AgafyaTM
The dream of a disease free world
Our approach with Agafya aims to treat Sickle cell disease (SCD) and Beta Thalassemia by redesigning hematopoietic stem cells to increase fetal haemoglobin (HbF) expression in the blood.
Current Line of Treatment
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Blood Transfusion- Risk of Complications
- Frequent Hospital Visits
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Bone marrow transplant- Low success rate
- Not scalable
- Lifelong dependency on immunosuppressive medication
Significant Worldwide Burden
Annual Cases
Sickel Cell
300K
Beta Thalassemia
60K
SCD and Beta Thalassemia are genetic disorders that deform the haemoglobin within red blood cells and hinder their ability to carry oxygen across the body. Both of these diseases are chronic in nature, possess painful symptoms and require continuous intervention for the duration of the patient’s life.
Agafya is an advanced autologous cell therapy that presents an accessible solution for SCD and Beta Thalassemia. Our approach with Agafya involves increasing HbF expression and using the increased HbF to suppress and substitute the mutated haemoglobin present in Sickle cell and Beta Thalassemia patients. We use an advanced CRISPR-Cas system (with minimal off targets) to target a gene within hematopoietic stem cells that is responsible for HbF suppression. By doing this we are able to substantially increase HbF levels in blood, thereby significantly reducing or completely eliminating symptoms of SCD or Beta Thalassemia in patients.
