Gene Therapy Approved by the FDA

December 22, 2017

Mid Atlantic Retina is pleased that the FDA has approved gene therapy, voretigene (Luxturna), for very specific uncommon genetic retinal diseases, caused by mutations in the gene RPE65 which may cause a form of Leber Congenital Amaurosis or retinitis pigmentosa.  These disorders cause severe loss of vision starting at birth or in early childhood.  Not all patients with Lebers or Retinitis Pigmentosa will benefit from this treatment as this treatment is only for patients with mutations in this specific gene.

Mid Atlantic Retina and Wills Eye Hospital are involved in very early stage clinical research trials for gene therapies in several genetic eye diseases but there are no other approved gene therapies at this time for other retinal diseases.

Genetic testing can be performed through the Wills Eye Ocular Genetics service.

To arrange a genetic testing consultation call 215-928-3240.




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