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Decode. Detect. Defeat: United for Sickle Cell Elimination by 2047
Decode. Detect. Defeat: United for Sickle Cell Elimination by 2047

Hindustan Times

time7 days ago

  • Health
  • Hindustan Times

Decode. Detect. Defeat: United for Sickle Cell Elimination by 2047

New Delhi: The National Sickle Cell Summit 2025 brought together a powerful cross-section of voices, including survivors, healthcare providers, scientists, patient advocacy groups, and senior policymakers, with one common goal: to eliminate Sickle Cell Disease (SCD) as a national public health concern by 2047. Organised by and Health Shots, in collaboration with the National Alliance of Sickle Cell Organisations and Novo Nordisk India, the event closely aligned with the goals of the National Sickle Cell Anaemia Elimination Mission, launched under the leadership of Prime Minister Narendra Modi ( National Sickle Cell Summit 2025 Left to right: Mr. Vivek Virendra Singh – Chief, Health (a.i.), UNICEF; Abhishek Singh – Deputy Editor, LiveMint; Shri Jual Oram – Hon'ble Minister of Tribal Affairs; Mr. Gautam Dongre – General Secretary, NASCO; Mr. Vikrant Shrotriya – Managing Director, Novo Nordisk India; Radhika Bhirani – Editor, The summit brought together public health leaders, policymakers, and patient advocates to accelerate India's mission to eliminate Sickle Cell Disease by 2047. While the summit's agenda focused on early detection, treatment access, and community engagement, what truly stood out were the voices of those who live with the condition every day. Their stories, layered with resilience and hope, became the emotional anchor around which discussions of policy and reform were built. Tackling a Generational Crisis Setting the tone for the summit, Smt. Anupriya Patel, Hon'ble Minister of State for Health and Family Welfare, acknowledged the scale and depth of the challenge. 'Sickle Cell Disease is not just a clinical burden; it's a social, economic, and generational burden,' she said. Her keynote traced the progress of the National Elimination Mission since its 2023 launch. By 2025, over 5.8 crore individuals had been screened, 2.54 crore had received status cards, and more than two lakh individuals were confirmed with SCD. She noted that the programme was not only about screening but also about providing access to care, counselling, and de-stigmatising the disease. This context helped ground discussions for the rest of the day, where each voice was built on the urgency she underscored. - last accessed on 23 July 2025 When Lived Experience Leads the Conversation The importance of patient advocacy was brought to life in the panel 'From Pain to Possibility', moderated by Vartika Tolani of Live Hindustan. It was here that Mr. Gautam Dongre, General Secretary of NASCO, shared his journey as a parent of two children with SCD. 'I have two children with Sickle Cell Disease. Our financial and social life collapsed. Doctors gave up. But we refused to,' he said. His experiences led to the creation of NASCO, a patient-led network working to influence policy through lived experience. Mr. Dongre highlighted that access to Hydroxyurea, a key drug in SCD management, must be made available even at the most remote sub-centres. He also challenged the misconception that SCD only affects tribal groups, clarifying that while the prevalence may be higher in tribal groups, it affects non-tribals as well. Additionally, Dongre raised concerns about the gendered stigma surrounding the disease, particularly the blame placed on women in families where children are diagnosed. He stressed the need for widespread genetic counselling and awareness campaigns that reach into every social segment. Press Note Details: Press Information Bureau – last accessed on 23 July 2025 Shaping Systems Around People Public health leadership was another strong theme throughout the day. Mr. Vivek Virendra Singh, Chief, Health (a.i.), UNICEF India, spoke about the importance of redesigning systems to support people, not merely diagnose them. 'You either live with the disease or suffer from it. Our job is to make sure systems help people live,' he said. He proposed a multi-ministerial approach involving Health, Tribal Affairs, Women and Child Development, and Education, to ensure that screening is followed by structured care pathways. Singh also highlighted the need for culturally sensitive messaging and the integration of survivor voices in the design of public health communication. He emphasised that chronic care for SCD should be normalised the same way it is for conditions like diabetes, with frontline workers empowered and trained to deliver compassionate care. Voices That Moved the Room Survivor testimonies provided some of the most impactful moments of the summit. Mr. Lalit Kishor Pargi, a Sickle Cell Warrior from Rajasthan, shared his late diagnosis at the age of 16 and how hospitalisation in 2021 led him to take up awareness work in tribal communities. 