Sickle Cell Disease: A Cure Within Reach, Justice Still Distant
White paper
Clinical
Introduction
Sickle cell disease (SCD) is a monogenic inherited blood disorder, characterized by abnormal hemoglobin in red blood cells. Oxygen is carried throughout the body via hemoglobin and a missense mutation in the β-globin gene results in a defective sickle-like form. This causes the formation of insoluble hemoglobin polymers and impairing of red blood cell membrane and ultimately leads to sickled erythrocytes. These crescent-shaped red blood cells impede blood flow as they are prone to clustering, restricting oxygen delivery to tissues and leading to extreme pain and even organ damage, known as vaso-occlusive events or crises. This is just the hallmark of the disease; additional symptoms include increased susceptibility to infections, anemia, and in severe cases, premature death.1
Sickle cell disease impacts 8 million people worldwide — 107,182 in the US — most of whom are of African descent.
This prevalence is believed to be because of the evolutionary advantage it offered against malaria.
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About the authors
Abdirazak Mohamed
Senior Drug Analyst, Citeline
Abdirazak Mohamed is a Senior Drug Analyst for Pharmaprojects, specializing in cardiometabolic, infectious, and genitourinary diseases, as well as vaccines. Since joining Pharmaprojects in 2022, he has contributed in-depth therapeutic area expertise and strategic insight into global drug development trends. Abdirazak holds a bachelor’s degree in pharmaceutical science and a master’s degree in drug discovery and pharma management. Prior to his current role, he worked as an Associate Scientific Assessor at the UK Medicines and Healthcare products Regulatory Agency (MHRA), gaining valuable experience in regulatory affairs.
Athanasia Agapiou
Drug Analyst II, Citeline
Athanasia Agapiou is a Drug Analyst II for Pharmaprojects, involved in various data enhancement projects and providing support to the Ask the Analyst service. She engages in drug landscape analyses and expert subject matter queries in the central nervous system and autoimmune disease space. Athanasia joined Pharmaprojects in 2024 and has a bachelor’s degree in biomedical science from the University of Surrey and a master’s degree in biomedical engineering from Imperial College London.
Use Case
Patient Engagement & Recruitment
Accelerating Diversity Enrollment in a Phase III COVID-19 Clinical Trial
A biotech need to rapidly meet diversity/inclusion goals for a high-profile Phase III COVID-19 trial. Citeline Connect helped the sponsor recover from a 72% decline in diversity-focused randomizations, delivering 20-25% of daily randomizations in 32 days.
References
1. National Heart, Lung, and Blood Institute (NHLBI) (n.d.) Sickle Cell Disease. Available from: https://www.nhlbi.nih.gov/health/ sickle-cell-disease [Accessed May 12, 2025]