Recruitment in genetically stratified clinical trials is often constrained by a simple problem: large screening volumes do not translate into eligible patients. Sponsors can process thousands of participants, yet only a small fraction meet protocol criteria after genetic testing. This creates delays, increases cost, and limits confidence in scaling recruitment programs.
Somerset, UK, 18th March 2026: Predictive Health Intelligence (PHI) and Sano Genetics today announced the completion of recruitment into the LiveWell study, with 996 participants enrolled from a single NHS site in less than a year.
Genome sequencing is now a core part of rare disease diagnostics in several healthcare systems. However, the path from sequencing technology to clinical impact still depends on infrastructure, interpretation, and coordinated healthcare delivery.
Recruiting and retaining patients in rare disease and genomic medicine research requires more than outreach. Many participants need support understanding genetic risk, the purpose of research, and the practical burden of trial participation.
Genetic screening has advanced rapidly over the past two decades. Sequencing is faster and far less expensive, and the ability to interpret genetic variants continues to improve. Despite these advances, screening is still not widely integrated into everyday care.
Rare disease trials operate under structural constraints that make site selection more important than in common disease programs. Patient populations are small, diagnosis often depends on genomic testing, and protocols frequently require specialized assessments and long-term follow-up.
Rare disease innovation has accelerated in recent years, particularly in cell and gene therapy (CGT). Yet for drug developers, one challenge remains persistent: translating scientific progress into therapies that patients can realistically access and benefit from.
In a recent episode of The Genetics Podcast, Patrick Short spoke with Dr. Ryan Dhindsa, Assistant Professor at Baylor College of Medicine and Investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, and Dr. Caleb Lareau, Assistant Professor and Investigator at Memorial Sloan Kettering Cancer Center. They discuss their recent study that used UK Biobank whole-genome sequencing to quantify Epstein-Barr virus (EBV) persistence in blood and links that signal to autoimmune disease risk and host genetics.
The majority of rare disease discovery has been linked to protein coding genes, where changes are easier to interpret and easier to capture in routine testing. The most recent episode of The Genetics Podcast reveals that the next wave of diagnoses may increasingly come from non-coding parts of the genome that have either been dismissed or overlooked.
Adeno-associated virus (AAV) gene therapy has moved from proof of concept to approved products. As more programs enter the clinic and target larger patient populations, manufacturing has become a defining factor in what is feasible. Process design, vector architecture, analytics, and scale all shape how quickly therapies reach patients and at what cost.