The US Food and Drug Administration approves the first gene therapy for a rare skin disease
The US Food and Drug Administration approves the first gene therapy for a rare skin disease
The US Food and Drug Administration approves the first gene therapy for a rare skin disease
The US Food and Drug Administration recently approved the first topical gene therapy intended for a rare skin condition, which will be used to treat wounds in patients 6 months of age and older.
The authority stated in a statement, according to the medical magazine “medscape”, that the new topical treatment treats people with rare DEB epidermolysis bullosa, which appears in two stages, recessive and dominant.
She added that the new treatment uses a non-recurrent herpes simplex virus type 1 (HSV-1) vector to deliver the COL7A1 gene directly into skin cells, restoring COL7 protein fibers that stabilize skin structure.
Patients with the disease usually suffer from mild cases with sores that mainly affect the hands, feet, knees, and elbows, but in its later stages it causes vision loss, deformity, and other serious medical complications that may be fatal.
