FDA signs off on study of CRISPR drug for sickle cell disease

Intellia Therapeutics (NTLA -2.0%) announces that the FDA has signed off on collaboration partner Novartis’ (NVS -0.6%) IND for a Phase 1/2 clinical trial evaluating a CRISPR/Cas9-based engineered cell therapy, OTQ923, for the potential treatment of adults with sickle cell disease (SCD). The action triggered a milestone payment from Novartis.

The company says OTQ923 was created by using CRISPR/Cas9 to edit the genome of hematopoietic stem cells to induce fetal (normal) hemoglobin expression. The edited cells are reinfused into the patient which should reduce the negative effects of sickle hemoglobin.

https://seekingalpha.com/news/3556807-fda-signs-off-on-study-of-crispr-drug-for-sickle-cell-disease