Research
Beam’s base editing therapy for sickle cell hits goal in pivotal trial
BioPharma Dive · By Jonathan Gardner ·
The Brief
- Beam's base editing therapy for sickle cell disease met its goal in a pivotal trial.
- Beam may submit a first-of-its-kind FDA application as soon as the end of the year.
Why it matters
A pivotal trial meeting its primary goal is a key step for a gene-editing treatment for sickle cell disease, and the planned FDA filing would be the next regulatory step toward patient access.
Who’s affected
- people with sickle cell disease and their clinicians
- researchers in gene and base editing
- #sickle cell disease
- #base editing
- #gene editing
- #clinical trial
- #gene therapy
The result represents a scientific milestone and positions Beam to submit a first-of-its-kind FDA application as soon as the end of the year.
Timeline
Pivotal trial reported to hit its goal
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Beam’s base editing therapy for sickle cell hits goal in pivotal trial