Anemia, Sickle Cell
Recent clinical, regulatory, research and industry developments relating to this disease.
Association Between Low Bone Density, Vertebral Fractures, and Pain in Sickle Cell Disease
Sickle Cell Disease: From Genetics to Curative Approaches.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 2 clinical trials expected to report results, the earliest in Q1 2027.
- Q1 2027Pilot Study to Evaluate the Safety and Feasibility of Induction of Mixed Chimerism in Sickle Cell Disease Patients With COH-MC-17: a Non-Myeloablative, Conditioning Regimen and CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant
- Q2 2028A Phase 1/2a, First-in-human, Randomized, Double-blinded, Placebo-controlled, Dose-finding Study in Healthy Volunteers and Participants With Sickle Cell Disease to Evaluate the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics, pH and Food Effect, and Preliminary Efficacy of BMS-986470
Clinical MilestonesViewHide
- 2026-01-07Assessing Combination Hydroxyurea and Exogenous Erythropoietin in Sickle Cell DiseaseResults posted
- 2026-07-06A Phase 1/2a, First-in-human, Randomized, Double-blinded, Placebo-controlled, Dose-finding Study in Healthy Volunteers and Participants With Sickle Cell Disease to Evaluate the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics, pH and Food Effect, and Preliminary Efficacy of BMS-986470Results expected Q2 2028
- 2026-03-05Pilot Study to Evaluate the Safety and Feasibility of Induction of Mixed Chimerism in Sickle Cell Disease Patients With COH-MC-17: a Non-Myeloablative, Conditioning Regimen and CD4+ T-cell-depleted Haploidentical Hematopoietic TransplantResults expected Q1 2027
- 2026-07-06ClinicalA Phase 1/2a, First-in-human, Randomized, Double-blinded, Placebo-controlled, Dose-finding Study in Healthy Volunteers and Participants With Sickle Cell Disease to Evaluate the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics, pH and Food Effect, and Preliminary Efficacy of BMS-986470Results expected Q2 2028
- 2026-03-05ClinicalPilot Study to Evaluate the Safety and Feasibility of Induction of Mixed Chimerism in Sickle Cell Disease Patients With COH-MC-17: a Non-Myeloablative, Conditioning Regimen and CD4+ T-cell-depleted Haploidentical Hematopoietic TransplantResults expected Q1 2027
- 2026-01-07ClinicalAssessing Combination Hydroxyurea and Exogenous Erythropoietin in Sickle Cell DiseaseResults posted
Clinical trials
The current development programme across all trial phases.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes8
- Anemia, Sickle Cell4
- Acute Pain1
- beta-Thalassemia1
- Chronic Pain1
- Cognitive Dysfunction1
- CRISPR-Cas Systems1
- Dietary Supplements1
- Genetic Therapy1
Leading journals5
- Annual review of genomics and human genetics1
- Blood advances1
- Medical science monitor : international medical journal of experimental and clinical research1
- Scientific reports1
- The New England journal of medicine1
Leading researchers8
- Afiukwa CA1
- Agu PC1
- Aja PM1
- Altshuler D1
- Arlet JB1
- Bartolucci P1
- Bismuth H1
- Calvet D1
Affiliations (unnormalised)6
- Université Paris Cité2
- Caritas University1
- Centre d'Investigation Clinique and Unité de Recherche Clinique1
- Centre d'Investigation Clinique Spécialisé en Biothérapie1
- Centre de formation et d'appui sanitaire1
- Department of Science Laboratory Technology (Biochemistry Option)1
Related conditions
Diseases frequently studied alongside this one. Number shows shared papers.
Disease profile
A grounded synthesis of the condition — overview, causes, mechanism, risk factors and current standard of care.
Sickle cell anemia is a chronic hemolytic blood disorder marked by episodic painful crises and involvement of multiple organs. It is the clinical expression of homozygosity for hemoglobin S, and sickle cell disease more broadly is described as a monogenic blood disease caused by a point mutation in the beta-globin gene.
It is caused by homozygosity for hemoglobin S, which arises from a point mutation in the gene coding for beta-globin. The supplied grounding also notes that genetic modulators, polymorphisms, and modifier genes can influence how the disease is expressed and how severe it becomes.
Under low-oxygen conditions, sickle hemoglobin polymerizes and causes red blood cells to sickle. This leads to chronic hemolytic anemia, painful crises, and pathologic involvement of many organs.
Genetic factors that modify globin expression or otherwise modulate disease severity can influence the clinical course. The grounding also indicates that polymorphisms and modifier genes affect expression and severity, but it does not provide non-genetic risk factors.
The supplied material supports treatment at the level of pharmacological and genetic approaches, including curative approaches and gene therapy. It also mentions disease management in the context of nutraceutical research, but does not establish nutraceuticals as standard care.
AI-generated summary grounded in MeSH and 2 peer-reviewed sources. Informational only — not medical advice. Generated 2026-07-07.
Reference
Authoritative identity, definition & identifiers.
A disease characterized by chronic hemolytic anemia, episodic painful crises, and pathologic involvement of many organs. It is the clinical expression of homozygosity for hemoglobin S.
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.