More Gene Therapy News

[monkeybus]

Alright, it's not AMN related, but some of these gene therapies are getting to be commonplace treatments. Has to be good news for the likes of us.

theguardian.com/science/201...

[Cambridge_Chris]

Do you follow the company BlueBird bio? They are developing/have developed a gene-therapy for ALD (*they* say specifically for cerebral ALD) bluebirdbio.com/research-de.... The problem will be the price, in the UK at least; I'm not sure that the health service is going to want to fork over something-in-the-region of half a million for a single treatment!

[monkeybus in reply to Cambridge_Chris]

Yes, that Lenti-D is stem cells,am I right?

Half a Mil! I try and be positive, but we got no chance at those prices. Though I'll stand by my idea of doing a crowd funding with one of us as a guinea-pig.

That said, it's just an infusion. The price will come down, but there is simply no rush with all this. Those HIV drugs, and even the contraceptive pill hit the market double quick. A huge demand from white, middle-class folks tends to grease the wheels somewhat.

ncbi.nlm.nih.gov/m/pubmed/2...

ncbi.nlm.nih.gov/pmc/articl...

[Cambridge_Chris in reply to monkeybus]

Yes, that's the one.

I like your optimism! There's going to have to be some agreement between the drug companies and healthcare payer, though, because it's a very different model; a one-shot cure is the opposite of drugs you take, and pay for, for years and years.