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Haemophilia A trial results 'mind-blowing'

bbc.com

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Re: Haemophilia A trial results 'mind-blowing'

#61

Post by jessriedel »

Chriky wrote: post #29
jessriedel wrote: post #23

Earlier quoted context omitted.

Thanks for sharing! Can I ask what considerations went into your decision? Will you be pursuing this now that there is more evidence that it's effective?

I mean, obviously I regret it now, but at the time I had no idea it would work. I'm sure the next trial will be extremely oversubscribed! I think my trial was actually for a low dose version to test safety of the vector and it didn't have any clinical effects. Its quite incredible though, there's only a few thousand people in the UK with haemophilia - this trial just cured like 0.5% of them

Yea, I definitely was not suggesting it was a mistake with the info you had. Most clinical trials don't work.

Thanks again for the info. Best of luck!

Re: Haemophilia A trial results 'mind-blowing'

#62

Post by fgimenez »

aaavl2821 wrote: post #47
alextheparrot wrote: post #5

What a fantastic gateway to show how gene therapy can completely resolve diseases. Many diseases are as simple as your eyes being brown vs blue - you simply have or lack particular proteins. I couldn’t find it on a cursory reading, does anyone know what vector they used to achieve liver sinusoidal cell specificity? My understanding is that many gene therapy studies have used blood cells, which can be pulled out of th…

AAV is the typical vector used for gene therapy. viruses are useful because they have evolved to insert foreign DNA into host cells. nothing humans can conceivably engineer has come close yet to the ability of viruses to do this however viruses have also evolved a lot of bad qualities that cause disease. AAV is nice because 1) it is not known to cause disease, 2) only causes a mild immune response (so the body doesnt…

I sincerely think we are in the gene therapy renaissance. Delivery has always been an issue, but there are new tricks coming out with regard to cell-specific targeting. We can piggy back off the work done for RNA-based therapeutics (mRNA, ASOs, RNAi).

As for the CNS - Voyager therapeutics has had some great readouts in September in gene therapy for Parkinson's. More data coming out in Q1 to see second part of study.

Re: Haemophilia A trial results 'mind-blowing'

#64

Post by ericb »

alextheparrot wrote: post #5

What a fantastic gateway to show how gene therapy can completely resolve diseases. Many diseases are as simple as your eyes being brown vs blue - you simply have or lack particular proteins. I couldn’t find it on a cursory reading, does anyone know what vector they used to achieve liver sinusoidal cell specificity? My understanding is that many gene therapy studies have used blood cells, which can be pulled out of th…

Is the virus transmissible?

Re: Haemophilia A trial results 'mind-blowing'

#65

Post by mrfusion »

aaavl2821 wrote: post #47
alextheparrot wrote: post #5

What a fantastic gateway to show how gene therapy can completely resolve diseases. Many diseases are as simple as your eyes being brown vs blue - you simply have or lack particular proteins. I couldn’t find it on a cursory reading, does anyone know what vector they used to achieve liver sinusoidal cell specificity? My understanding is that many gene therapy studies have used blood cells, which can be pulled out of th…

AAV is the typical vector used for gene therapy. viruses are useful because they have evolved to insert foreign DNA into host cells. nothing humans can conceivably engineer has come close yet to the ability of viruses to do this however viruses have also evolved a lot of bad qualities that cause disease. AAV is nice because 1) it is not known to cause disease, 2) only causes a mild immune response (so the body doesnt…

Why can’t you take immunosuppressants taking the treatment the second time around?

Re: Haemophilia A trial results 'mind-blowing'

#66

Post by king07828 »

This is awesome. I have Crohn's and am looking forward to an eventual cure.

One thing I don't understand is why big pharma and VCs seem to focus on rare diseases instead of a gene therapy for changing eye color or hair color. With eye/hair color gene treatments, patients could undergo the therapy multiple times (if desired) and the pool of patients is all humans (not just rare diseases sufferers). This allows for amortization of the R&D expense over substantially more treatments. Plus the advances in gene therapy technology could then be applied to therapies for rare diseases

Re: Haemophilia A trial results 'mind-blowing'

#67

Post by aaavl2821 »

fgimenez wrote: post #62
aaavl2821 wrote: post #47

Earlier quoted context omitted.