'I was diagnosed at 16, after years of pain and hospital visits. But from my hospital bed in 2021, I decided no one should suffer silently like I did,' he said. In the same panel, Ms. Swati Panika, a survivor from Madhya Pradesh, highlighted the daily obstacles faced by those living with SCD, from navigating education with frequent absences to dealing with stigma and lack of workplace support. 'Managing Sickle Cell with school and hospital visits was exhausting. But I chose to be a warrior, not a victim,' she said. Her advocacy underscored the need for job security, flexible attendance policies in schools, and wider access to genetic counselling. Together, their stories grounded the summit in lived reality, reminding every stakeholder that policy must begin with people. Bridging Policy and Implementation State-level leaders brought attention to what implementation looks like on the ground. In the panel 'From Mission to Movement', moderated by Abhishek Singh, health officials from Jharkhand, Rajasthan, and Maharashtra described how mobile vans and community-level health camps are being used for large-scale screening. The Screening and Early Detection (SED) portal, a digital tool for tracking screenings and follow-ups, was recognised as a critical link between data collection and ongoing care. Chhattisgarh was acknowledged for leading in screening coverage. At the same time, Odisha emerged as the state with the highest number of diagnosed cases — a statistic that reflects both proactive engagement and the deeper prevalence of the condition. These case studies demonstrated that the national mission is no longer confined to policy documents; it is being adapted and implemented in villages, towns, and health sub-centres. Aligning Innovation with Inclusion A dedicated panel on 'Innovation in Action' brought together researchers and public health experts focused on making medical advances more inclusive. In a fireside chat, Mr. Vikrant Shrotriya, Managing Director of Novo Nordisk India, discussed the private sector's role in supporting sustainable change. 'Innovation must be grounded in inclusion. We need to scale what works — not in labs, but in lives,' he said. He emphasized that in conditions like Sickle Cell Disease (SCD), trust is as critical as treatment. 'Our strategy is simple: listen first, then act. Whether it's through wellness hubs, frontline training, or partnerships with state systems, our goal is to co-create care with communities.' Highlighting Novo Nordisk's latest pipeline breakthroughs, Mr. Shrotriya also stressed that innovation must go beyond symptom management: 'The goal is not just to manage SCD, but to truly transform outcomes. Families living with this disease have waited long enough for meaningful solutions.' On the research front, scientists such as Dr. Manisha Madkaikar, Dr. Giriraj Chandak, and Prof. Soumendra Mohan Patnaik called for more genomic studies tailored to Indian populations, especially tribal communities. Their collective appeal was to design clinical interventions that are socially accepted, economically viable, and linguistically accessible. This shift—from lab-centred innovation to patient-centred delivery—emerged as one of the summit's most powerful thematic takeaways. Reframing Health as Justice Bringing a policy perspective grounded in social justice, Shri Jual Oram, Hon'ble Minister of Tribal Affairs, made an impassioned case for aligning health delivery with tribal empowerment. 'For tribal India, this mission is not just medical — it's justice,' he said. Oram stressed the importance of tribal leadership in eliminating stigma and ensuring that diagnosis is accompanied by dignity. His comments reframed the Sickle Cell Mission as not merely a medical programme, but a vehicle for equity and rights — one that must recognise cultural nuances, challenge misinformation, and place affected communities at the centre of the conversation. This call for equity echoed across the summit and became a unifying thread from science to policy. Conclusion: Beyond the Summit In his closing remarks, LiveMint Deputy Editor Abhishek Singh summed it up simply: 'We didn't just listen to experts. We heard from survivors.' The National Sickle Cell Summit 2025 was more than an event; it was a call to act. Real change begins when lived experiences shape every decision, from rural clinics to national policy. India's journey to eliminate Sickle Cell Disease by 2047 must be built not just on data, but on dignity, dialogue, and shared responsibility, ensuring no one is left behind. Note to the Reader: This article has been produced on behalf of the brand by HT Brand Studio and does not have journalistic/editorial involvement of Hindustan Times. Promomats number: IN25BRO00011

