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CRISPR eliminates HIV-1 infection in live animals

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Re: CRISPR eliminates HIV-1 infection in live animals

#121

Earlier quoted context omitted.

One factor is that by far the biggest profits are to be had in the US. Maybe you can get approved in Australia or Sweden, but that will pay negligible sums compared to getting into the US market.

Could try for market pressure - for example, that male birth control being developed in India we keep hearing about. I bet it'll get approved here faster than normal because of how desired it is.

Related anecdote: to this day, in Japan access to the Pill remains limited because of vague "safety" concerns, despite decades of use. However, when Viagra appeared, it was approved in months...

Re: CRISPR eliminates HIV-1 infection in live animals

#122
post #53
post #32

Back in 2011, there was an announcement from MIT about a new approach to a broad-spectrum antiviral that appeared to work.[1] This goes way beyond an AIDS-specific cure. But it was at MIT Lincoln Labs, which doesn't usually do bio. So the researcher moved to Draper Labs, but didn't get much funding. Then that funding ran out. Now the guy behind this is trying to get funding on Indiegogo.[3] The problem seems to be th…

This is the type of innovation we exchange for a sense of absolute security in having highly-vetted decade-long bio R&D and extensive backlogs at the FDA for approvals to sell products on the market. It's go big or go home because the regulatory risk/timeline is so extensive. Most of these things die quietly or never get started so we never really see the true costs of what it takes to push the pharma world forward.…

This is nowhere near the "This is getting tangled up in the FDA" phase. Funding speculative science is hard, and expensive, and this is the type of innovation we exchange for wanting more certain returns on investment, and nothing more than that.

Re: CRISPR eliminates HIV-1 infection in live animals

#123
post #32

Back in 2011, there was an announcement from MIT about a new approach to a broad-spectrum antiviral that appeared to work.[1] This goes way beyond an AIDS-specific cure. But it was at MIT Lincoln Labs, which doesn't usually do bio. So the researcher moved to Draper Labs, but didn't get much funding. Then that funding ran out. Now the guy behind this is trying to get funding on Indiegogo.[3] The problem seems to be th…

If he's not getting funding perhaps his work doesn't hold up under scrutiny? I'm a geologist and biology is greek to me so I don't know what to make of it.

This is correct. Everybody I know in the biz has said his work doesn't pass a wide range of sniff tests.

Re: CRISPR eliminates HIV-1 infection in live animals

#124
post #2

Gene therapy (and CRISPR) is ultimately going to eliminate most if not every ailment/issue/genetic problem that we have, including aging and eye problems. Very thankful we are reaching the point where we can go into our cells and fix ourselves.

I dedicated a significant fraction of my life to the idea that gene therapy would become a viable medical treatment. It seems unlikely to really become a high-impact treatment.

Being able to change DNA in cells is one thing; actually being able to show your treatment does what it's supposed to and didn't have negative effects is hard. There are a limited number of diseases where gene therapy should work great, but there are a wide range of others where it won't, until solve multiple grand-challenge class problems.

Re: CRISPR eliminates HIV-1 infection in live animals

#125

Earlier quoted context omitted.

I almost wonder if running your own space program is cheaper. As in the amount of regulatory BS you have to go through due to the monopolistic behavior of the large pharmaceutical companies...

And yet, if you're not really planning on selling the techniques you find on the open market could you save yourself a lot of time by ignoring the FDA?

There's a metric ton of other regulations, at least in the US, mandating how any human level research is done. Depending on what one is doing, it's not necessarily less annoying

Re: CRISPR eliminates HIV-1 infection in live animals

#126

Earlier quoted context omitted.

I almost wonder if running your own space program is cheaper. As in the amount of regulatory BS you have to go through due to the monopolistic behavior of the large pharmaceutical companies...

And yet, if you're not really planning on selling the techniques you find on the open market could you save yourself a lot of time by ignoring the FDA?

There's a metric ton of other regulations, at least in the US, mandating how any human level research is done. Depending on what one is doing, it's not necessarily less annoying

Re: CRISPR eliminates HIV-1 infection in live animals

#127
post #60

Earlier quoted context omitted.

