gene editing, HIV, Aids, clinical trials

Clinical Gene Editing Emerges As Promising Path To HIV/AIDS Cure

An academic paper published in the National Library of Medicine suggests a relatively new technology has the potential to provide a solution to HIV/AIDS. The paper, authored by Mourya Hussein, Mariano A. Molina, Ben Berkhout, and Elena Hererra-Carrillo, argues that employing CRISPR technology, which can be used to edit genes, to target host and viral genes, activate antiviral host factors, and inhibit viral replication, could lead to the elimination of the virus.

HIV currently affects 38 million people worldwide, resulting in approximately 800,000 annual deaths from virus-related complications.

The paper acknowledges that while promising, there are challenges in developing a viable pathway to curing HIV/AIDS through gene therapy, such as the risk of affecting genes unrelated to HIV/AIDS production and spread in the body. Nonetheless, the study recommends further examination of gene-editing therapy through clinical trials to assess its effectiveness.

In July 2023, the FDA fast-tracked a trial for gene therapy relating to HIV/AIDS to test the efficacy and safety of using CRISPR to treat the disease in conjunction with antiviral drugs. According to Medical Professionals Reference, a fast-track designation from the FDA is intended to accelerate the development of promising treatment for conditions for which either no treatment exists, or the therapy being investigated is expected to provide advantages over currently available treatments.

Excision BioTherapeutics CEO Daniel Dornbusch, whose company received the designation, said in a press release, “We are pleased with the FDA’s decision to grant Fast Track designation to EBT-101. This designation underscores the importance of finding a cure for people living with HIV and bolsters Excision’s efforts to rapidly develop potentially curative therapies for significant unmet medical needs.”

Following that news, MIT Technology Review reported on Oct. 25 that Excision added the gene-editing tool to the bodies of three people living with HIV. The company directed the gene-editing tool to eliminate the virus but has not yet revealed any data about the effects of the treatment, which leaves experts in the field wondering exactly how effective it has been at achieving the intended goal.

Fyodor Urnov, a genome editing expert at the University of California-Berkeley, told MIT, “This is an exceptionally ambitious and important trial,” before adding that it “would be good to know sooner than later” the effects of the treatment, “including, potentially, no effect.” 

Excision says the trials have yielded no major negative side effects and that it has plans to increase doses of the treatment in 2024. The company says it wants to have six additional patients receive three and 10 times the amount of treatment delivered thus far.

William Kennedy, senior vice president of Excision’s Clinical Development division, says that the full results for the original three clinical participants will not be available until 2024.

Kamel Khalil, a Temple University professor who helped start the company, says that even if the treatments don’t cure HIV, they represent an important step toward development in treating the disease, as he told MIT. “Even if we don’t completely cure [HIV], we might be getting a significant delay in the rebound of the virus. That could set us up for the next stage, like any drug where there are first and second generations.”

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