Background
A blood disorder where red blood cells are shaped like a crescent or sickle instead of being round.
What is sickle cell disease?
Casgevy, which uses CRISPR-Cas9 gene editing therapy to upregulate fetal hemoglobin synthesis, and Lyfgenia, which uses gene addition therapy to transplant gene-modified autologous hematopoietic stem cells with modified beta-globin genes in them.
What are 2 FDA approved Gene Therapies for SCD?
Medium level awareness, 48% of providers felt knowledgeable about gene therapies for SCD.
What kind of awareness do healthcare providers have regarding SCD gene therapies?
Sickle cell disease is caused by a change in the gene that makes hemoglobin, the part of the blood that carries oxygen.
What causes sickle cell disease?
- Financial: therapies are very expensive and insurance often doesn’t cover it or only offer partial coverage.
- Geographical: Therapies are not accessible to rural or global populations.
What are financial and geographical barriers to access for new gene therapies?
Sickle Cell Disease Training and Mentoring Program for Primary Care Providers program (STAMP)
What is an example of SCD-related education program for healthcare providers?
Pain aka "sickle cell crisis", feeling tired due to having less hemoglobin (and thus oxygen to organs) than others aka anemia. Blocked blood flow leading to swelling or even damage to organs.
What are symptoms of sickle cell disease?
A sense of medical mistrust for marginalized populations. There is misinformation and lack of public education on gene therapies.
What are sociocultural barriers to accessing new gene therapies?
Isolation
Name an emotion felt by some patients and caregivers dealing with SCD gene therapies.