Critical Path Institute (C-Path) formed a collaboration of organizations and will conduct planning and logistics for the consortium to begin work in furthering sickle cell anemia research.
The consortium will include scientists, clinicians, and representatives from the biopharmaceutical industry, governmental regulatory agencies, academic institutions, and patient advocacy organizations.
Lynn Hudson, chief science officer at C-Path told us, “The regulatory path that the new consortium will forge for sickle cell treatments should facilitate development of treatments for a number of rare diseases.”
The companies within the consortium have not yet been disclosed. DDCF stated that this information will be revealed during the planning phase of the project.
Betsy Myers, program director, and Sindy Escobar-Alvarez senior program officer from DDCF’s Medical Research Program told us, “A consortium model can benefit all participants by doing the work that otherwise each of them might have to do alone. A consortium model would save time, effort and resources. Diverse voices that are critical to the development of clinical trial endpoints will be heard.”
Going forward Myers and Escobar-Alvarez believe that the consortium can affect change in sickle cell disease research by establishing the identification of clinical trial endpoints and biomarkers for effective treatment measures and regulatory approval.
’Most commonly‘ inherited disease in US
Sickle Cell Anemia is an inherited blood disorder affecting roughly 90,000 to 100,000 Americans, according to DDCF. The company also cites that the disease is the “most common” inherited disease in the US.
According to the National Institute of Health (NIH), 1 in 13 individuals with African ancestry are born with the cell trait for sickle cell anemia and 1 in 365 of individuals with African ancestry are born with the disease, however, a person of any ancestry can inherit the gene.
The disease causes blood cells to “sickle” or become misshapen. Sickle cells lose flexibility and cannot stick to vessel walls. Due to sickle cells dying early individuals with sickle cell anemia often have lower red blood cell counts than those without the condition, causing fatigue, painful swelling in hands and feet, and severe complications, in some cases.