FDA approves first gene therapy for hereditary disease — Spark Therapeutics’ Luxturna

On December 19, 2017, the US FDA approved a new gene therapy to treat children and adults with a genetic condition that often results in blindness. Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl) is a one-time gene therapy product that treats biallelic RPE65 mutation-associated retinal dystrophy, an inherited retinal disease that frequently progresses to complete blindness. Approval

Expanded genetic code with non-natural DNA rewrites life as we know it

Biological organisms use DNA to encode heritable information with just four nucleotide bases: adenine (A), thymine (T), cytosine (C), and guanine (G). These bases make up different three-letter codon sequences that produce 20 amino acids that make up proteins. Scientists have now tinkered with that standard genetic alphabet, opening up new possibilities for semi-artificial life.

FDA and the future of medicine: Advancing regenerative medicine options for patients with safety and science at the forefront

On November 16, 2017, the US FDA announced a policy framework for regenerative medicine, releasing two final and two draft guidance documents. These documents reflect the agency’s commitment to modernize the system to incorporate promising cell and gene therapies and provide a structure for these options to be introduced in a fast, effective, and safe