UK-based biotech N4 Pharma has announced encouraging in vivo results from its first study exploring Nuvec, its proprietary oral gene delivery platform, in the treatment of inflammatory bowel disease (IBD). The study tested Nuvec particles loaded with both small interfering RNA (siRNA) and messenger RNA (mRNA), demonstrating marked improvements across several key indicators of disease.

The lead programme, N4 101, is an orally administered anti-inflammatory candidate that uses Nuvec to transport nucleic acid therapeutics directly to the gut. The platform is being developed to offer a less invasive, more targeted approach to conditions such as IBD, which affects millions worldwide and is often treated with immune-suppressive drugs delivered by injection.

In this latest study, N4 Pharma investigated the performance of both single (siRNA) and dual-loaded (siRNA and mRNA) Nuvec formulations, with and without a targeting agent. According to the company, the findings provide early but promising evidence of Nuvec’s potential to deliver gene therapies orally with high precision and sustained efficacy.

The study reported several key observations:

• Both single and dual-loaded Nuvec particles demonstrated clear improvements across major indicators of colitis, including the Disease Activity Index, colon length, and body weight loss, when compared with control groups.

• The addition of a targeting agent further enhanced the therapeutic effect, showing a greater reduction in TNF alpha—an inflammatory marker—than the untargeted therapies.

• Six days after the final administration, the targeted Nuvec particles continued to demonstrate a near-complete reduction in TNF alpha levels in intestinal tissue, suggesting a sustained therapeutic effect.

• The study also confirmed that Nuvec particles successfully delivered nucleic acid cargos to the gut via oral administration, which is typically a major challenge for gene therapies.

Nigel Theobald, chief executive officer of N4 Pharma, said: “We are delighted with these very promising in vivo data. Demonstrating a clear increase in performance through targeting the treatment to specific cells is the holy grail of nucleic acid therapeutics, and these results place Nuvec at the leading edge of nucleic acid delivery technologies. While early-stage, these findings significantly strengthen our dataset as we move into optimisation studies and continue broader development efforts across our platform. With the support of our recent fundraise, we look forward to progressing discussions with potential commercial partners and updating the market as we advance this exciting programme.”

N4 Pharma is developing Nuvec as a non-viral delivery system for RNA and DNA therapeutics. The platform is being positioned for use in oncology and chronic inflammatory diseases, where existing delivery methods are often invasive or limited by systemic toxicity.