National mission uncovers 18.85 lakh sickle cell cases
National mission uncovers 18.85 lakh sickle cell cases

United News of India

time22-07-2025

  • Health
  • United News of India

National mission uncovers 18.85 lakh sickle cell cases

New Delhi, July 22 (UNI) The ongoing ambitious National Sickle Cell Anemia Elimination Mission has revealed a significant public health challenge, with more than 18.85 lakh individuals identified as either suffering from Sickle Cell Disease (SCD) or carrying the trait, according to the data shared by the Union Health Ministry today. As of now, six crore individuals have been screened against a target of seven crore. Sickle cell disease — also called sickle cell anemia — is a group of inherited disorders that affect hemoglobin, the major protein that carries oxygen in red blood cells. Of those screened, 2.15 lakh individuals have been diagnosed with the disease, and 16.7 lakh have been identified as carriers. Health experts have flagged the numbers as concerning, especially given the chronic nature of the illness and the burden it places on affected tribal populations. A total of 2.6 crore health cards have been distributed by states to facilitate continued monitoring and support, as per the statement by the Ministry. Madhya Pradesh, Gujarat, Rajasthan, Telangana, Karnataka, and Uttarakhand have reported remarkable progress in meeting their screening targets. However, the highest number of diagnosed cases has emerged from Odisha, Chhattisgarh, Madhya Pradesh, Maharashtra, and Gujarat — indicating high disease prevalence in these regions. Screening is being carried out using validated Point-of-Care Testing (POCT) kits that allow for rapid and confirmatory diagnosis. A centralised digital dashboard and SCD portal have been established to consolidate data and improve coordination among states, says the statement. The Mission, inaugurated on July 1, 2023, by Prime Minister Narendra Modi in Shahdol, Madhya Pradesh, seeks to eliminate sickle cell anemia by 2047. It envisions universal screening of seven crore individuals aged 0–40 years in tribal and high-burden districts by FY 2025–26, supported by robust awareness campaigns and counselling services. Health authorities have underscored the need for intensified outreach, sustained counselling, and follow-up mechanisms for those diagnosed. The large number of affected individuals now identified underscores both the scale of the challenge and the urgency of addressing it through integrated national and state-level health systems. UNI AJ SSP

Sanofi, CSL Behring, ITB-Med LLC, Novo Nordisk A/S, Fortrea, Inc., Novartis, Afimmune, Pfizer, ADDMEDICA SASA
Sanofi, CSL Behring, ITB-Med LLC, Novo Nordisk A/S, Fortrea, Inc., Novartis, Afimmune, Pfizer, ADDMEDICA SASA

Globe and Mail

time21-07-2025

  • Health
  • Globe and Mail

Sanofi, CSL Behring, ITB-Med LLC, Novo Nordisk A/S, Fortrea, Inc., Novartis, Afimmune, Pfizer, ADDMEDICA SASA

