- First-ever randomized, placebo-controlled Phase 1 trial - published in The Lancet Microbe - supports SNIPR001’s safety, tolerability, and restriction to the gastrointestinal tract in healthy participants
- Oral SNIPR001 was well tolerated across three dose levels and was recovered from stool in a dose-proportional manner, without meaningful systemic exposure
- Gut microbiome composition remained stable during and after dosing of SNIPR001
- Recruitment for SNIPR001’s ongoing Phase 1b clinical trial in hematological cancer patients is now more than halfway complete
COPENHAGEN, Denmark, March 03, 2026 (GLOBE NEWSWIRE) -- SNIPR Biome ApS (“SNIPR”), the company pioneering CRISPR-based microbial gene therapy, today announced publication of the final results from its first-in-human Phase 1 clinical trial of SNIPR001 in The Lancet Microbe. SNIPR001 is a novel CRISPR–Cas-armed bacteriophage therapeutic designed to selectively reduce colonization of Escherichia coli (E. coli) in the gastrointestinal tract, including antibiotic-resistant strains, while preserving the broader commensal microbiota.
SNIPR also provided a clinical update on its ongoing Phase 1b trial of SNIPR001 in patients with hematological cancer undergoing hematopoietic stem-cell transplantation (HSCT). Recruitment in this multi-center US study is now more than halfway complete.
Publication highlights from the Phase 1 study
The randomized, placebo-controlled, double-blind, first-in-human, dose-escalation Phase 1 trial (NCT05277350) was conducted in healthy volunteers and evaluated safety and tolerability, as well as recovery/biodistribution, pharmacodynamics, and microbiome composition following oral dosing of SNIPR001 twice daily for seven days.
Key findings of SNIPR001 published in The Lancet Microbe include:
- Favorable safety and tolerability profile: No serious adverse events reported in SNIPR001 dose groups. The number of participants with adverse events was not significantly higher in SNIPR001-treated participants compared with placebo.
- Restriction to the gastrointestinal tract: Functional SNIPR001 was recovered from stool in dose-proportional concentrations, while SNIPR001 was not meaningfully detected in plasma or urine.
- Microbiome preservation: Gut microbiota composition did not significantly differ between the SNIPR001 and placebo groups during and after dosing.
- Pharmacodynamic signal of target engagement: The largest observed reduction in E. coli levels versus placebo was 78% at day 14 in the highest-dose group, though the study was not powered for pharmacodynamic endpoints and the change did not reach statistical significance.
Eric van der Helm PhD, Head of Business Development, commented: “Having the final Phase 1 results published in The Lancet Microbe is a major milestone for SNIPR and an important validation of our scientific approach. This peer-reviewed publication demonstrates that SNIPR001 can be administered orally with a favorable safety and tolerability profile, remain restricted to the gastrointestinal tract, and preserve the broader gut microbiome. We believe this is a true validation of CRISPR-enabled medicine as a precision approach to tackling antibiotic-resistant bacteria.”
Advancing SNIPR001 to address a critical unmet need in vulnerable cancer patients
Patients with hematological cancer undergoing HSCT are at high risk of life-threatening bloodstream infections, which can be caused by translocation of gut pathogens such as E. coli. Antibiotic resistance further complicates prevention strategies and can limit the effectiveness of standard-of-care approaches. SNIPR001 is designed as a precision decolonization strategy targeting E. coli in the gut while minimizing disruption to the overall microbiome - an approach intended to help reduce infection risk without the broad collateral damage associated with conventional antibiotics.
Phase 1b clinical trial update
The ongoing Phase 1b clinical trial (NCT06938867) is a randomized, double-blind, placebo-controlled study evaluating orally administered SNIPR001 in patients with hematological cancer undergoing HSCT. The trial is being conducted at eight centers in the United States and targets enrollment of 24 patients. SNIPR today announced that recruitment is now more than halfway complete, reflecting strong site engagement and continued progress toward evaluating SNIPR001 in the intended high-risk patient population.
Dr. med. Christian Grøndahl, CEO and Co-founder of SNIPR, commented: “Building on these Phase 1 results, we are focused on advancing SNIPR001 in the patient population where the need is greatest. We are pleased to share that recruitment in our ongoing Phase 1b trial in hematological cancer patients is now more than halfway complete. This study is an important next step toward evaluating SNIPR001’s potential to reduce the risk of E. coli bloodstream infections in vulnerable patients undergoing stem-cell transplantation, and we look forward to progressing the program as recruitment continues.”
Funding
Research reported in this press release is supported by CARB-X. CARB-X’s funding for this project is provided in part with federal funds from the U.S. Department of Health and Human Services (HHS); Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority; under agreement number: 75A50122C00028, and by awards from Wellcome (WT224842) and Germany’s Federal Ministry of Research, Technology and Space (BMFTR). The content of this press release is solely the responsibility of the authors and does not necessarily represent the official views of CARB-X or any of its funders.
Media Contact
Christian Grøndahl | +45 2020 2747 | cg@sniprbiome.com
