -- Cellectis (the 'Company') (Euronext Growth: ALCLS- NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it will report financial results for the first quarter 2025 ending March 31, 2025 on Monday, May 12, 2025 after the close of the US market. The press release will be available in the Investors section of Cellectis' website: Cellectis will not host a conference call to discuss these results. Our investors relations team remains available for questions at investors@ About Cellectis Cellectis is a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies. The company utilizes an allogeneic approach for CAR T immunotherapies in oncology, pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients, and a platform to develop gene therapies in other therapeutic indications. With its in-house manufacturing capabilities, Cellectis is one of the few end-to-end gene editing companies that controls the cell and gene therapy value chain from start to finish. Cellectis' headquarters are in Paris, France, with locations in New York and Raleigh, NC. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS). To find out more, visit and follow Cellectis on LinkedIn and X. For further information on Cellectis, please contact: Media contacts: Pascalyne Wilson, Director, Communications, +33 (0)7 76 99 14 33, media@ Patricia Sosa Navarro, Chief of Staff to the CEO, +33 (0)7 76 77 46 93 Investor Relations contact: Arthur Stril, Chief Financial Officer & Chief Business Officer, investors@ Attachment
Yahoo
28-04-2025
- Business
- Yahoo
Cellectis Presents Non-Viral Gene Editing and Base Editing Innovation at the ASGCT Annual Meeting
NEW YORK, April 28, 2025 (GLOBE NEWSWIRE) -- Cellectis (the 'Company') (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today unveils research data on TALEN®-mediated non-viral transgene insertion for advancing cellular and gene therapies, and advancements in genetic editing using TALE base editors (TALEB), at the Society of Gene and Cell Therapy (ASGCT) annual meeting, that will be held on May 13-17, 2025 in New Orleans. The data are presented in two posters: Title: TALEN®- Mediated non-viral Transgene Insertion for the Advancement of Cellular and Gene Therapies. Cell and gene therapy approaches can use gene-editing tools and introduce transgenes to modify disease-associated genes, thus providing a potential therapeutic solution for a wide array of diseases. In this work, Cellectis combines TALEN®-mediated gene editing with non-viral delivery of transgene for advancing cellular and gene therapies, and explores gene insertion-efficacy and cellular health using single-stranded DNA (ssDNA) for payload delivery in different cell types. This innovative approach has the potential to address the challenges associated with traditional lentiviral viral methods or AAV-mediated transgene insertion such as manufacturing constraints, potential genomic toxicities or limited payload size. Research data show: Non-viral methods for gene editing: TALEN® mediated gene editing combined with non-viral templates (linear and circular ssDNA) can be used for highly efficient gene insertion in T-cells as well as hematopoietic stem and progenitor cells (HSPCs) promoting viability and insertion specificity. Advantages of Circular ssDNA over viral vectors: Transcriptomic analysis and in vivo data demonstrate that CssDNA-mediated cell engineering allows better maintenance of HSPC fitness as well as more stable gene editing, compared to traditional viral donor template-mediated transgene delivery. « The implementation of these gene-editing techniques holds significant potential for the development of next-generation therapies, aiming to provide alternative efficient, and safe therapeutic options for patients with cancer, autoimmune diseases, monogenic disorders, and various other conditions. » said Beatriz Aranda Orgilles, Ph.D., Associate Director – IO and business development analyst at Cellectis. Title: High fidelity C-to-T editing with TALE base editors TALE base editors (TALEB) are fusions of a transcription activator-like effector domain (TALE), split-DddA deaminase halves, and a uracil glycosylase inhibitor (UGI). The C-to-T class of TALEB edits double-stranded DNA by converting a cytosine (C) to a thymine (T) and does not involve DNA strand nick. Cellectis has developed a method to characterize the efficiency of this conversion and examined various factors influencing TALEB activity. This method also takes advantage of a highly precise and efficient knock-in of ssODN in primary T cells to develop an assay to assess how the composition and spacer variations of target sequences affect TALEB activity/efficiency. Research data show: Efficiency of C-to-T editing: TALEB enables efficient conversion of C to T. Variations in target sequences and surrounding bases affecting editing efficiency. An educated choice of the TALEB architecture further allows to control the editing outcome. Assessment of off-target editing risks: Studies have been conducted to evaluate off-target editing, showing no detectable editing in primary cells at previously described sites, highlighting the specificity of TALEB for potential therapeutic applications. «It is inspiring to