First Patient Dosed With CRISPR Therapy Targeting E. Coli Colonization

The first patient has been dosed in a new phase 1 clinical trial from SNIPR BIOME, exploring safety and tolerability of SNIPR001, an orally administered CRISPR-based therapy.¹

The therapy, which had been previously granted fast track designation by the FDA back in January 2022,² is intended to reduce colonization of E. coli in the gut in a high-risk population of patients with hematological cancer at risk for neutropenia.

Being developed in partnership with CARB-X, SNIPR001 targets and eliminates E. coli in the gut in order to help prevent transfer of the bacteria to the bloodstream, where it can result in life-threatening infection.

The phase 1 clinical trial (NCT05277350) has a target enrollment of 36 healthy volunteers who will be randomly assigned to receive 1 of 3 ascending doses of SNIPR001 or placebo twice daily for 7 days. Follow-up will be conducted over 6 months following the last dose.

The primary end point is safety and tolerability through day 35, including severity of adverse events and medically attended adverse events. Additional secondary outcomes are AEs through day 187, as well as functional quantification of the study drug in blood, feces, and urine.

“E. coli was recently highlighted as one of the leading pathogens associated with anti-microbial-resistance and death in a systematic review published by the scientific journal The Lancet, so there is an urgent need for new medicines targeting E. coli,” said SNIPR BIOME cofounder and CEO, Christian Grøndahl, in a statement.

The investigational therapeutic utilizes SNIPR BIOME’s proprietary CRISPR/Cas technology that essentially programs the adaptive immune system to target and kill bacteria based on their specific genome, in turn avoiding unintended targets and leaving the rest of the microbiome intact. SNIPR utilizes DNA-based CRISPR-guided vectors to illicit a rapid kill response.

In addition to this specific indication, SNIPR BIOME can also harness its technology to modulate the microbiome, including selectively killing disease-associated bacteria or the introduction of therapeutic proteins and peptides.

REFERENCES

1. SNIPR BIOME Initiates First-in-Human Clinical Trial with SNIPR00. News release. SNIPR BIOME. April 20, 2022. https://www.prnewswire.com/news-releases/snipr-biome-initiates-first-in-human-clinical-trial-with-snipr001-301528709.html

2. SNIPR BIOME Announces U.S Food and Drug Administration grants Fast Track Designation for SNIPR001 for Prevention of Bloodstream Infections in Hematologic Cancer Patients. News release. SNIPR BIOME. January 25, 2022. https://static1.squarespace.com/static/5bacc67990f9041ab0d5b0c1/t/61eff30c5e4be110d3a84261/1643115277303/Press+Release+SNIPR001+Fast+Track.pdf