I do agree, but that is just the good news. In other news although we can harbor optimistic thoughts for scientists, medical professionals, lawmakers and maybe even politicians all around the globe to do the "right" thing to preserve human life in a currently recognizable form, but there is not a force on heaven or earth that is going to stop parents from trying to give their children every advantage.

> to do the "right" thing "right" according to whom? nature itself doesn't believe that

Despite academic/political/military/banking/corporate alignment against fundamental morals, "right" appears to be in agreement with nature/natural selection. The States had a more "right" society founded om constitutional and civil rights that was a big part of its climb from an English colony to the world power in less than 150 years after the constitution was written.

The reason people probably have strong convictions about right or wrong is most likely because of nature, not despite it. Groups that had this genetic trait built tribes, societies that had greater success in the long term.

In the short term, individuals, or even groups of individuals, can be successful by acting amorally, but this comes as a cost to their group's long term success--the cancer analogy.

Right now there is limited competition between groups in the world. Most of what we should be competing against is the coming extinction event when something else out there gets a whiff of all the artificial elctromagnetic radiation. Probably the reason we hear so little in the cosmos is because it is not a very competitive strategy.

Re: CRISPR eliminates HIV-1 infection in live animals

#128
post #32

Back in 2011, there was an announcement from MIT about a new approach to a broad-spectrum antiviral that appeared to work.[1] This goes way beyond an AIDS-specific cure. But it was at MIT Lincoln Labs, which doesn't usually do bio. So the researcher moved to Draper Labs, but didn't get much funding. Then that funding ran out. Now the guy behind this is trying to get funding on Indiegogo.[3] The problem seems to be th…

The expensive part of getting a drug to market isn't proving that it can kill cells in a petri dish. Lots of stuff can do that, bleach, hydrogen peroxide... The expensive part is the clinical trial. You try out the compound in the chemical woodchipper that is the human body, and see what happens. Almost all drugs fail at this point: http://blogs.sciencemag.org/pipeline/archives/2017/01/23/i-d... >The timing of this r…

This is all true.

The flip side of this, however, is that "trial phase failure" does not conclude "ineffective biologic." There are many other variables to Clinical Trials, including flaws in trial design, time spent and difficulty in operations, and biased reporting:

https://well.blogs.nytimes.com/2013/01/14/clinical-trials-fl...

http://www.outsourcing-pharma.com/Preclinical-Research/Parki...

https://www.iths.org/blog/news/avoid-common-biostatistical-f...

Re: CRISPR eliminates HIV-1 infection in live animals

#129
post #55

From the abstract: Intravenously injected quadruplex sgRNAs/saCas9 AAV-DJ/8 excised HIV-1 proviral DNA and significantly reduced viral RNA expression in several organs/tissues of Tg26 mice. In EcoHIV acutely infected mice, intravenously injected quadruplex sgRNAs/saCas9 AAV-DJ/8 reduced systemic EcoHIV infection, as determined by live bioluminescence imaging. Can a mod change the title to reflect the findings? The or…

That abstract looks completely consistent with the submitted title. I don't see any reason to change it.

"Eliminates" is a very loaded term in viral therapy. This paper doesn't show elimination of virus, it shows that the integrated viral genome can be excised in -some- cells by administration of adeno-associated virus harboring the CRISPR system.

Re: CRISPR eliminates HIV-1 infection in live animals

#130
post #32

Back in 2011, there was an announcement from MIT about a new approach to a broad-spectrum antiviral that appeared to work.[1] This goes way beyond an AIDS-specific cure. But it was at MIT Lincoln Labs, which doesn't usually do bio. So the researcher moved to Draper Labs, but didn't get much funding. Then that funding ran out. Now the guy behind this is trying to get funding on Indiegogo.[3] The problem seems to be th…

Given billionaires running their own space programs it makes me wonder why there aren't more billionaires running biologic research companies. Between life extension and immunity to (or protection from) really pathological conditions it seems like their interests would be aligned with folks like this.

Not just billionaires.

One of my besties is directly funding early stage Lyme Disease research. Directly to the lab and researchers. Bypassing orgs, foundations, panels, etc.

He has a vested interest in accelerating the process and has already benefitted from their findings.

This direct funding model will become a significant strategy, as it becomes ever easier to find and connect interested parties.

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