FDA clears first-of-its-kind trial to see if editing CRISPR genes can cure HIV – Endpoints News

The FDA has cleared a company trying to use CRISPR gene editing to cure HIV to begin clinical trials.

The study, led by startup Excision BioTherapeutics, will likely begin early next year, Marketing Director Lisa Danzig said, after final details were chopped up. It will involve three groups of three healthy HIV-infected patients, with each group receiving a sequentially higher dose as the company tests the safety of the new therapy.

The therapy uses CRISPR, a tool often compared to molecular scissors, to extract HIV that has coiled up in DNA from a patient’s cells. The virus’s ability to squeeze into DNA is one of the characteristics that has made it so impervious to curative efforts, despite four decades of research, and the Excision trial will mark one of the first efforts to eliminate the virus latent directly from the DNA.

Excision will provide CRISPR packaged in AAV9, a non-pathogenic virus commonly used in gene therapy. At a time when high-dose AAV is under intense scrutiny, the company will stick to relatively low doses that have worked well in animal studies.

The trial will initially only test the safety and effectiveness of therapy in excising the virus, but ultimately Excision hopes to test it as a remedy by withdrawing patients from the standard pills used to control the virus and seeing if the infection returns. . Danzig, however, declined to say when they might take this step.

The trial builds on early research from Kamel Khalili, a Temple University professor who first tested the concept in cells in 2014. He was confident but cautious about their prospects in human trials, highlighting 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 it reaches the point of completely eliminating the virus. “

Experts are largely skeptical of the possibility, noting how HIV can hide, sleeping in body cells for years. But they agreed it could one day be a promising part of a healing combination.

Excision and Temple researchers are also working on these combinations, experimenting with new approaches to combine CRISPR with therapies that boost the immune system’s ability to kill infected cells.

“This clinical trial is a huge step, but it is one of many steps,” said Tricia Burdo, a professor at Temple and an expert in monkey studies who led some of the preclinical research. “What’s the next step in combining them with other technologies? “