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Biotech Medicine

FDA Approves Human Clinical Trials of a Possible CRISPR-Based HIV Cure (fiercebiotech.com) 44

Posted by EditorDavid from the fingers-crossed dept.
"A CRISPR-Cas9 gene editing technology that has shown promise in clearing HIV from mice is headed into human testing," reports Fierce Biotech: We don't like to throw the word "cure" around here. But Excision BioTherapeutics thinks the therapy could replace standard-of-care retroviral therapy, which keeps HIV from replicating but does not remove it from the body. That means patients stay on the treatment, which can cause serious side effects and affect quality of life. Now with the start of human testing, the real path to see if this new and lauded tech can accomplish this really begins.

HIV integrates its genetic material into the genome of a host cell, meaning available therapies just can't remove it. A team of scientists at Temple University and the University of Nebraska Medical Center managed to remove the virus completely from mice during preclinical testing using a combination of CRISPR and antiretroviral therapy. They also found no adverse events that could be linked to the therapy in the study, published back in 2019... EBT-101 has since been tested in nonhuman primates, which showed it reached every tissue in the body where HIV reservoirs reside.

Excision licensed the therapy from the universities with a goal of moving it into clinical trials. Now, the FDA is on board. The biotech plans to initiate a phase 1/2 clinical trial later this year, according to the statement.

The technology used by Excision was licensed from the lab of famed CRISPR pioneer Jennifer Doudna. The company is also working on similar treatments for other viruses, including herpes and hepatitis B.
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FDA Approves Human Clinical Trials of a Possible CRISPR-Based HIV Cure More

FDA Approves Human Clinical Trials of a Possible CRISPR-Based HIV Cure

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  • no adverse events (Score:4, Funny)

    by nospam007 ( 722110 ) * on Saturday September 18, 2021 @02:40PM (#61808441)

    "They also found no adverse events that could be linked to the therapy in the study, "

    Not a single mouse complained.

  • How long does it take for the CRISPR payload to enter and modify all the cells in the body?

    • How long does it take for the CRISPR payload to enter and modify all the cells in the body?

      In the clinical trial, patients will be kept on antiretrovirals for three months after the EBT-101 injection.

  • Triple strategy (Score:5, Interesting)

    by backslashdot ( 95548 ) on Saturday September 18, 2021 @03:03PM (#61808513)

    3 methods are needed simultaneously, because HIV mutates so much.

    1. Excision of virus from genomic DNA of every cell in the body (this method isnâ(TM)t perfect, because some provirus will inevitably not be excised.)
    2. Immunity mediated by CD8 t-cells, such as the Louis Picker vaccine does. This is so that if any viruses crop up they can be destroyed. Normally vaccines rely on CD4 t-cells, but CD4 is what HIV targets to infect .. oops.
    3. Gene knockout of CCR5 and CXCR4 receptor from cells. Luckily neither receptor appears to have any important function. CCR5 is the most important one to knock out and some people are lucky enough to be immune to HIV because they were born without CCR5. The only two people to have been determined to be fully cured of HIV received a CCR5 deficient stem cell transplant.

    I donâ(TM)t see a viable cure without those 3 angles simultaneously. Each one individually is susceptible to mutation. For example the excision targeting will miss cells that have a different virus DNA sequence than the exact sequence being targeted. And that is assuming it can even get into every cell (it cannot.)

    • Cue regular expression CRISPR upgrade.

      • Doctor A: OH MY GOD! What happened to this poor man?!
        Doctor B: The regular expression was perfect, I swear!
        Doctor A: WHERE IS HIS SKIN?!
        Doctor B: ...
        Doctor A: ...
        Doctor B: OK, it might be have had a slight flaw.

  • What are they doing, somehow editing every cell in the body to remove or inactivate the gene for CCR5?

  • I've talked to a few of them to find out where their view actually comes from.

    And actually, it's just a simple trust issue due to feeling forced into things against their will. Which stems from them having neither the education or resources to check things for themselves, nor the experiences with authorities that would make them trustworthy. Which, honestly, are completely understandable.
    Many are quite open to learning how it all works. IFF you treat them with respect, and approach it the right way. It may

    • This isn't a vaccine, fwiw.

      • I think the anti-vaxers will be fine with this, as you say, not a vaccine. I don't see the anti-vaxer crowd refusing care in ER's around the country. They get sick, and welcome modern medical care. Ironic as it is.

  • Wow. First real shot at a cure. (Score:3)

    by WindBourne ( 631190 ) on Saturday September 18, 2021 @05:57PM (#61808873) Journal
    Ok, this tech came about after I got my first degree (microbio/genetic engineering) so I just had to look this up:
    Apparently, this gRNA(guide RNA) works with enzymes to "delete, insert or otherwise alter the targeted RNA or DNA" [wikipedia.org]
    This is even more on how gRNA works. [addgene.org]

    This is probably the FIRST real shot at a cure. But this leaves open multiple questions:
    1) how does this get to every instance of HIV inside of the chromosomes? There are a lot that will be hidden deep inside of multiple sites.
    2) This needs a sequence of basepairs to trigger the cuts. IOW, it does not cut JUST the HIV, but it actually cuts based on a specific set of base pairs that will be at the start/end of a HIV. What are the odds of those being in other genes?
    3) a chromosome can/will contain multiple instances of HIV. What would prevent this from cleaving the start of instance 1, and cleave the end of instance 2, removing all of the genetic material in-between. Keep in mind that it is not just genes in there, but sequence that control the tertiary structure of the DNA which helps to decide which genes to express.

    I can see in a small set of chromosomes like a bacteria or starfish, etc. that it would be possible to cleave out and have an idea, but it seems like this will cleave large amounts of genes. Of course, the answer might be that the cells that are discombobulated i.e. cut wrongly, will simply die and be absorbed.

    • 1) how does this get to every instance of HIV inside of the chromosomes? There are a lot that will be hidden deep inside of multiple sites.

      They necessarily don't need it to, HIV-excised cells apparently cannot be re-infected easily .. so as long as there is a population of HIV resistance cells, that might be OK I guess. (Reference: https://www.ncbi.nlm.nih.gov/p... [nih.gov] ) . I've never seen 100% transfection with CRISPR in vitro, let alone in-vivo .. so I doubt anyone is expecting for 100% excision.

      2) This needs a sequence of basepairs to trigger the cuts. IOW, it does not cut JUST the HIV, but it actually cuts based on a specific set of base pairs that will be at the start/end of a HIV. What are the odds of those being in other genes?

      Zero. Ok, damn near zero. That said, it will probably go off target (not because of a duplicate sequence, but other reasons) in a small percentage of cel

  • The vast majority of clinical trials fail. 70% at stage II.

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