Gene-editing science comes to Abu Dhabi as IROS and Mammoth Biosciences launch landmark clinical trial

A groundbreaking partnership brings cutting-edge CRISPR technology to the UAE for the first time, marking a pivotal moment in the emirate's development of genetic medicine expertise.

The intersection of molecular biology and clinical medicine reaches a new frontier in Abu Dhabi with the launch of the UAE’s first clinical trial of a gene-editing therapy delivered via lipid nanoparticles. IROS, the contract research organisation within M42, has partnered with Mammoth Biosciences to evaluate MB-111, an investigational treatment targeting severely elevated blood triglycerides in adults with persistent chylomicronemia.

The therapy represents a notable departure from conventional approaches to metabolic disease. Rather than managing symptoms through diet and medication, MB-111 employs Mammoth’s ultracompact CRISPR gene-editing system, CasPhi, to directly address the genetic basis of the condition. The treatment uses tiny lipid-based particles to deliver the gene-editing machinery into liver cells, where it switches off the APOC3 gene, reducing production of a protein central to triglyceride regulation. This approach aims to achieve lasting therapeutic effect through a single intravenous infusion.

Persistent chylomicronemia, whether inherited or arising from a combination of genetic and other factors, leaves patients with dangerously high triglyceride levels that can trigger recurrent, life-threatening pancreatic inflammation. For those whose condition persists despite conventional treatment, the burden is considerable: restrictive diets, ongoing medication and repeated hospitalisation become facts of daily life.

The collaboration builds upon an agreement signed in June 2026 between Abu Dhabi’s Department of Health, M42 and Mammoth Biosciences to advance gene-editing therapies. As we reported when M42’s clinical research division began examining precision nutrition for GLP-1 patients, Abu Dhabi is establishing itself as a centre of expertise in advanced therapeutic approaches. This trial extends that commitment by bringing next-generation gene-editing science into clinical development within the emirate.

Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi, characterised the undertaking as a practical step toward developing local genetic medicine expertise. The Emirati Genome Programme, she noted, has deepened understanding of genetic variation in the population, creating a foundation upon which rigorous research into potential treatments can now be constructed. Dr. Fahed Almarzooqi, Chief Executive Officer of M42’s Integrated Health Solutions, emphasised that the ambition extends beyond a single trial: the partnership aims to build a centre of expertise capable of advancing successive generations of genetic medicines and positioning Abu Dhabi as a destination for their clinical development.

What it means for Abu Dhabi

This trial signals Abu Dhabi’s emergence as a location where frontier science meets clinical rigour. By hosting the first evaluation of such technology in the region, the emirate demonstrates its commitment to not merely adopting advanced therapies but to pioneering their development and establishing the expertise necessary for their evaluation. The partnership unites genomic knowledge accumulated through population-scale research with advanced healthcare infrastructure and clinical research capabilities, creating a unique ecosystem for genetic medicine.

For patients within Abu Dhabi and the broader region, the trial opens the possibility of access to transformative treatments at the earliest stage of clinical evaluation. More broadly, the evidence generated through this research may inform future care pathways throughout the UAE and beyond, positioning Abu Dhabi’s healthcare system as a source of knowledge for treating complex genetic conditions. The development of local expertise in genetic medicine strengthens the emirate’s capacity to address rare diseases and advance therapeutic science for generations to come.

Source: WAM