The director of the Sickle Cell Disease Program at Nemours Children’s Hospital discussed what clinics need in order to start administering gene therapy effectively. Some of them have already failed disease modifying therapies and are really looking for something else, but some haven't tried disease modifying therapies yet, and we might direct them that way first.” · Vertex Pharmaceuticals' and CRISPR Therapeutics’ gene editing therapy · exagamglogene autotemcel (exa-cel; marketed as Casgevy) and bluebird bio’s gene addition therapy · lovotibeglogene autotemcel (lovo-cel; marketed as Lyfgenia) were approved by the FDA for the treatment of sickle cell disease (SCD) in late 2023. 67th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 6 to 9, 2025, in Orlando, Florida, CGTLive® spoke with Robin E. Miller, MD, the director of the Sickle Cell Disease Program at Nemours Children’s Hospital, to learn more about the practical considerations for treatment centers that are integrating or considering integrating gene therapy into their clinical care for SCD. Miller stressed that centers need key infrastructure, including SCD specialists, transplant experts, a robust blood bank, a stem cell lab, and experience with bone marrow transplant in SCD. Also important are psychosocial supports, including psychologists and social workers familiar with the unique challenges that families of patients with SCD face. She also explained that determining candidacy requires multidisciplinary input and shared decision making. Families must understand that while gene therapy is promising, it is a long process and carries risks. Some patients may be better suited for other therapies or traditional stem cell transplant, especially if a matched donor is available. Miller emphasized the importance of clearly explaining risks, benefits, and practical factors like recovery time and long-term follow-up. ... Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.

Original title: “Robin E. Miller, MD, on Integrating Gene Therapy into Clinical Care for Sickle Cell Disease | CGTlive®”

Blood stem cell gene therapy to treat hemoglobinopathies is beginning to transform health for small numbers of patients in the U.S. and Europe, where these conditions qualify as rare diseases. Yet hemoglobinopathies are common globally, disproportionately affecting low- and middle-income countries ... Blood stem cell gene therapy to treat hemoglobinopathies is beginning to transform health for small numbers of patients in the U.S. and Europe, where these conditions qualify as rare diseases. Yet hemoglobinopathies are common globally, disproportionately affecting low- and middle-income countries (LMICs), creating an ethical imperative to ensure access where disease burden is greatest. Gene Therapy - Cost-effectiveness of gene therapy for sickle cell disease in Uganda: tailoring high-income evidence to Uganda’s context Cost-effectiveness analysis (CEA) models are seldom adapted to low-income settings, where limited data and resources constrain efforts to contextualize high-income evidence. Here, we present a novel framework to evaluate high-income country authorized gene therapies in LMIC contexts. Uganda, where sickle cell disease (SCD) imposes a major burden and no curative therapies are available, is the test case. The Use of Cost-Effectiveness Analysis in Sickle Cell Disease: A Critical Review of the Literature. Pharmacoeconomics. 2021;39:1225–41. ... Herring WL, Gallagher ME, Shah N, Morse KC, Mladsi D, Dong OM, et al. Cost-Effectiveness of Lovotibeglogene Autotemcel (Lovo-Cel) Gene Therapy for Patients with Sickle Cell Disease and Recurrent Vaso-Occlusive Events in the United States.

Original title: “Cost-effectiveness of gene therapy for sickle cell disease in Uganda: tailoring high-income evidence to Uganda’s context | Gene Therapy”

cost effectiveness of gene therapy for sickle cell disease in uganda tailoring high income evidence to uganda s context

Original title: “Cost-effectiveness of gene therapy for sickle cell disease in Uganda: tailoring high-income evidence to Uganda's context.”

