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.

Nov 17, 2014
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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

Copyright 2014 Evernow Publishing Ltd

Victoria English

Editor, MedNous, a publication of Evernow Publishing Ltd

Co-founder and editor of Evernow Publishing Ltd. International journalist with previous full-time editorial positions at Informa Plc, Thomson Reuters, McGraw-Hill and Dow Jones Inc. Have worked as a correspondent covering finance in New York, Amsterdam, Brussels and London, and covering healthcare and the life sciences in London.

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