Three people with inherited diseases successfully treated with CRISPR • Hacker News Board

Re: Three people with inherited diseases successfully treated with CRISPR

#71

Post by perl4ever » Wed, Jun 17, 2020, 3:21 AM UTC

DrAwdeOccarim wrote: post #68

perl4ever wrote: post #64

Earlier quoted context omitted.

I've heard of those, but it's interesting to me that you list those and not, say, Illumina.

I mean, they are a pick and shovel play. They don't make drugs? Plus, sequencing is great but it's getting saturated. Plus, ideas like direct sequencing using nanopores and chemical modification of nucleic acid to make the nanopore signal stronger may disrupt.

Reading a lot of 10-Ks, you get the impression that many if not most biotech companies want to appeal to investors who find "pick and shovel" companies appealing. Even companies that are basically drug companies often try to portray themselves as having a revolutionary "platform".

I'm not saying your attitude is obviously wrong, but there must be a lot of people whose imagination is captured by making tools and the potential of that to change the world. The development of a tool like optogenetics, for instance, makes me feel like amazing things can be accomplished. It's leverage.

Re: Three people with inherited diseases successfully treated with CRISPR

#72

Post by DrAwdeOccarim » Fri, Jun 19, 2020, 9:42 AM UTC

perl4ever wrote: post #71

DrAwdeOccarim wrote: post #68

Earlier quoted context omitted.

I mean, they are a pick and shovel play. They don't make drugs? Plus, sequencing is great but it's getting saturated. Plus, ideas like direct sequencing using nanopores and chemical modification of nucleic acid to make the nanopore signal stronger may disrupt.

Reading a lot of 10-Ks, you get the impression that many if not most biotech companies want to appeal to investors who find "pick and shovel" companies appealing. Even companies that are basically drug companies often try to portray themselves as having a revolutionary "platform". I'm not saying your attitude is obviously wrong, but there must be a lot of people whose imagination is captured by making tools and the p…

Yea sure, I love pick and shovel plays. I think they're the ones you make the easy money from like ThermoFisher, Becton Dickinson, EMD/Milipore, Sigma-Aldrich, etc. I was thinking more like FAANG companies that are huge, pay awesome, and make the final product, though I guess you could say advertising is a bit of a pass-through income generator.

Maybe you and I have different definitions of 'platform' company? I define as a way to iterate to get new products, like Regeneron uses their humanized mice to crank-out mAbs, or Genetech/Amgen use recombinant molecular biology to make mAbs, or Vertex uses their in vitro ion channel conductance screening method to make new structure correctors for CFTR...

Re: Three people with inherited diseases successfully treated with CRISPR

#73

Post by enchiridion » Sat, Jun 20, 2020, 4:38 PM UTC

rnet85 wrote: post #65

refurb wrote: post #63

Earlier quoted context omitted.

I’m not a doctor, but I would say yes, you’d lose any acquired immunity since you kill off all the memory b-cells that store that information. But you’d also gain the donor’s immunity, and if they’ve been vaccinated, the recipient would have that immunity as well.

That is not correct. Trained B cells are predominantly stored in lymph nodes. You also don’t gain host immunity by marrow transfer.

So B cells are what responsible for learned immunity?

Re: Three people with inherited diseases successfully treated with CRISPR

#74

Post by Paperweight » Sun, Jun 21, 2020, 5:27 AM UTC

shock wrote: post #9

Paperweight wrote: post #8

But will the drug approval agencies allow genetically engineered therapies for anything disruptive? For example, they've been blocking the caries vaccine for decades.

> blocking the caries vaccine for decades Please give more details.

It's a simple model - make a genetically engineered S. mutans that slightly out-competes the existing, but without generating the lactic acid waste products that softens tooth enamel. https://en.wikipedia.org/wiki/Caries_vaccine#Attempts_using_...

Dr. Jeffrey D. Hillman invented it in the 1970s-1980s at Harvard under NIH grants (https://grantome.com/grant/NIH/R01-DE004529-10). Swab inoculation and you're done. It worked perfectly in animals and humans.

In the 1990s, he founded Oragenics, to try to commercialize it. The FDA gave them the runaround for about 20 years before they finally mysteriously gave up. In 2016, he got a 17-year patent, so I guess it'll be shelved until 2033 at least.

There's something fundamentally wrong with this picture. Why should a simple application of genetic engineering from the 1980s take 50+ years to make it to market? It's either a complete scam or a conspiracy.