FDA approves First Gene Therapy That Fixes Hereditary Blindness

From The First Gene Therapy That Fixes Hereditary Blindness May Finally Get FDA Approval

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.

however

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.