A recently published pre-clinical trial study tested EBT-001 a therapeutic against Simian Immunodeficiency Virus in rhesus macaques. This novel therapeutic was developed by scientists at the Lewis Katz School of Medicine at Temple University and published in the journal Gene Therapy.
SIV is a type of retrovirus that primarily infects certain species of non-human primates, including monkeys and apes. SIV is closely related to HIV (Human Immunodeficiency Virus), which affects humans. SIV infections can sometimes lead to simian AIDS in these animals, similar to how HIV can lead to AIDS in humans.
SIV has been studied extensively as a model for understanding the origins and transmission of HIV.
EBT-001 Effective at Different Concentrations
EBT-001 is a CRISPR-Cas9 gene editing therapy that is purported to remove SIV and its genes from all the reservoirs of the organism, as SIV just like HIV can hibernate in the body for years.
EVT-001 was incorporated into the AAV9 (adenovirus vector). EBT-001 transports SaCas9 (endonuclease) and two guide RNAs designed to precisely focus on various sections of the SIV genome, including the viral LTRs and the Gag gene (important structural gene).
The findings presented in this study show that when a single EBT-001 treatment is given through a vein at three different dose levels (1.4 × 10^12, 1.4 × 10^13, and 1.4 × 10^14 genome copies per kilogram), it effectively spreads throughout the body, targeting specific areas where the SIV virus is known to hide.
When the researchers gave the monkeys EBT-001 through a vein in different amounts, it spread to different parts of the body where the virus hides. They did not observe any unintended effects or abnormal health changes in the monkeys, and the monkeys returned to their normal weight after the treatment.
Importantly, the monkeys that received the two highest doses of EBT-001 showed better immune cell counts compared to those receiving standard antiretroviral treatment.
Overall, these findings confirm that the EBT-001 treatment is safe, spreads effectively, and can edit the virus’s genetic material within the body. This supports the ongoing development of CRISPR-based gene editing as a potential treatment for HIV in humans.
Current HIV treatment plans often involve lifelong antiretroviral therapy, which have side effects and require strict adherence. EBT-001’s success could potentially reduce the need for long-term ART, offering patients a more convenient and sustainable treatment option.
The observation of improved immune cell counts in the monkeys that received higher doses of EBT-001 suggests that this therapy might not only target the virus but also possibly boost the immune system’s response. This could potentially lead to better control of the virus and improved overall health.
While this study focused on SIV as a model for HIV, the techniques and insights gained from this research could have broader implications for treating other viral infections and genetic diseases through targeted gene editing.