As Uganda rolls out mandatory nationwide screening of newborns for sickle cell disease this month, a gene therapy celebrated in the United States and Europe remains financially out of reach for most patients in Africa. Ayoola Olajide, president of Ikorodu Sickle Cell Club in Nigeria and a person living with sickle cell disease, told SciDev.Net: “For Africa, gene therapy treatment currently exists only on paper.” · According to the World Health Organization, about 515,000 babies are born each year with sickle cell disease, a lifelong, inherited blood disorder. Advocates stress that while gene therapy is causing a buzz in wealthy countries, African governments must prioritise prevention, screening and accessible treatment. Africa has the highest burden of inherited blood disorders globally, Bayigga noted. Short-duration psychedelic therapy shows promise for major depression treatment ... “You must be extremely wealthy to even think about it and I know 99 per cent of the Nigerians cannot afford it so it’s of no use to many.” · Nigeria carries the world’s largest burden of sickle cell disease, with an estimated 150,000 babies born with the condition each year. Millions more live with the disorder and many lack access to consistent treatment. In Uganda, the Ministry of Health launched a policy this month (9 February) requiring all babies born in health facilities to be screened for the disease at birth, free of charge, in a bid to reduce preventable deaths. Official figures show an estimated 20,000 Ugandan children are born with the condition each year, with up to 80 per cent dying before the age of five, largely due to delayed diagnosis and preventable complications. While African governments expand early detection and basic treatment, high-income countries have been adopting the first approved gene-editing therapy designed to cure sickle cell disease.

Original title: “Breakthrough gene therapy for sickle cell disease remains out of reach in Africa”

The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.

Original title: “Sickle Cell Gene Therapies Casgevy and Lyfgenia Still Lacking Traction 2 Years In”

sepsis induced coagulopathy sic is it monolithic

Original title: “Sepsis induced coagulopathy (SIC): is it monolithic?”

The Global Sickle Cell Disease and thalassemia gene therapy market size was US 83 87 Million in 2024 and is expected to reach US 380 42 Million by 2033 growing at a CAGR of 18 4 during the forecast period ... Press release - DataM intelligence 4 Market Research LLP - Sickle Cell Disease and ?-Thalassemia Gene Therapy Market Set for Explosive Growth to US$ 380.42 Million by 2033, Led by North America's 48.5% Market Share - published on openPR.com Introduction Sickle Cell Disease (SCD) is a group of inherited blood disorders caused by mutations in the hemoglobin gene, leading to abnormally shaped red blood cells. These "sickle" shaped cells result in chronic anemia, vaso-occlusive crises, organ damage, and reduced life expectancy. The Global Sickle Cell Disease and β-thalassemia gene therapy market size was US$ 83.87 Million in 2024 and is expected to reach US$ 380.42 Million by 2033, growing at a CAGR of 18.4% during the forecast period 2025-2033. Market expansion is propelled by advancements in gene-editing technologies like CRISPR/Cas9 and lentiviral vectors, enabling precise genetic corrections and potential cures. Rising clinical trials demonstrate long-term safety and efficacy, while regulatory approvals for therapies such as CASGEVY, LYFGENIA, and ZYNTEGLO boost adoption and investment. Increasing disease prevalence affecting over 6 million globally, along with partnerships accelerating commercialization, further fuel demand. Get a Free Sample PDF Of This Report (Get Higher Priority for Corporate Email ID):- https://www.datamintelligence.com/download-sample/sickle-cell-disease-scd-and-beta-thalassemia-gene-therapy-market?ram Key Industry Developments United States: ✅ February 2026: CRISPR Therapeutics highlighted ongoing momentum in CASGEVY launch for SCD and β-Thalassemia, noting strong patient demand, over 50 activated authorized treatment centers, and robust payer support including a new CMS outcomes-based agreement for equitable access.

Original title: “Sickle Cell Disease and β-Thalassemia Gene Therapy Market Set for Explosive Growth to US$ 380.42 Million by 2033, Led by North America's 48.5% Market Share”

comparison of mortality and complications after total hip arthroplasty in patients with sickle cell disease a retrospective match controlled study from t rkiye

Original title: “Comparison of Mortality and Complications After Total Hip Arthroplasty in Patients with Sickle Cell Disease: A Retrospective Match-controlled Study from Türkiye.”

concomitant occlusion of the deep dorsal vein and dorsal artery of the penis in a sickle cell anaemia patient

Original title: “Concomitant occlusion of the deep dorsal vein and dorsal artery of the penis in a sickle cell anaemia patient.”

