New off-the-shelf gene editing may treat sickle cell without transplant

Single dose safely corrected primate stem cells to levels considered curative

An experimental, single-dose gene-editing therapy from Tessera Therapeutics successfully corrected the genetic mutation that causes sickle cell disease (SCD) in the majority of blood-forming stem cells in nonhuman primates, reaching levels that surpass what is expected to cure the disease.

The new preclinical data were presented by Tessera at the annual meeting of the American Society of Gene and Cell Therapy and announced in a company press release.