see the advancement of Cellectis' TALE technology into a new tool that is available in our gene editing toolbox. Our ability to understand and fine-tune the editing capacity of TALE base editors has equipped us with another efficient and specific approach that can be used to support novel gene editing and gene therapy applications. » said Louisa Mayer, Ph.D., Scientist II and Supervisor – Innovation & Gene Editing at Cellectis. Overall, the results of this study enhance the control and use of TALEB, allowing for the design of highly efficient and specific TALEB compatible with future therapeutic applications. The abstracts are live on the ASGCT website. The posters will be available on Cellectis' website the first day of the event. About Cellectis Cellectis is a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies. The company utilizes an allogeneic approach for CAR T immunotherapies in oncology, pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients, and a platform to develop gene therapies in other therapeutic indications. With its in-house manufacturing capabilities, Cellectis is one of the few end-to-end gene editing companies that controls the cell and gene therapy value chain from start to finish. Cellectis' headquarters are in Paris, France, with locations in New York and Raleigh, NC. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS). To find out more, visit and follow Cellectis on LinkedIn and X. TALEN® is a registered trademark owned by Cellectis. Cautionary Statement This press release contains 'forward-looking' statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by words such as' can,' 'potential,' 'has the potential to,' 'aiming to,' and 'will' or the negative of these and similar expressions. These forward-looking statements are based on our management's current expectations and assumptions and on information currently available to management. Forward-looking statements include statements about the potential of our innovation programs, in particular our gene editing innovations. These forward-looking statements are made in light of information currently available to us and are subject to numerous risks and uncertainties, including those described in our Annual Report on Form 20-F as amended and in our annual financial report (including the management report) for the year ended December 31, 2024 and subsequent filings Cellectis makes with the Securities Exchange Commission from time to time, which are available on the SEC's website at as well as other known and unknown risks and uncertainties may adversely affect such forward-looking statements and cause our actual results, performance or achievements to be materially different from those expressed or implied by the forward-looking statements. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. For further information on Cellectis, please contact: Media contacts: Pascalyne Wilson, Director, Communications, + 33 (0)7 76 99 14 33, media@ Patricia Sosa Navarro, Chief of Staff to the CEO, +33 (0)7 76 77 46 93 Investor Relations contact: Arthur Stril, Chief Financial Officer & Chief Business Officer, investors@ Attachment ASGCT posters announcement PR-ENGLISH(1)Sign in to access your portfolio
Yahoo
07-03-2025
- Business
- Yahoo
Cellectis to Report Fourth Quarter and Full Year 2024 Financial Results on March 13, 2025
NEW YORK, March 07, 2025 (GLOBE NEWSWIRE) -- Cellectis (the 'Company') (Euronext Growth: ALCLS- NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it will report financial results for the fourth quarter and full year 2024 ending December 31, 2024 on Thursday, March 13, 2025 after the close of the US market. The publication will be followed by an investor conference call and webcast on Friday, March 14, 2025, at 8:00 AM ET / 1:00 PM CET. The call will include the Company's fourth quarter and full year 2024 results and an update on business activities. Details for the call are as follows: Dial in information: Domestic: +1-800-225-9448 International: +1-203-518-9708 Conference ID: CLLS24 Webcast Link: About Cellectis Cellectis is a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies. The company utilizes an allogeneic approach for CAR T immunotherapies in oncology, pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients, and a platform to develop gene therapies in other therapeutic indications. With its in-house manufacturing capabilities, Cellectis is one of the few end-to-end gene editing companies that controls the cell and gene therapy value chain from start to finish. Cellectis' headquarters are in Paris, France, with locations in New York and Raleigh, NC. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS). To find out more, visit and follow Cellectis on LinkedIn and X. TALEN® is a registered trademark owned by Cellectis. For further information on Cellectis, please contact: Media contacts: Pascalyne Wilson, Director, Communications, +33 (0)7 76 99 14 33, media@ Patricia Sosa Navarro, Chief of Staff to the CEO, +33 (0)7 76 77 46 93 Investor Relations contact: Arthur Stril, Chief Financial Officer & Chief Business Officer, investors@ Attachment 20250307_FY 2024 earnings call announcement_ENGLISHSign in to access your portfolio
