Safety, recovery, and pharmacodynamics of CRISPR-Cas therapeutic SNIPR001: a phase 1, randomised, double-blind, first-in-human, dose-escalation study - PubMed

5 days ago

SNIPR001 is a CRISPR-Cas-armed bacteriophage cocktail targeting E. coli in the gastrointestinal tract, designed to spare commensal microbiota.
A phase 1, randomized, double-blind, dose-escalation study assessed SNIPR001's safety, tolerability, and pharmacodynamics in healthy participants.
Participants received doses of 10^8, 10^10, or 10^12 PFU of SNIPR001 or placebo twice daily for 7 days, with follow-up until day 187.
Primary outcome was the incidence and severity of adverse events, with secondary outcomes including recovery, biodistribution, and microbiome impact.
Results showed only mild to moderate adverse events, with no significant difference between treatment and placebo groups.
SNIPR001 was detected in stool in dose-proportional concentrations but not meaningfully in blood or urine, and was undetected after 6 months.
A reduction in E. coli levels was observed but was not statistically significant, with no major disruption to gut microbiome composition.
The study supports SNIPR001's safety and justifies further clinical development in a phase 1b/2a trial.

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