BOSTON — The trouble had started over a decade ago, when the Hogans noticed something wasn’t right with their son Jack. As a baby, he would spend hours staring into the lights in his nursery. Later, he sometimes walked into walls or fell down the stairs. When they asked him to pick up his toys, he wouldn’t — not because he didn’t want to, it turned out, but because he didn’t have the peripheral vision with which to see them strewn across the floor. They soon found out he was night-blind, too.

This past Sunday, his parents drove him five hours from New Jersey to Boston in the hope that he might regain some of his vision. On Tuesday, he was to be the first person in the U.S. to receive a gene therapy for a rare inherited disease since the treatment had hit the market.

Unlock this article by subscribing to STAT Plus and enjoy your first 30 days free!


What is it?

STAT Plus is a premium subscription that delivers daily market-moving biopharma coverage and in-depth science reporting from a team with decades of industry experience.

What's included?

  • Authoritative biopharma coverage and analysis, interviews with industry pioneers, policy analysis, and first looks at cutting edge laboratories and early stage research
  • Subscriber-only networking events and panel discussions across the country
  • Monthly subscriber-only live chats with our reporters and experts in the field
  • Discounted tickets to industry events and early-bird access to industry reports

Leave a Comment

Please enter your name.
Please enter a comment.

  • I see the next saga has been published…but what in the world is Neapolitan? My computer doesn’t recognize is and I can’t access it. I am highly peeved. I subscribe to Stat Plus, seems like I should be able to read whatever is advertised there!

  • I am writing from India. Three of My family members also affected in this kind of disease . So please let me know when this treatment is going to launch in India . We are waiting eagerly.

  • It would be interesting to know if research into the treatment was funded, and or partly funded by taxpayers through government grants. It seems that often, government / taxpayer funded research by drug companies. If this is the case, the high price tag is not justified.

  • As a parent my son has RP this is fantastic news. We too thought he was a clumsy child, not watching where he was going, kicking things over, completely blind in dimly lit areas. We live in the UK, no way can we afford the price tag of such a therapy, but price tag aside, this opens the door for all sorts of personalised medicine for genetic diseases, not just RP. Brilliantly written article and no matter which way you look at it, after searching for years with no potential cure, this means at last there is true hope on the horizon. Well done to the teams of people involved, lab staff, researchers, hospital staff, surgeons, professors, parents and patients. Please follow up this article with progress reports. We hope its a 100% success !!

  • I also have RP, was diagnosed at the age of 26yrs back in 1993. Now at the age of 51 life is extremely different. This is an exciting progression. However, I question the price! If the price were heavily reduced and more people had access the investors and people involved would make their money back and more. Would love to have the opportunity to have my vision back. Writing from Australia.

  • As wonderful as it seems to give this one child hope for better sight, the reality of health care is that there is no magic pot of gold to pay for astronomical costs of treatment. Premiums/deductibles/co-pays are now so high that even folks with health insurance unable often to pay for needed care and thus go without. At nearly 20% of GDP, the US has hit a functional wall blocking further rises in overall health care spending. It has become a zero-sum game. The inescapable ethical dilemma is that this single $850,000 treatment will mean that unknown others will receive $850,000 less in health care.

  • Excellent piece of writing, and exciting medicine. The price of the drug, and how that price it was determined, where the funding for research for the drug came from, to what extent Spark bore any costs or participated in that research, and whether those funding the research receive any share of profits from the enterprise, is worthy of a separate piece. With some digging, you may learn from early news reports that Spark Therapeutics was spun off late in the day to reap the commercial benefit of this, the first successful gene therapy, developed over the course of more than a decade by a small team of scientists at the University of Pennsylvania, and a retinal surgeon who developed the technique of delivery to the retina. Sometimes these true heros are given credit in these stories, but mostly not. But the most important point, from a health policy perspective, is how this therapy was developed, funded, priced, who stands to financially benefit, and whether the model helps, in any way, to sustain further research in this vital area.

  • Dr. Commander and his staff have been nothing short of the most patient, caring and competent group of medical professionals. I’ve been a patient of his for several years and he’s improved my visual abilities and quality to an amazing degree.

Sign up for our Daily Recap newsletter

A roundup of STAT’s top stories of the day in science and medicine

Privacy Policy