LOCUS BIOSCIENCES

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.

• BARDA has provided $24 million in funding to Locus Biosciences as part of a larger $85 million award to advance LBP-EC01, a CRISPR-engineered bacteriophage therapy for resistant E. coli infections. • The novel therapy, which combines bacteriophage technology with CRISPR/Cas3 gene-editing, has shown promising results in phase 1b trials and is currently undergoing phase 2 evaluation in the ELIMINATE trial. • With UTIs affecting 150 million people annually and 80% caused by E. coli, this innovative approach addresses an urgent public health threat identified by both the CDC and WHO.

• 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.

• Leading pharmaceutical companies including Merck, Pfizer, and SNIPR Biome will convene in Boston to discuss advancements in bacteriophage therapy development and clinical applications. • The summit will focus on addressing clinical translatability challenges and exploring bioinformatics applications for improved phage therapy sequencing and data processing. • Industry experts from 90+ organizations will collaborate to demonstrate clinical utility of bacteriophage therapies and forge partnerships in combating antimicrobial resistance.