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Haemophilia A trial results 'mind-blowing'

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Re: Haemophilia A trial results 'mind-blowing'

#131
post #26

I hope they can expand this to other genetic problems. There are hundreds of genetic issues science knows about. Just off the top of my head I can think of high cholesterol, cystic fibrous, and sickle cell anemia. I know there are others, but I'm just a layman not a medical doctor. Those who have any of the above would like to be cured.

The trick right now is in knowing exactly what gene to express. It's like the Ionis Huntington's trial, just one well-known gene to suppress (albeit in cerebrospinal fluid, so the stage 1 trials were interesting). And of course, the first-wave funding will go to stuff that absolutely, positively will kill a person stone dead.

As soon as there's multiple targets it gets more complicated. Familial Hypercholesteroleamia could be any of three genes in it's least complex setup. I wouldn't be surprised to see a scam "therapy" marketed for it by the end of the year...

Re: Haemophilia A trial results 'mind-blowing'

#132
post #53

Earlier quoted context omitted.

Thankfully artificial pancreas are looking like a good option until we can get to curing type 1. Not only does it save the number of shots like pumps we have today, but it can measure the sugar level and keep the swings down to a minimum. http://www.jdrf.org/research/artificial-pancreas/

yeah artificial pancreas seems to be the most promising near-term development. the field has suffered from a lack of funding for translational research as VCs have been focused on other diseases, but hopefully that changes. semma (a startup turning stem cells into beta cells) raised $114M last week

Why are they focused on other diseases? Diabetes is hardly a rare disease, nor one with a low impact on its sufferers. So if what you say is true, what is it that's broken about the market for diabetes treatment?

I often hear it said that problems such as this are caused by cures being less profitable compared to drugs, because drugs are purchased repeatedly, cures only once. But competition is supposed to solve that problem. Is inappropriate regulation the real problem?

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