Exploring the latest emerging drugs for the treatment of sickle cell disease

The landscape of therapy in patients with SCD has recently moved toward more personalized therapeutic approaches. Promising preliminary data were obtained with some disease-modifying agents and gene therapies became available and promising for curing patients. However, the high cost remains a major limiting factor for these new therapies, which are far from being used in all patients, even in the more developed countries. Hydroxyurea is still regarded as the best disease-modifying treatment for SCD and allogeneic hematopoietic stem cell transplantation as the standard 'curative' therapy. Continued research is still warranted to sustain therapeutic advancements and development in SCD.

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Publication Details

Journal
Expert Opinion on Emerging Drugs
Published
2026-03-23
DOI
https://doi.org/10.1080/14728214.2026.2650178
Primary Topic
Hemoglobinopathies and Related Disorders
Type
article
Field-Weighted Citation Impact
0.00
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article

Exploring the latest emerging drugs for the treatment of sickle cell disease

Giovanna Cannas
Expert Opinion on Emerging Drugs
Hemoglobinopathies and Related Disorders
article

Exploring the latest emerging drugs for the treatment of sickle cell disease

Giovanna Cannas
article en

Abstract

The landscape of therapy in patients with SCD has recently moved toward more personalized therapeutic approaches. Promising preliminary data were obtained with some disease-modifying agents and gene therapies became available and promising for curing patients. However, the high cost remains a major limiting factor for these new therapies, which are far from being used in all patients, even in the more developed countries. Hydroxyurea is still regarded as the best disease-modifying treatment for SCD and allogeneic hematopoietic stem cell transplantation as the standard 'curative' therapy. Continued research is still warranted to sustain therapeutic advancements and development in SCD.

Expert Opinion on Emerging Drugs
Hôpital Edouard Herriot (FR), Cells Therapy (Poland) (PL)
Industry, innovation and infrastructure
Openalex Percentile: Top 13%
Hemoglobinopathies and Related Disorders
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