A patient who developed multidrug-resistant E. coli following a transplant has recovered following treatment with an investigational clustered regularly interspaced short palindromic repeats (CRISPR) gene therapy.
The 65-year-old kidney transplant patient had developed progressive, multidrug-resistant E. coli malakoplakia, a rare disease characterised by intracellular bacterial persistence, post-transplant. While the patient initially received antibiotics to treat the infection and mass that arose from the bladder wall and prostate, these were not successful.
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The patient then received SNIPR001, an investigational CRISPR gene therapy developed by Danish biotech SNIPR Biome, in addition to standard-of-care therapy, including antibiotics and adjunctive treatments.
Within one week of initiation, the patients’ abdominal cutaneous lesions improved markedly, with several fully healed by four weeks, accompanied by substantial reduction of the intra-abdominal mass on serial imaging from 744.6 cm3 at baseline to 373.4 cm3 at week eight. After a year, this had decreased even further to 82 cm3, reflecting an 89% reduction.
The patient data has been published in a peer-reviewed expanded access case report in Clinical Infectious Diseases on 11 August.
The patient was treated by infectious disease investigators at the University of California San Diego under a single-patient emergency investigational new drug (eIND) application. SNIPR said the data demonstrate the translational potential of SNIPR001 in a real-world compassionate-use setting.
Dr Christian Grøndahl, CEO and co-founder of SNIPR, said: “This case report marks an important clinical milestone for SNIPR001 and highlights the potential of our engineered CRISPR medicine platform to address difficult-to-treat, drug-resistant E. coli infections. We are grateful to the investigators at UC San Diego and to the patient and care team involved in this compassionate-use case, which provides valuable clinical insight into the potential role of SNIPR001 beyond prevention of bloodstream infections.”
SNIPR001 is a CRISPR-armed phage therapeutic that is currently being developed for the prevention of E. coli bloodstream infections in patients with haematological malignancies and is also being explored for the treatment of active E. coli infections.
It is currently in a randomised, double-blind, placebo-controlled Phase Ib/IIa trial (NCT06938867) that is investigating the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of orally administered SNIPR001 in 24 patients with haematological cancer.
SNIPR previously completed a Phase Ia trial (NCT05277350) in the US, which the company said demonstrated the safety of and target engagement of SNIPR001 with E. coli in the gut of healthy volunteers without disturbing the overall gut microbiome.
CRISPR hold promise in AMR, analysts believe
GlobalData published a report, “CRISPR Gene Editing in Infectious Diseases: Market Overview”, looking into what programmable gene editing technologies are being investigated in clinical trials to address long-standing unmet needs in both viral and bacterial infections, with companies such as Excision BioTherapeutics, BDGene Therapeutics, Locus Biosciences, and SNIPR Biome leading the charge.
The report highlights how CRISPR-enhanced phage therapy is gaining traction as a means to combat antimicrobial resistance (AMR). Companies such as Locus Biosciences and SNIPR Biome are developing engineered bacteriophages that selectively eliminate antibiotic-resistant E. coli, with Phase I/II trials already underway.
Speaking when the report was released, Abigail Harris, infectious disease analyst at GlobalData, said: “While early trial results are encouraging, key opinion leaders (KOLs) interviewed by GlobalData highlight delivery challenges, immune responses, and regulatory uncertainty as major hurdles to widespread adoption. New delivery platforms, such as lipid nanoparticles and adeno-associated viruses (AAVs), are under active investigation to enable safe, targeted in vivo editing.”
“Despite current challenges, CRISPR-based therapeutics hold the potential to shift the treatment paradigm for some of the most persistent infections worldwide. As more data emerges from clinical trials, gene-editing-based approaches in infectious disease are expected to see increased momentum, investment, and regulatory support,” she added.
