An application to the U.S. Food and Drug Administration is expected to be supported by data that showed risto-cel's benefit in 31 patients. As a medical scientist for 18 years, he worked in both academia and industry, where his research focused on the discovery of new vaccines and medicines to treat inflammatory disorders and infectious diseases. Steve is a published author in multiple peer-reviewed scientific journals and a patented inventor. Gene-edited cell therapy U.S. Beam Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to approve ristoglogene autogetemcel, or risto-cel, its single-dose, gene-edited cell therapy candidate for people with sickle cell disease (SCD), as early as year’s end. Disease burden heavy for SCD patients in US, despite promise of gene therapy Base editing is a precise gene-editing technology that can create a change in the DNA’s sequence without cutting through both side rails of the DNA’s twisted ladder, a characteristic structure known as its double helix. Such cuts can damage DNA and create unwanted genetic abnormalities. Interim trial data is described in the study “Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease,” which was published in The New England Journal of Medicine.

Original title: “Beam to seek OK of gene-edited cell therapy risto-cel for sickle cell disease”

beyond pertussis bordetella holmesii bacteremia in children with sickle cell disease a case series with literature review

Original title: “Beyond Pertussis: Bordetella holmesii Bacteremia in Children With Sickle Cell Disease: A Case Series with Literature Review.”

Shares of Immunovant, Alto and Oric Pharmaceuticals fell this week on clinical trial updates. Elsewhere, Orca Bio’s cell therapy hit a delay and Korsana entered a reverse merger.

Original title: “Immunovant eye drug flunks Phase 3 studies; Beam sickle cell data published in NEJM”

Durable expression of anti-sickling fetal hemoglobin and reduction in sickle hemoglobin observed In patients with sickle cell disease treated with ristoglogene autogetemcel, mean anti-sickling fetal hemoglobin levels were above 60% with a mean durable reduction in corresponding sickle hemoglobin below 40%. The median time to neutrophil and platelet engraftment was relatively short -- 17.5 days and 19 days, respectively. No patients had an investigator-reported severe vaso-occlusive crisis after engraftment. Treatment with the investigational base-editing gene therapy ristoglogene autogetemcel (risto-cel) showed promise in patients with sickle cell disease, according to an interim analysis of the phase I/II BEACON study. As described by Heeney and colleagues, unlike other gene therapy strategies, risto-cel introduces precise edits at the HBG1 and HBG2 promoter regions to inhibit BCL11A binding and promote the expression of anti-sickling fetal hemoglobin. "This targeted approach preserves overall BCL11A expression, avoiding disruption of its broader biologic functions," they pointed out. Gene-editing strategies are designed to directly address the underlying genetic mutation responsible for the disease or induce the production of fetal hemoglobin. This approach was endorsed by the FDA with the approval of exagamglogene autotemcel (exa-cel; Casgevy) -- a CRISPR/Cas9 gene-edited therapy -- for use in patients ages 12 and older with sickle cell disease and transfusion-dependent beta thalassemia. Locatelli pointed out that ex vivo gene therapy is resource-intensive and ill-suited to treat large numbers of patients in countries with limited resources, where most patients with sickle cell disease and transfusion-dependent beta thalassemia live.

Original title: “Base-Editing Gene Therapy Shows Promise in Sickle Cell Disease | MedPage Today”

Gene therapies cost $2.1M to $4.25M. Insurance barriers and Medicaid gaps are reshaping access for rare disease families. A single infusion for a child with this fatal neurodegenerative disease carries a price tag higher than most people will earn in a lifetime. Hemgenix, approved in 2022 for hemophilia B, costs $3.5 million. Lyfgenia, for sickle cell disease, is $3.1 million. Casgevy, the first CRISPR-based gene therapy by Vertex, is $2.2 million. Bluebird bio developed gene therapies for beta-thalassemia (Zynteglo), sickle cell disease (Lyfgenia), and cerebral adrenoleukodystrophy (Skysona). These were not failed therapies. They worked. Patients got better. NewsGene TherapyHealthcare AccessRare Disease PolicyMarch 29, 2026 · Gene therapies offer cures for previously untreatable rare diseases, but prices reaching $4.25 million create devastating access barriers. Therapies that literally cure genetic diseases once thought incurable are now available. But for the families who need them most, these breakthroughs have become locked behind a pricing wall so high it is often impenetrable. The gap between scientific innovation and patient access has never been wider. A therapy that works exists, yet most patients cannot access it. This is not a hypothetical problem. It is the daily reality for families navigating a rare disease diagnosis.

Original title: “Gene Therapy Pricing | Rare Disease Access Barriers | Trial Friend”

cryopreservation of red blood cells from sickle cell disease patients for comparison of oxygen gradient ektacytometry parameters between laboratories to enable data harmonization

Original title: “Cryopreservation of Red Blood Cells From Sickle Cell Disease Patients for Comparison of Oxygen Gradient Ektacytometry Parameters Between Laboratories to Enable Data Harmonization.”

As more advanced cell and gene therapies, including gene editing technologies, progress through drug development, there is increased emphasis on the importance of stakeholders, including people living with disease, caregivers, and healthcare professionals, to communicate using clear, accurate, ... As more advanced cell and gene therapies, including gene editing technologies, progress through drug development, there is increased emphasis on the importance of stakeholders, including people living with disease, caregivers, and healthcare professionals, to communicate using clear, accurate, and consistent language. The biotechnology company bluebird bio developed a suite of educational resources for patients on gene therapy aimed at an audience of “beginners to advanced learners”, including concepts and terms used to describe lentiviral gene therapy for beta-thalassemia and sickle cell disease [37]. Topics include descriptions and examples of genetic diseases, gene therapy approaches and clinical trials, a comprehensive glossary defining terms used in their resources, and step-by-step explanations of the gene therapy process [37]. The Thalassaemia International Federation has also produced a comprehe Similarly, gene editing terms are described in the GenePossibilities educational materials developed by Vertex Pharmaceuticals for patients and HCPs, hosted on separate dedicated sites [40, 41]; notably, the patient glossary includes phonetic pronunciation. It is interesting that, despite the availability of lexicons for diseases such as hemophilia, beta-thalassemia, and sickle cell disease, patient interest and uptake of gene therapies have been slow [42]. The lexicon has been beta-tested with two large hemophilia patient advocacy groups, the National Bleeding Disorders Foundation in the USA and the European Haemophilia Consortium. The collaborative, community-based approach used in the development of the hemophilia lexicon makes it particularly well-suited for further testing, as the initial participants represent a cross-section of key stakeholders involved in hemophilia gene editing therapy.

Original title: “Building a gene editing lexicon: a model for rare and inherited disorders | Gene Therapy”

asthma screening in children with sickle cell disease in french guiana

Original title: “Asthma screening in children with sickle cell disease in French Guiana.”

Referred to the House Committee on Energy and Commerce.

Original title: “HR 5178: Sickle Cell Disease Comprehensive Care Act”

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”