Julian Adams discusses Gamida Cell’s CordIn for rare genetic diseases.

Each year, approximately 100,000 patients in the United States are living with sickle cell disease (SCD), a disease for which bone marrow transplantation is currently the only clinically established cure. SCD disproportionately impacts African Americans, affecting one in every 365 births. Patients diagnosed with SCD who are indicated for bone marrow transplantation face significant challenges in finding donor matches:

– Family members are often not suitable donors, as they may carry the associated genetic trait
– Ethnic minorities are underrepresented in donor registries, further limiting availability of a match
– As a result of multiple blood transfusions, patients with SCD have allo-activated immune systems that cause a high rate of graft failure

Gamida Cell is harnessing the potential of NAM technology to address the issues faced by patients with sickle cell disease and other rare genetic diseases through the development of CordIn. CordIn is a potential curative treatment for rare genetic diseases where a bone marrow transplant is an established cure, including sickle cell disease, thalassemia and aplastic anemia.