Exavir Therapeutics, a biotechnology company dedicated to eliminating HIV and other viral infections, recently released the results of a groundbreaking and promising preclinical study that highlights what may be the next step to curing HIV: literally cutting the virus from an individual’s DNA where it has incorporated itself into the genome. This “excision” is accomplished through the use of lipid nanoparticle-delivered CRISPR-Cas9. For those unfamiliar with this technology, CRISPR-Cas9 is effectively a biological “machine” that allows for the cutting and removal of specific sections of DNA. The lipid nanoparticle (or LNP) serves as a means of delivering CRISPR-Cas9 to the DNA so it can enter and begin cutting away the virus.
In simpler terms, think of the CRISPR-Cas9 as a pair of scissors. While they’re very good at cutting, they still need a hand to guide them. In this metaphor, RNA is the hand that shows the CRISPR-Cas9 scissors where to go and where to cut. Both of them are housed inside the LNP, which is essentially a small ball of fat that acts like a box built to hold the scissors and their instructions. It’s also what allows CRISPR-Cas9 to enter the DNA where it can take over, target the virus, and start cutting.
Once CRISPR-Cas9 has completed its task, researchers look at the DNA to see how much HIV remains — and the current results are very encouraging, to say the least. “Our system is demonstrating near complete HIV elimination in vitro across several assays, with no detectable o -target edits,” Howard Gendelman, scientific cofounder of Exavir Therapeutics and professor in the Department of Pharmacology at the University of Nebraska Medical Center, said in a press release. What this means is that, in a series of tests, the virus was reliably and effectively removed from the DNA because CRISPR-Cas9 and RNA were able to accurately target the virus.
According to Exavir, this technology resulted in “up to 100 percent suppression of [the] virus,” adding that its findings support that this treatment, used as antiretroviral therapy, has the potential to completely eradicate HIV.
Without question, this data marks an incredible advancement in the fight against HIV. That said, it’s still many years away from being approved by the Food and Drug Administration for use, as it’s still very early in the research and development process. The data comes from research conducted using cell lines that, while derived from humans, aren’t exactly representative of people. In other words, the process still needs a great deal of further study. Ultimately, these results serve as a very encouraging proof of concept — and one we will have to follow closely to see how it develops in the coming years.