FDA Advisers Endorse Gene Therapy To Treat Form of Blindness (cbsnews.com)

An anonymous reader quotes a report from CBS News: A panel of U.S. health advisers has endorsed an experimental approach to treating inherited blindness, setting the stage for the likely approval of an innovative new genetic medicine. A panel of experts to the Food and Drug Administration voted unanimously in favor of Spark Therapeutics' injectable therapy, which aims to improve vision in patients with a rare mutation that gradually destroys normal vision. The vote amounts to a recommendation to approve the therapy. According to Spark Therapeutics' website, inherited retinal diseases are a group of rare blinding conditions caused by one of more than 220 genes. Some living with these diseases experience a gradual loss of vision, while others may be born without the ability to see or lose their vision in infancy or early childhood. Genetic testing is the only way to verify the exact gene mutation that is the underlying cause of the disease.

Comments

This is HUGE

[virtualXTC]

Considering that what this drug is treating isn't a life-threatening condition, the vote to approve this drug shows just how confident regulators are that this form of direct gene modification is likely to prove to be safe in the long-term. Because of this, we can expect a flood of applications for new gene therapies in the next few years.

Advisory Committee Materials & Presentations

[drunken_boxer777]

The FDA Advisory Committee materials and presentations are here, for those interested.

Note that this is for a very specific genetic cause of blindness, where a mutation in a gene for an enzyme results in effective loss of that specific enzyme. The drug is a retroviral vector encoding only the missing gene. Other causes of blindness, genetic or otherwise, wouldn't benefit from this treatment.

Pretty amazing, and a long time coming for gene therapy, since Jesse Gelsinger's death prompted a long close look at using adenoviral vectors for gene therapy.

Re:Sneaky

[Humbubba]

I take your point that genetic testing can be used against us by insurance companies. This is a very powerful tool with awesome benefits and consequences. Genetic testing, medicine and genetic engineering has the potential to make a "better" human race. And therein lies the problem.

In Brave New World, Aldous Huxley foresees genetic engineering used to subordinate the lower classes by making them stupid and purging creative and destructive thought processes. Yuval Harari, in Homo Deus, predicts the rich and powerful will use genetic engineering to give their offspring immortality, happiness and even more power, and in the process create another kind of human. At some point these super humans will integrate with AI in a singularity that renders unnecessary current human affairs - and current humans.

Even if the bleak predictions of Huxley or Harari do not come true, once we tamper with our genetic code, there's no turning back. We lose the nature's evolutionary sorting in favor of designer genes. I'm not sure which best for the survival of the species.