RealFeedApp Base editing treatment freed sickle cell patients from severe pain
Beam Therapeutics announced positive results from a pivotal trial of its base editing treatment, which aims to precisely alter individual nucleotides to address sickle cell disease symptoms.
- Field
- gene therapy
- What they did
- Beam Therapeutics demonstrated that its base editing therapy helped patients with sickle cell disease avoid severe pain episodes by precisely changing individual DNA letters.
- Why it matters
- These results represent the first positive late-stage readout for a base editing drug approach, and the company plans to submit an FDA approval application by the end of the year.
#base editing#sickle cell#gene therapy#fda#beam therapeutics
