Gene editing experts are hopeful that a cure for HIV is within reach after more than four decades of research and over 700,000 American deaths. In a groundbreaking trial, three patients in California have been injected with genetic material and an enzyme called CAS9, which has shown promising results in removing sections of the HIV DNA and eliminating the virus.
The trial is utilizing the gene-editing technology CRISPR, which brings the possibility of a cure for HIV closer than ever before. The focus of the current trial is primarily on proving the safety of the treatment, with data on its effectiveness expected to be available next year.
The new gene therapy employs the use of an inactivated virus to deliver the gene editing tool to immune cells. By targeting specific segments of the HIV genome, the therapy renders the virus inactive. This development is significant as HIV, once considered a death sentence, became a manageable chronic disease with the introduction of antiviral medications in the mid-90s.
However, despite available treatments, 1.2 million Americans still live with HIV and face the risk of their infection resurfacing and progressing to AIDS. HIV has the ability to hide in immune cells and evade destruction, making it difficult to eliminate. The highly targeted nature of the CRISPR technology used in the trial offers hope in overcoming these challenges.
Excision BioTherapeutics, a San Francisco-based biotech firm, is conducting the trial. Three patients with HIV have already received the gene therapy, and another six will follow. The early stages of the trial have shown the treatment to be safe, prompting the company to plan for further expansion in which higher doses of the therapy will be administered. Efficacy data from the trial is expected to be available in 2024.
While there have been previous cases of individuals effectively cured of HIV, such as the Berlin Patient and the London Patient, these involved risky stem cell transplants. Timothy Ray Brown, known as the Berlin Patient, was declared HIV-free in 2007 but unfortunately passed away in 2020 from terminal leukemia. Adam Castillejo, known as the London Patient, became the second person to be cured of HIV after undergoing a stem cell transplant in 2016.
The CRISPR gene therapy trial conducted by Excision BioTherapeutics offers hope for a potential cure for HIV. However, further research and data are needed to determine its efficacy. Additionally, the trial provides a unique approach to gene editing in infectious diseases, opening doors for potential treatments for other conditions as well, such as sickle cell anemia.
As the world eagerly awaits the results of this groundbreaking trial, scientists and patients alike are hopeful that this gene therapy could mark a significant turning point in the fight against HIV and bring us one step closer to a cure.