The Key Sickle Cell Disease Companies in the market include - Vertex Pharmaceuticals/CRISPR Therapeutics, Pfizer, Glycomimetics, Sanofi, CSL Behring, ITB-Med LLC, Novo Nordisk A/S, Fortrea, Inc., Novartis, Afimmune, Pfizer, ADDMEDICA SASA, CRISPR Therapeutics/Vertex Pharmaceuticals, Agios Pharmaceuticals, Alexion Pharmaceuticals, Chugai Pharmaceutical/Roche, Editas Medicine, Sangamo Therapeutics, Beam Therapeutics, CSL Behring, and others. DelveInsight's 'Sickle Cell Disease Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Sickle Cell Disease, historical and forecasted epidemiology as well as the Sickle Cell Disease market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom. Some of the key facts of the Sickle Cell Disease Market Report: Among the 6MM countries the Sickle Cell Disease market size was valued ~USD 650 million in 2023 and is anticipated to grow with a significant CAGR during the study period (2020-2034) 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 June 2024, Vertex Pharmaceuticals shared long-term results for its gene therapy, Casgevy (exa-cel), in patients with sickle cell disease or transfusion-dependent beta-thalassemia (TDT). In 2023, the United States held the largest market share for Sickle Cell Disease among the 6MM, with approximately USD 603 million, followed by France and the UK. At present, treatments for Sickle Cell Disease include NSAIDs, blood transfusions, chelating agents, nutritional supplements, and broad-spectrum antibiotics. Additionally, the US FDA has approved several therapies for this condition, such as DROXIA (hydroxyurea), ENDARI (L-glutamine oral powder), ADAKVEO (crizanlizumab-tmca), and OXBRYTA (voxelotor). In 2023, the United States had the largest total number of prevalent Sickle Cell Disease cases among the 6MM. In the US, Sickle Cell Anemia (hemoglobin S/S or hemoglobin S/β0-thalassemia) was the most common subtype of Sickle Cell Disease among the type-specific prevalent cases in 2023. In the EU4 and the UK, France had the highest prevalence of Sickle Cell Disease cases, followed by the UK, while Spain reported the lowest number of cases. Key Sickle Cell Disease Companies: Vertex Pharmaceuticals/CRISPR Therapeutics, Pfizer, Glycomimetics, Sanofi, CSL Behring, ITB-Med LLC, Novo Nordisk A/S, Fortrea, Inc., Novartis, Afimmune, Pfizer, ADDMEDICA SASA, CRISPR Therapeutics/Vertex Pharmaceuticals, Agios Pharmaceuticals, Alexion Pharmaceuticals, Chugai Pharmaceutical/Roche, Editas Medicine, Sangamo Therapeutics, Beam Therapeutics, CSL Behring, and others Key Sickle Cell Disease Therapies: CASGEVY (exagamglogene autotemcel), Inclacumab, GMI-1687, BIVV003, CSL889, Siplizumab, Etavopivat Tablets, VIT-2763, Crizanlizumab, Epeleuton, Voxelotor, Hydroxycarbamide, Exagamglogene autotemcel, Mitapivat, Canakinumab, ALXN1820, Crovalimab, EDIT 301, BIVV003, BEAM101, Hemopexin, and others The Sickle Cell Disease epidemiology based on gender analyzed that Sickle Cell Disease affects males and females equally The Sickle Cell Disease market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Sickle Cell Disease pipeline products will significantly revolutionize the Sickle Cell Disease market dynamics. Sickle Cell Disease Overview Sickle cell disease (SCD) comprises inherited red blood cell disorders resulting from an abnormality in the structure of hemoglobin known as sickle hemoglobin (HbS), which encodes the beta hemoglobin subunit. Sickle Cell Disease Epidemiology The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends. Sickle Cell Disease Epidemiology Segmentation: The Sickle Cell Disease market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Sickle Cell Disease Drugs Uptake and Pipeline Development Activities The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Sickle Cell Disease market or expected to get launched during the study period. The analysis covers Sickle Cell Disease market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share. The report also covers the Sickle Cell Disease Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies. Sickle Cell Disease Therapies and Key Companies Discover more about therapies set to grab major Sickle Cell Disease market share @ Sickle Cell Disease Treatment Landscape Scope of the Sickle Cell Disease Market Report Study Period: 2020–2034 Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom)] Key Sickle Cell Disease Companies: Vertex Pharmaceuticals/CRISPR Therapeutics, Pfizer, Glycomimetics, Sanofi, CSL Behring, ITB-Med LLC, Novo Nordisk A/S, Fortrea, Inc., Novartis, Afimmune, Pfizer, ADDMEDICA SASA, CRISPR Therapeutics/Vertex Pharmaceuticals, Agios Pharmaceuticals, Alexion Pharmaceuticals, Chugai Pharmaceutical/Roche, Editas Medicine, Sangamo Therapeutics, Beam Therapeutics, CSL Behring, and others Key Sickle Cell Disease Therapies: CASGEVY (exagamglogene autotemcel), Inclacumab, GMI-1687, BIVV003, CSL889, Siplizumab, Etavopivat Tablets, VIT-2763, Crizanlizumab, Epeleuton, Voxelotor, Hydroxycarbamide, Exagamglogene autotemcel, Mitapivat, Canakinumab, ALXN1820, Crovalimab, EDIT 301, BIVV003, BEAM101, Hemopexin, and others Sickle Cell Disease Therapeutic Assessment: Sickle Cell Disease current marketed and Sickle Cell Disease emerging therapies Sickle Cell Disease Market Dynamics: Sickle Cell Disease market drivers and Sickle Cell Disease market barriers Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies Table of Contents 1. Sickle Cell Disease Market Report Introduction 2. Executive Summary for Sickle Cell Disease 3. SWOT analysis of Sickle Cell Disease 4. Sickle Cell Disease Patient Share (%) Overview at a Glance 5. Sickle Cell Disease Market Overview at a Glance 6. Sickle Cell Disease Disease Background and Overview 7. Sickle Cell Disease Epidemiology and Patient Population 8. Country-Specific Patient Population of Sickle Cell Disease 9. Sickle Cell Disease Current Treatment and Medical Practices 10. Sickle Cell Disease Unmet Needs 11. Sickle Cell Disease Emerging Therapies 12. Sickle Cell Disease Market Outlook 13. Country-Wise Sickle Cell Disease Market Analysis (2020–2034) 14. Sickle Cell Disease Market Access and Reimbursement of Therapies 15. Sickle Cell Disease Market Drivers 16. Sickle Cell Disease Market Barriers 17. Sickle Cell Disease Appendix 18. Sickle Cell Disease Report Methodology 19. DelveInsight Capabilities 20. Disclaimer 21. About DelveInsight About DelveInsight DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach. Media Contact Company Name: DelveInsight Contact Person: Gaurav Bora Email: Send Email Phone: +14699457679 Address: 304 S. Jones Blvd #2432 City: Albany State: New York Country: United States Website:

