Gene therapy: “It provides insight into the mechanisms of life itself”

Professor Robert MacLaren on the exciting potential of CRISPR gene editing and patients seeing stars for the first-time following treatment

Blue DNA

Professor Robert MacLaren, of Oxford University, is leading work to develop gene therapies for inherited retinal conditions through Nightstar Therapeutics. The company was acquired in 2019 by US biotechnology company, Biogen, for $US 877 million.

What is the story behind the name of your gene therapy company, Nightstar Therapeutics?

One night I was at my computer and a patient emailed me to say he could see a night star. This was a positive thing. I said, ‘If you are still there can you go and check which eye it was.’ He came back and said, ‘It is definitely the eye that I had the gene therapy in.’

Subsequently, when we developed the gene therapy company, the name NightStar was an obvious one because since then several of our patients have described seeing night stars after gene therapy. It symbolizes looking up at the heavens and hope for the future, but there is also that practical element of achieving better vision at night.

What do you find rewarding about the gene therapy research you are involved in?

Although I am an ophthalmologist treating patients, when we are looking at the mechanisms of the disease, we get right down to the molecular level, looking at how the DNA is assembled and how it is processed.

It is very exciting as a physician in any specialty to be able to treat disease at the genetic level. Ultimately if the disease occurs at the genetic level and you treat it there, you are treating the origin of the disease itself. If you are just giving drugs to try and correct the genetic disease or compensate for it, it may help but that is not a real treatment. It provides insight into the mechanisms of life itself seeing how DNA is processed and how the genes evolve.

What are the benefits of CRISPR gene editing?

You can treat potentially any disease with that, whereas gene replacement therapy is limited to small genes. This is still good but ideally if you correct the mutation it is a better treatment than replacing the whole gene.