Gene-Editing Therapies for the Treatment of Sickle Cell Disease

Sickle cell disease is caused by a mutation in the HBB gene that results in a lack of functional hemoglobin and sickle-shaped red blood cells that can accumulate in blood vessels and lead to vaso-occlusive crises.

Our scientists developed a method for treating sickle cell disease by using mRNA encoding gene-editing proteins in donor or patient hematopoietic cells to promote the expression of functional hemoglobin.

Gene Editing Therapies for the Treatment of Sickle Cell Disease is protected by a pending U.S. patent (with additional patents pending in other countries).

• Replicate natural expression of functional hemoglobin using site-specific genome engineering of a patient’s cells
• Achieve high-efficiency delivery of mRNA encoding gene-editing proteins to hematopoietic cells ex vivo using electroporation
• Administer a cell replacement therapy following an immune cell depletion treatment
• Develop a single product that can treat sickle cell disease with a single administration

• Achieve complete replacement of functional HSCs and a persistent therapeutic benefit
• Gene edit somatic cells to avoid the risk of germline transmission
• Combine with Factor’s Engineered Protein-Encoding RNA to streamline manufacturing of sickle cell therapies
• Combine with Factor’s Chromatin Context-Sensitive Gene-Editing Endonuclease for high-specificity ex vivo or in vivo gene editing