New sickle cell anaemia screening test kit to cost less than Rs 100, to be launched soon: DG-CSIR
New sickle cell anaemia screening test kit to cost less than Rs 100, to be launched soon: DG-CSIR

Indian Express

time18-07-2025

  • Health
  • Indian Express

New sickle cell anaemia screening test kit to cost less than Rs 100, to be launched soon: DG-CSIR

A test kit to screen sickle cell anaemia – a hereditary blood disorder – developed by the Council of Scientific and Industrial Research will cost less than Rs 100, will be launching in the next six months, said Dr N Kalaiselvi, Director General , Council of Scientific and Industrial Research in Pune on Friday. 'This affordable test kit uses one drop of blood collected from the patient, undergoes the polymerase chain reaction (PCR) and results are available in half an hour. The test will identify whether the person is a patient, a carrier or at risk and we are hoping to bring it to the market in the next six months,' the CSIR chief said on the sidelines of the second edition of the National Scientists Round Table Conference (NSRTC) 2025. The three-day event,(July 18 to 20) was inaugurated today at MIT World Peace University. Sickle Cell Disease is caused by a mutation in the hemoglobin gene and leads to chronic anaemia, severe pain episodes and organ damage. However despite its serious health impact, the disease often remains neglected due to limited awareness and underdiagnosis – especially in remote areas. India accounted for 14.5 per cent of the global SCD births in 2023 with over 42,000 newborns affected that year. The country launched a National Sickle Cell Anaemia Elimination Mission in July 2023. However parallel scientific initiatives have been underway and across CSIR labs the focus was on point-of-care diagnostic tools. 'While the screening test kit is expected to cost less than Rs 100, we are also aiming to bring down the treatment cost to Rs 50 lakh. The treatment for one patient is estimated at Rs 28 crore in the United States and focused efforts are underway at our labs on genome editing approaches for correcting the sickle cell mutation for a potential cure. We are hoping to bring down the treatment cost to approximately Rs 50 lakh,' Dr Kalaiselvi said. Artificial Intelligence (AI) is now playing a vital role across multiple sectors, including energy and road traffic management. The DG CSIR spoke about some of the success stories including the project i-RASTE and how the initiative is in the process of expanding to other states. 'In the field of experimental chemistry and materials science, AI is transforming the way we explore new interfaces — for example, when selecting metals or materials. Traditionally, an experimental chemist would go through multiple trial-and-error cycles, but AI can now help minimize the number of iterations by predicting likely outcomes,' the DG-CSIR said. 'In battery technology, particularly with lithium cobalt oxide batteries used in mobile phones, overcharging can lead to thermal runaway and potentially cause fires. AI is now being integrated into battery management systems to detect overheating early and automatically cut off charging to prevent such incidents,' she added. AI also holds great promise in reducing the cost and time involved in scientific research. 'While the exact cost savings are yet to be quantified, the potential is clear,' Dr Kalaiselvi added. On hydrogen technology, the DG-CSIR said that Indian Oil Corporation is making significant investments. She also mentioned hydrogen adoption in e-mobility has been limited so far because of high costs around $3 per kg. 'For hydrogen to become economically viable, especially for widespread use in transportation, the cost needs to come down to around $1 per kg.' In her keynote address, Dr. N. Kalaiselvi, DG-CSIR, said, 'Nobel laureates like Einstein said truth is science, and Tagore believed science is truth but after visiting MIT-WPU, I would add that science is also peace. What's truly inspiring is the mindset shift among students, they're not just seeking jobs, they're building startups and creating employment. This silent revolution is made possible through science and must be nurtured,' she said. Prof. Dr. Ashutosh Sharma, Former Secretary, Department of Science and Technology (DST), Dr Shekhar Mande, former DG-CSIR and Dr. Vijay . Bhatkar, Founder Director, C-DAC was also present at the inaugural event . On the occasion the prestigious Vigyan Maharshi Award was posthumously conferred upon the late Dr. Jayant Narlikar, one of India's most celebrated astrophysicists. The award, which carries a cash prize of Rs 5 lakh, was received by his daughter, Dr. Leelavati Narlikar. Anuradha Mascarenhas is a journalist with The Indian Express and is based in Pune. A senior editor, Anuradha writes on health, research developments in the field of science and environment and takes keen interest in covering women's issues. With a career spanning over 25 years, Anuradha has also led teams and often coordinated the edition. ... Read More

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 Drugs Market 2034: Clinical Trials, Prevalence, EMA, PDMA, FDA Approvals, Medication, Statistics, NICE Approval.Revenue and Companies by DelveInsight

Globe and Mail

time07-07-2025

  • 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. 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