Gene therapy receives ‘breakthrough’ status
The US Food and Drug Administration has granted ‘breakthrough therapy’ status to a gene therapy that is in Phase 3 development for patients with an inherited eye disorder that can lead to blindness. The developer is Spark Therapeutics of Philadelphia, Pennsylvania.
The US Food and Drug Administration has granted ‘breakthrough therapy’ status to a gene therapy that is in Phase 3 development for patients with an inherited eye disorder that can lead to blindness. The developer is Spark Therapeutics of Philadelphia, Pennsylvania.
Breakthrough designation means a developer is eligible for a faster review of its product at the FDA. It is awarded to products that promise to significantly improve the treatment of a disease compared with an existing therapy.
The Spark gene therapy is intended to correct a mutated gene that is responsible for the production of retinal pigment epithelium-specific 65 protein, an important agent for sight. Mutations of the gene can lead to the loss of protein and to Leber’s congenital amaurosis, a severe early onset degeneration of the retina.
The therapy involves delivering the RPE65 gene in an adeno-associated virus to the back of the eye. This is done through a subretinal injection.
The Phase 3 trial is being conducted at the Children’s Hospital of Philadelphia. It is expected to report in the second half of 2015.
Spark Therapeutics announced the breakthrough therapy designation on 6 November 2014.
– Victoria English
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