Gene-editing therapy frees first trial participant from SCD symptoms

A patient with severe sickle cell disease (SCD) remained free of two key SCD symptoms — including painful vaso-occlusive crises — for more than a year after receiving a single dose of CS-206, Correctsequence Therapeutics‘ investigational gene-editing therapy, new data show.

The woman represents the first patient treated with CS-206 in a China-based Phase 1 clinical trial (NCT06565026), which is still recruiting an estimated five people with the inherited blood disorder, the company announced in a press release. According to the developer, the woman has shown no signs of anemia, marked by low red blood cell counts, nor experienced vaso-occlusive crises, known as VOCs, since receiving treatment.