Excision BioTherapeutics Administers CRISPR Gene-Editing Therapy to Combat HIV
In a groundbreaking development, three patients have been treated with CRISPR gene-editing therapies in an attempt to eliminate HIV from their bodies. This revolutionary treatment, known as EBT-101, was administered by the San Francisco biotech company Excision BioTherapeutics, which utilized technology based on Nobel Prize-winning research by UC Berkeley’s Jennifer Doudna.
While the results of the treatment have not yet been disclosed by the company, it has been reported that EBT-101 is safe and has caused no major side effects. Excision BioTherapeutics presented these findings at a recent meeting in Brussels. Six additional individuals will also receive the treatment, potentially at UC San Francisco, using higher doses.
Participating in the research program carries some risks, as participants are required to discontinue their protective anti-HIV drugs for 12 weeks after receiving the gene-editing treatment. This allows researchers to determine if the virus has been completely eradicated. The data from these trials will be presented at a medical conference next year.
Excision BioTherapeutics’ senior vice president of clinical development, Dr. William Kennedy, expressed optimism about the potential of this new drug. He stated, “We are opening the door for how this new drug will work and what potential it has for people living with HIV. Ultimately, we see this as a fundamentally new approach.”
In addition to its potential for treating HIV, EBT-101 may also be applicable for other chronic infections in which the virus lies latent, such as hepatitis and herpes. Unlike previous treatments, EBT-101 leaves human DNA intact, making it a safer and more targeted therapeutic solution.
Excision BioTherapeutics received the FDA’s “fast track” designation for EBT-101 last July after successful experiments in animals. The treatment effectively removed a virus related to HIV from the genomes of rhesus monkeys. It also eliminated HIV from nine of 23 mice in earlier studies.
While these results are promising, it is important to note that there is still a significant leap from success in animal models to human trials. Patient recruitment for the trials is taking place at various locations, including Quest Clinical Research in San Francisco, Washington University in St. Louis, and Cooper University in Camden, New Jersey.
Since the isolation of the AIDS virus 40 years ago, treatment has significantly transformed the care of HIV patients. Antiretroviral drugs, when taken consistently, can suppress the virus and prevent illness and transmission. However, a cure is necessary to effectively end the global HIV pandemic. Currently, nearly 39 million people are living with HIV worldwide, with approximately 77% of them receiving treatment.
Only three cases of HIV cure have been recorded thus far, all of which were initiated through bone marrow transplants from donors with a specific genetic mutation. However, these transplants were primarily targeting cancer rather than directly tackling HIV, making them impractical for the average HIV patient.
Excision BioTherapeutics’ research is based on the work of Kamel Khalili, a professor at Temple University in Philadelphia and director of its Center for NeuroVirology and Gene Editing. The California Institute of Regenerative Medicine has also provided support for the company’s research.
CRISPR gene editing, discovered by biologist Jennifer Doudna, has shown potential in curing genetic diseases by precisely removing problematic segments of a person’s DNA. This technology is now being employed in the treatment of various diseases, including sickle cell anemia, nerve disease, and congenital blindness.
In the case of HIV, Excision BioTherapeutics’ approach using CRISPR involves cutting the virus’s DNA at two specific locations, removing essential genes and inhibiting replication. The potential of this treatment has received cautious enthusiasm from long-term HIV survivors and activists, who note the complex nature of the virus and the challenges that come with eradicating it entirely.
However, Excision BioTherapeutics’ therapy presents hope for a potentially lifelong cure for HIV, freeing patients from the need to take daily antiretroviral medication. The company plans to monitor patients for 15 years to assess the long-term effects of the treatment.
As the world continues to battle the HIV epidemic, Excision BioTherapeutics’ research represents a significant step forward in the quest for an HIV cure. “We’ve got to have hope because the epidemic isn’t over,” remarked Matt Sharp, an AIDS activist who has lived with the virus for 38 years. With ongoing research and breakthroughs like EBT-101, the dream of a cure for HIV may soon become a reality.