Following over a year of slow uptake, Vertex Pharmaceuticals and CRISPR Therapeutics expect Casgevy revenues to nearly triple in 2026, as patient access to the sickle cell disease and beta thalassemia gene therapy grows.

Original title: “Vertex, CRISPR Set Lofty Goal for Casgevy Gene Therapy as Patient Starts Ramp”

Jiang: Our goal is to be able to treat or cure every rare genetic disease. The FDA approved CRISPR genome editing treatment for <strong>sickle cell therapy</strong> in 2023—which is quite impressive since CRISPR technology is relatively new.

Original title: “The Future of Gene-Editing Treatments for Rare Diseases | Yale School of Medicine”

Read twice and referred to the Committee on Health, Education, Labor, and Pensions.

Original title: “S 735: Sickle Cell Disease and Other Heritable Blood Disorders Research, Surveillance, Prevention, and Treatment Act of 2025”

Referred to the House Committee on Energy and Commerce.

Original title: “HR 1796: Sickle Cell Disease and Other Heritable Blood Disorders Research, Surveillance, Prevention, and Treatment Act of 2025”

Original title: “Charter Amendments, Establishments, Reestablishments, Renewals, and Terminations; Sickle Cell Advisory Committee”

NHS Scotland has approved a one-time CRISPR gene therapy for severe sickle cell disease, offering durable reduction in vaso-occlusive crises for eligible patients. This decision follows a full submission assessed through the SMC orphan medicine process and applies only in the context of an approved NHS Scotland Patient Access Scheme, which allows confidential pricing arrangements for high-cost therapies. SCD is a chronic, inherited blood disorder caused by mutations in the beta-globin gene, leading to the production of sickle haemoglobin. Clinical experts highlighted its potential to deliver durable disease modification after a single intervention, reducing dependence on hydroxycarbamide, blood transfusions, and recurrent hospital care. Exagamglogene autotemcel is a personalised, autologous, cell-based gene therapy that uses CRISPR/Cas9 gene-editing technology to modify a patient’s own stem cells. All patients experienced treatment-related adverse events, which were primarily attributable to busulfan conditioning treatment rather than to the gene therapy itself. Common events included febrile neutropenia, infections, and cytopenias, typically occurring within the first 6 months after infusion. No cases of graft failure or graft-vs-host disease were reported. Cite this: Edited by Zeel Mehta. Scotland Approves Gene Therapy for Sickle Cell Disease - Medscape - January 20, 2026.

Original title: “Scotland Approves Gene Therapy for Sickle Cell Disease”

compound heterozygosity for hemoglobin s and d punjab an interplay of two haemoglobinopathies rare mimic of sickle cell disease

Original title: “Compound Heterozygosity for Hemoglobin S and D-Punjab: An Interplay of Two Haemoglobinopathies/Rare Mimic of Sickle Cell Disease.”

New supplement from the American Journal of Preventive Medicine describes the state of sickle cell disease related care in the United States.

Original title: “Advancements in Sickle Cell Disease - Media Advisory”

The Advisory Committee for Immunization Practices voted today 13 to 1, with 1 abstention, to recommend that infants at increased risk for meningococcal disease should be vaccinated with 4 doses of HibMenCY at 2, 4, 6, and 12 through 15 months. These include infants with recognized persistent complement pathway deficiencies and infants who have anatomic or functional asplenia including sickle cell disease. HibMenCY can be used in infants ages 2 through 18 months who are in communities with serog

Original title: “CDC Advisory Committee on Immunization Practices Recommends HibMenCY for Infants at Increased Risk for Meningococcal Disease”

The first national estimate on the health status of children with sickle cell disease revealed that black children with sickle cell disease are more likely to have intellectual disabilities, hearing deficits, and frequent severe headaches or migraines than black children without sickle cell disease.

Original title: “CDC Releases Study on Children With Sickle Cell Disease”