Gene therapies are coming, but I’m not sure who will be able to afford them any time soon:
New gene therapy, to be priced at $4.25 million, has already transformed children’s lives
A new gene therapy for an ultra-rare disease will have a wholesale cost of $4.25 million, making it the world’s most expensive drug.
The one-time treatment, Lenmeldy, won US regulatory approval on Monday to correct the underlying cause of a hereditary condition called early-onset metachromatic leukodystrophy, or MLD.
MLD is a fatal disease in which infants sometimes start to lose the ability to walk and talk. Orchard Therapeutics said the drug’s price “reflects its clinical, economic and societal value” in a statement Wednesday.
I will throw out a guestimate, but nobody really knows. I would say:
5 to 10 years for RCTs for consenting/willing adults (for age-related therapies) - you could argue that MiniCircle is alreadying doing this in Roatan.
10 to 20 years, but perhaps more likely 30 years for the reasonable cost aspect (but again, MiniCircle is doing their preliminary non-permanent “gene therapy” for $30,000 or so, so we’ll see.
The issue is that there are so many uncertainties.
About 20 years ago the gene therapy startup scene was hot in the SF Bay Area, companies like Avgenics and many others were getting tons of funding and everyone though the future was bright and happening quickly.
I had friends in the gene therapy business here in the SF Bay Area 20 years ago when a FDA-approved gene therapy trial that was taking place went horribly wrong. A young 19 year old man who was participating in the trial died when his body responded very negatively to the viral vector they were using. His death hit all the major newspapers in the USA at the time, front page. It killed the entire industry virtually immediately. All the funding went away, and all the startups died in pretty short order. And the venture money would not look at the field for a long time… like 15 years went by before they would start investing again.
We have some gene therapy biologists here in our forums so perhaps they can comment.
But I think that most of these gene therapy companies today are still using viral vectors (the shell of a virus that can integrate into our DNA) to get the gene segments integrated, but there are still many problems with this as these are random integrations. Since you don’t know where the newly inserted gene is going, it could disrupt an existing well-functioning gene. I don’t know how well the work is going for targeted gene therapies, so that you can specifically insert the new gene segment into a targeted spot of the genome. perhaps someone else here has current knowledge in this area.
You also have random political factors that can hugely impact the work of biotech startups; when George Bush was elected president he shut down all the embryonic stem cell research in the country virtually overnight. Could a similar situation happen with gene therapy if it seemed to be politically convenient? Its certainly possible.
While a lot of good progress is being made in gene therapy now, it seems to me there are still significant hurdles to overcome to make it more broadly available and less expensive. I believe some of the hurdles are things like doing targeted delivery of the genes (vs. random), optimal delivery techniques to maximize transfection rates/ integration in the right areas of the body to be effective, and navigating the intellectual property landscape to get access to the different technologies a given company may need to use to delivery the therapy.
