Gene therapy typically uses an engineered virus to administer a patient with a faulty gene with a corrected version. Rather than simply responding to the symptoms of the condition in question, it attempts to make changes to the individual’s genetic make-up in order to solve the problem at its root.
Luxturna fixes a mutation in a gene known as RPE65, which is responsible for telling the body how to produce a protein that’s essential for normal eyesight. It introduces billions of engineered virus particles bearing a corrected version of the gene to the retinal cell, via a quick injection to the eyes.
It’s not an outright cure, and it doesn’t give recipients full 20/20 vision. There’s currently no data on how long its effects last, so there’s a chance that patients’ sight might begin to recede once again over time.
Cost is also a major factor in how accessible it is. Two of the treatment’s biggest competitors, Strimvelis and Kymriah, cost around $700,000 and $475,000 respectively.
It’s a lot of money to try something that is unlikely permanent, so at the moment this remains for very few privileged humans. But what an incredible step forward.