The FDA has authorized a company trying to use the CRISPR gene edition to cure HIV to initiate clinical trials.
Lisa DanzigThe study, led by start-up Excision BioTherapeutics, is likely to begin early next year, CMO said Lisa Danzig, after final details are finalized. These will be three groups of three healthy HIV patients, and each group will have a sequentially higher dose as the company tests the safety of the new therapy.
Therapy uses CRISPR, a tool often compared to molecular scissors, to remove HIV that has coiled into the DNA of a patient’s cells. The ability of the snake snake in its DNA is one of the characteristics that has made it so impervious to healing efforts, despite four decades of research, and the Excision trial will mark one of the first efforts to directly eliminate the latent virus. of DNA.
The excision will deliver CRISPR packaged in AAV9, a non-pathogenic virus commonly used in gene therapy. At a time when high-dose AAV is under scrutiny, the company will adhere to relatively low doses that it found work well in animal studies.
The trial will initially only test the safety and degree of excision of the virus with therapy, but eventually Excision hopes to test it as a cure by removing patients from the standard pills used to control the virus and checking if the infection returns. Danzig, however, did not want to say when they could take that step.
The trial is based on early research by Kamel Khalili, a Temple University professor who first tested the concept in cells in 2014. He was confident, but cautious, about his prospects in human trials, noting animal data showing that CRISPR attacked cells throughout the body.
“This tells us that the strategy has the ability to excise viral DNA,” Khalili said. “The question is whether or not to get rid of the virus completely.”
Experts are largely skeptical that they can, observing the extent to which HIV can be hidden, sleeping in the body’s cells for years. But they agreed that one day it could be a promising part of a healing combination.
Excision and Temple researchers are also working on these combinations, experimenting with new approaches to combining CRISPR with therapies that increase the immune system’s ability to eliminate infected cells.
“This clinical trial is a big step, but it’s a multi-step step,” said Tricia Burdo, a Temple professor and expert on monkey studies who led parts of the preclinical research. “What’s the next step in combining them with other technologies?”