In 1983, the identification of HIV, the virus responsible for AIDS, marked a grim era where contracting the virus was often considered a death sentence. Today, antiretroviral drugs have transformed the scenario, enabling people to manage the virus effectively, though a cure remains elusive. San Francisco-based biotech firm Excision BioTherapeutics aims to change that narrative with its groundbreaking gene-editing infusion, EBT-101.

Recent reports from the company have highlighted positive safety outcomes in the experimental drug’s one-off gene-editing treatment. The trial involved three patients, and no severe side-effects were observed. However, efficacy results are expected only by 2024, leaving anticipation for a potential breakthrough in the quest for an AIDS cure.

Despite the availability of antiretroviral drugs, the global toll of AIDS-related deaths remains significant, emphasizing the urgent need for a cure. Excision BioTherapeutics’ approach utilizes Crispr, a gene-editing technology inspired by bacterial cells’ natural defense mechanisms. Crispr acts like a miniature robot, precisely targeting and disabling the virus by cutting large sections of its DNA, hindering replication.

Originally discovered 35 years ago, Crispr has made substantial strides in the last decade, particularly in treating inherited diseases like sickle cell disease. Excision BioTherapeutics’ application of Crispr to combat HIV signifies a promising avenue in the ongoing battle against the virus.

Antiretroviral drugs, while life-saving, can induce side-effects, making the pursuit of alternative treatments crucial. HIV’s ability to evade the immune system presents a complex challenge, with the virus strategically hiding within immune cells. Crispr’s core-targeting approach enhances the chances of rendering the virus inactive, providing a potential breakthrough in the quest for a cure.

The journey from successful testing in lab animals to human trials is a critical step in validating the efficacy and safety of Crispr-based treatments. Excision BioTherapeutics’ EBT-101, having shown promise in mice, rats, and macaques with the simian form of HIV, reflects a significant leap towards human trials.

While the safety results are encouraging, the path to a cure involves extensive testing on larger groups and addressing affordability concerns, particularly in regions with a higher prevalence of HIV. Excision BioTherapeutics’ advancements bring a glimmer of hope, suggesting that a cure for AIDS may be on the horizon.

By Impact Lab