CRISPR and Herpes: The Cutting-Edge Gene Editing Approach to a Herpes Cure

Herpes simplex virus (HSV), which causes oral and genital herpes, remains a lifelong infection with no current cure.

Traditional antiviral medications manage symptoms but cannot eliminate the virus, which hides in nerve cells in a dormant state.

Recent advances in gene editing, particularly CRISPR technology, have opened new possibilities for directly targeting and disabling latent HSV DNA, offering hope for a functional cure.

This article provides an in-depth overview of CRISPR’s role in herpes research, the latest breakthroughs, and the challenges ahead.

What is CRISPR and How Does It Work?

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a revolutionary gene editing tool that allows precise targeting and modification of DNA sequences.

It uses a guide RNA to locate specific DNA regions and a Cas9 enzyme to cut the DNA at those sites.

For herpes treatment, CRISPR can be engineered to target and cut the viral DNA integrated into host nerve cells, disrupting the virus’s ability to replicate and reactivate.

This technology is highly versatile and has been adapted for a wide range of genetic diseases, cancers, and infectious diseases.

Its precision and relative ease of design make it a powerful candidate for addressing latent viral infections that have been historically difficult to treat.

CRISPR-Based Therapies in Herpes Research

Several research groups and biotech companies are developing CRISPR-based gene therapies targeting HSV.

Notably, Excision BioTherapeutics has presented data on their candidate EBT-104, a CRISPR-Cas9 therapy designed to excise critical regions of the HSV-1 genome from latent reservoirs, particularly targeting HSV-1 keratitis.

Preclinical studies show that EBT-104 can significantly reduce latent HSV DNA and viral gene expression in animal models, demonstrating high efficacy and specificity.

This represents a major step toward a one-time treatment for sustained suppression or potential elimination of HSV infection.

In addition to Excision BioTherapeutics, academic institutions are also exploring variations of CRISPR and other gene editing tools to improve delivery methods and reduce off-target effects. Viral vectors, nanoparticles, and lipid-based carriers are being tested to reach the nerve cells effectively, which remain a significant delivery challenge.

Advantages of CRISPR for Herpes Treatment

Unlike traditional antivirals that only suppress active viral replication, CRISPR targets the latent viral DNA integrated into nerve cells, which is the root cause of recurrent herpes outbreaks.

This precision allows for the possibility of permanently disabling or removing the virus from infected cells, potentially providing a functional cure.

CRISPR therapies can be designed to minimize off-target effects, improving safety profiles compared to earlier gene editing methods.

Moreover, CRISPR’s modularity means that future therapies could be adapted quickly to target different HSV strains or other latent viruses, making it a versatile platform for antiviral treatment.

Challenges and Considerations

Despite promising results, several challenges remain before CRISPR-based herpes therapies become clinically available. Delivering gene editing components efficiently and safely to the nerve cells where HSV resides is complex. Ensuring specificity to avoid unintended genetic alterations in human DNA is critical to prevent side effects. Regulatory approvals require extensive safety and efficacy data from clinical trials, which are still in early phases for herpes applications.

Additionally, immune responses to delivery vectors or the CRISPR components themselves could limit treatment effectiveness or cause adverse effects, necessitating careful design and testing.

Current Status and Future Outlook

As of 2025, multiple CRISPR clinical trials for various diseases are underway, with herpes-focused programs advancing preclinically and preparing for human studies. Excision BioTherapeutics is progressing toward an Investigational New Drug (IND) application for EBT-104, aiming to initiate clinical trials for HSV-1 keratitis treatment. Fred Hutch’s gene editing approach is also moving closer to clinical translation, with ongoing collaborations to ensure regulatory compliance and safety.

While a CRISPR-based herpes cure is not yet available, the rapid pace of research and technological improvements make it a promising prospect within the next decade.

Continued investment, public awareness, and scientific collaboration will be essential to overcome remaining hurdles and bring these therapies to patients.