AAV is the typical vector used for gene therapy. viruses are useful because they have evolved to insert foreign DNA into host cells. nothing humans can conceivably engineer has come close yet to the ability of viruses to do this however viruses have also evolved a lot of bad qualities that cause disease. AAV is nice because 1) it is not known to cause disease, 2) only causes a mild immune response (so the body doesnt…

I sincerely think we are in the gene therapy renaissance. Delivery has always been an issue, but there are new tricks coming out with regard to cell-specific targeting. We can piggy back off the work done for RNA-based therapeutics (mRNA, ASOs, RNAi). As for the CNS - Voyager therapeutics has had some great readouts in September in gene therapy for Parkinson's. More data coming out in Q1 to see second part of study.

i agree, the hemophilia data looks really good, and avexis' spinal muscular atrophy product looks great as well. i dont know much about voyager but just looked at their press release for the study; looks like they are delivering a gene to increase dopamine production? do you know if this could be a disease altering therapy or just a "better" levodopa?

its still pretty hard from what i can tell to deliver oligos to specific cells. delivery has been a huge challenge for ASOs, mRNA etc. viral vectors and autologous cell therapies have been used but have limitations, and antibody-tagged "targeted" nanoparticles have also been tried with varying degrees of success.

theres been some really clever developments in delivering oligos with cationic lipid nanoparticles to macrophages. biontech has an approach where theyve made nanoparticles that are naturally taken up by macropinocytosis by dendritic cells and macrophages. if you get the overall charge of the nanoparticle right (net negative i believe), the dendritic cells migrate to the spleen (rather than the lung with positive charge), where they can present antigen encoded with mRNA to t cells. they also have some interesting mRNA tech that increases the transfection efficiency, as it is hard to prevent oligos from getting degraded in lysosomes before they are translated. genentech paid them $310M upfront for a preclinical asset

Re: Haemophilia A trial results 'mind-blowing'

#68

Post by Kalium »

king07828 wrote: post #66

This is awesome. I have Crohn's and am looking forward to an eventual cure. One thing I don't understand is why big pharma and VCs seem to focus on rare diseases instead of a gene therapy for changing eye color or hair color. With eye/hair color gene treatments, patients could undergo the therapy multiple times (if desired) and the pool of patients is all humans (not just rare diseases sufferers). This allows for amo…

I can think of a couple of reasons. First, they think of themselves as companies that address diseases. They are concerned with the ill, rather than people seeking cosmetic changes.

Second, I can see some potential for major backlash. Literally going into the business of designer babies? Ethically tricky. Doing it only for the rich, because it'll be incredibly expensive at first? "Tricky" doesn't begin to cover it. Public policy in first-world countries is not always noted for being far-sighted and even-handed, and a public backlash could easily shut down all gene therapies.

Re: Haemophilia A trial results 'mind-blowing'

#69

Post by pishpash »

king07828 wrote: post #66

This is awesome. I have Crohn's and am looking forward to an eventual cure. One thing I don't understand is why big pharma and VCs seem to focus on rare diseases instead of a gene therapy for changing eye color or hair color. With eye/hair color gene treatments, patients could undergo the therapy multiple times (if desired) and the pool of patients is all humans (not just rare diseases sufferers). This allows for amo…

How much can you charge for cosmetic changes that can be easily done by less expensive means? I'm sure they've done the math.

Re: Haemophilia A trial results 'mind-blowing'

#70

Post by frgtpsswrdlame »

king07828 wrote: post #66

This is awesome. I have Crohn's and am looking forward to an eventual cure. One thing I don't understand is why big pharma and VCs seem to focus on rare diseases instead of a gene therapy for changing eye color or hair color. With eye/hair color gene treatments, patients could undergo the therapy multiple times (if desired) and the pool of patients is all humans (not just rare diseases sufferers). This allows for amo…

>I have Crohn's and am looking forward to an eventual cure.

Check this out, it has a lot of interesting information: http://www.crohnsmapvaccine.com

Could be interesting although obviously still in phase 1.

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