Hemoglobinopathies

As the pioneers of gene-based treatments for sickle cell disease and beta thalassemia, CRISPR Therapeutics is also working on next-generation approaches with the goal of expanding the global impact of gene editing.

The inherited hemoglobinopathies sickle cell disease (SCD) and β-thalassemia result from mutations in a gene that encodes a key component of hemoglobin, the oxygen carrying molecule in blood. These genetic disorders of hemoglobin (hemoglobinopathies) result in reduced life expectancy and currently require lifetime treatment, such as regular blood transfusions and frequent hospitalizations.

We advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic in 2018, and this treatment is now approved in some countries for certain eligible people living with SCD or β-thalassemia.

Our approach to treat SCD and β-thalassemia is designed to switch back on expression of a different form of hemoglobin called fetal hemoglobin (HbF), which is naturally present in all people at birth. Increased levels of HbF are intended to substitute for the diseased adult hemoglobin in patients with SCD and β-thalassemia, with the goal of reducing or eliminating symptoms.

Our next frontier

We are investigating two next-generation approaches that have the potential to significantly expand the addressable population with SCD and β-thalassemia (please visit our Pipeline page for more information).

• We are advancing our wholly owned targeted pre-treatment conditioning program through research studies. This work could significantly expand the number of patients eligible for current treatments.
• We have ongoing research efforts to enable in vivo editing of hematopoietic stem cells. We believe this could obviate the need for pre-treatment conditioning altogether, expand geographic reach, and enable the treatment of diseases or conditions beyond SCD and β-thalassemia.