Excision's CRISPR HIV Treatment Shows No Early Cure Potential

An early-stage study involving a CRISPR-based gene editing therapy developed by Excision BioTherapeutics aimed at curing HIV has yielded underwhelming results, according to reports.

The Phase I trial, which included five patients, demonstrated that Excision's CRISPR therapeutic did not significantly suppress the HIV virus. According to STAT News, three participants who discontinued their antiretroviral therapy (ART) experienced a viral rebound and had to resume their conventional treatments.

Despite these setbacks, there were some promising signs. One patient managed to keep the virus under control for 16 weeks after stopping antiretroviral treatment before experiencing a rebound. Ordinarily, HIV levels tend to resurge approximately four weeks after stopping ART. Additionally, the experimental CRISPR treatment appeared to reduce the number of infected cells in this patient, according to STAT.

CEO Daniel Dornbusch expressed optimism about the safety data from the Phase I study. He stated that the findings indicate that "in-vivo CRISPR can be done in a safe way in humans." The company intends to leverage these insights to develop a new gene editing candidate for other chronic viral infections, such as herpes and hepatitis B.

Excision's leading candidate, EBT-101, is a CRISPR-based gene editor designed to excise the HIV pro-viral DNA from all infected cells. Delivered via an AAV9 vector, EBT-101 employs two guide RNAs that target three specific sites in the HIV genome, thereby reducing the chances of viral escape.

The company is promoting EBT-101 as a potentially curative treatment for chronic HIV and is progressing towards further clinical trials.

In October 2023, Excision shared safety data from its early Phase I/II study of EBT-101, which revealed no serious adverse events or dose-limiting toxicities among the three patients who had received the gene editor. There were four mild toxicities potentially linked to EBT-101, all of which resolved without requiring intervention.

The preliminary data also indicated that EBT-101 remained detectable in the blood four weeks post-treatment.

Beyond HIV, Excision is utilizing its proprietary CRISPR-based gene-editing platform to target herpes 1 and 2 through EBT-104, and hepatitis B via EBT-107.

Several major biotech firms are also addressing the HIV crisis, including Gilead and GSK. Gilead's portfolio includes the oral antiretroviral pill Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide), whereas GSK and its partner ViiV Healthcare market the oral pill Tivicay (dolutegravir) and the long-acting injectable Cabenuva (cabotegravir/rilpivirine).

While these treatments are effective at suppressing the virus, a definitive cure remains elusive, presenting ongoing challenges in the field. In March 2024, the World Health Organization raised concerns about increasing resistance to Tivicay.

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