Gene Therapy: The Rocky Road to Success

The manipulation of genes to treat disease once seemed only to be the basis for an average science fiction novel. And if your story restored an individual’s vision by using a virus to replace a mutant version of a gene with a healthy one, you might have a best seller on your hands. But we all now know that this is not fiction; it is a description of Luxturna, the first FDA-approved gene therapy for an inherited retinal disease caused by mutations in both copies of the RPE65 gene. The concept of gene therapy was officially established back in 1972. And although gene therapy is considered a relatively new area of therapy, the field can be dated even further back to 1928, when Frederick Griffith first described the transforming principle. By 1968, Rogers and Pfuderer performed the first proof-of-concept viral mediated gene transfer. Fast-forwarding to 2003, China became the first country to approve a gene therapy for clinical use. By 2009, the first successful phase III trial of a gene therapy had occurred in the EU and in 2012, the EMA recommended the first gene therapy product for approval. Following on from this success, the FDA approved Luxturna, the first gene therapy for an inherited disease, in 2017

For further information and full text, see The Medicine Maker (https://themedicinemaker.com/manufacture/gene-therapy-the-rocky-road-to-success?registration-finished=1&cHash=34e6f08e3d10f752ab36557a2c3f0034)

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