Jim Allen:
General reminder for those living with HIV regarding posting in this Forum section: https://forums.poz.com/index.php?topic=11292.0
Jim Allen:
POZ.com article in full: https://www.poz.com/article/crispr-gene-editing-removes-virus-cells
In Brief:
--- Quote ---A single injection of a novel CRISPR gene-editing therapy known as EBT-100 safely and effectively removed simian immunodeficiency virus (SIV)—a virus related to HIV—from viral reservoirs in monkeys, according to a report in Nature Gene Therapy. The first human trial of the EBT-100 started last year.
“The long timeframe of the study and the use of high doses of the gene-editing construct help confirm the safety of EBT-001,” lead study author Tricia Burdo, PhD, of the Lewis Katz School of Medicine at Temple University, said in a news release. “Our preclinical work in non-human primates was essential for allowing us to establish the criteria for applying EBT-101 in clinical studies and enabling the [Food and Drug Administration] authorization for an HIV-specific gene-editing therapy to move forward.”
Senior investigator Kamel Khalili, PhD, of Temple University, and colleagues have been studying gene therapy to cure HIV for more than a decade. In 2014, they published a groundbreaking study showing that a CRISPR-Cas9 tool could delete a segment of integrated proviral DNA that includes the gene for HIV’s Gag protein, which is necessary for viral replication. A study published in 2019 showed that the technique could cut out integrated HIV genes and clear latent viral reservoirs in humanized mice. The next year, an early study in monkeys showed that the CRISPR tool excised segments of integrated SIV from blood cells and viral reservoir tissues.
“Our study supports safety and demonstrates evidence of in vivo SIV editing of a CRISPR gene-editing technology aimed at the permanent inactivation of virus in a broad range of tissues in a large, preclinical animal model, using a one-time injection of the treatment,” Khalili said. “The outcome of the preclinical model set the stage for the ongoing clinical trial of EBT-101.” --- End quote ---
Jim Allen:
https://www.poz.com/article/crispr-hiv-gene-therapy-looks-safebut-work
--- Quote ---EBT-101, a novel CRISPR-based gene-editing therapy from Excision BioTherapeutics, was not associated with any serious adverse events in the first three treated study participants, researchers reported last week at the European Society for Gene & Cell Therapy annual meeting in Brussels. But the presentation was frustratingly short on data about whether the treatment works to control HIV.
According to the company, study participants will be followed for 48 weeks after EBT-101 administration, and all eligible participants will be assessed for sustained viral suppression after stopping antiretrovirals in an analytical treatment interruption starting at week 12. After the initial study, they will be enrolled in a long-term follow-up study (EBT-101-002).
The first participant who received EBT-101 in the summer of 2022 is already well past the 12-week mark when the treatment interruption should have commenced, but Excision has not provided any further information about his status. The company said in its press release that additional data would be presented in 2024.
While these initial results appear promising, it is far too soon to say that a functional cure for HIV is on the horizon.
“Scientists tell me that this is going to be part of a cure some day. And I shrug my shoulders and say, ‘Here we go again,’” long-term survivor and advocate Matt Sharp told the San Jose Mercury News. “Now we just have to get the research done. We’ve got to have hope, because the epidemic isn’t over.” --- End quote ---
So, basically, there is no substantial update. I see that some other popular media outlets have been publishing misleading headlines this past week; I suppose it is the same old story: Publish whatever sells papers, gets clicks, improves your stock position or gets you funding, and the truth and damage caused be dammed.
gorka:
not sure about this group but number of groups are gearing up for cell therapy or gene therapy approach using crispr. it has been very succesful for some cancers so far. hoping it will eventually work for hiv