EXCISION BIOTHERAPEUTICS

• Excision BioTherapeutics presented new data from its CRISPR-based therapies targeting hepatitis B virus (EBT-107) and herpes simplex virus keratitis (EBT-104) at the ASGCT 2025 Annual Meeting in New Orleans. • EBT-107 demonstrated potential to reduce clinically-relevant biomarkers and HBV DNA integration, suggesting progress toward functional remission of chronic hepatitis B. • EBT-104 previously showed complete elimination of viral shedding in 92% of treated rabbit eyes, highlighting its potential as a curative approach for HSV-1 keratitis, the leading cause of corneal blindness in the US.

CRISPR Medicine News tracks approximately 250 clinical trials involving gene-editing therapeutic candidates as of February 2025, with more than 150 trials currently active across diverse therapeutic areas.

Excision BioTherapeutics' EBT-101, a CRISPR-based gene editing therapy, demonstrates safety and temporary HIV infection suppression in early trials.

• CRISPR-Cas9 gene editing has achieved a significant milestone with FDA approval of Casgevy for sickle cell disease and transfusion-dependent beta thalassemia. • Clinical trials are underway for CRISPR-based therapies targeting urinary tract infections, hereditary transthyretin amyloidosis, hereditary angioedema, and cardiovascular diseases. • CRISPR technology is being explored for chronic conditions like type 1 diabetes, systemic lupus erythematosus, and HIV, with early trials showing potential for disease management. • Challenges remain in addressing the high costs of CRISPR therapies and establishing regulatory standards to manage potential off-target effects and ethical implications.

CRISPR-based therapies are entering a new phase with the first approval of Casgevy for sickle cell disease and transfusion-dependent beta thalassemia.