Sano blog

patient recruitment rare disease patient engagement

Long-term follow-up in clinical trials is a compounding asset for rare disease programs

Rare disease trials demand years of investment to identify, genetically screen, enroll, and engage patients. When the study closes, that effort disperses. Commu...

precision medicine patient recruitment rare disease genetic testing

OptiPrime speeds prime editing but patient identification lags

On August 12, 2026, the David Liu lab at the Broad Institute published OptiPrime, a machine-learning model that predicts how well prime-editing guide RNAs (pegR...

The Genetics Podcast featuring Stevie Ringel

rare disease podcast recap

Podcast recap: Stevie Ringel on building operational capacity for rare disease drug development

Rare and ultra-rare disease research has produced real scientific breakthroughs over the past decade: antisense oligonucleotides, gene therapies, and gene editi...

Why cell and gene therapy programs stall between protocol and patient

Cell and gene therapy has solved problems that seemed intractable a decade ago. The science of editing genomes, replacing defective genes, and engineering patie...

precision medicine genetic epidemiology genetic testing

Inherited genetics linked to CAR-T toxicity and expansion

CAR-T therapies carry meaningful safety risks, and toxicity is known to vary with factors including disease characteristics, patient factors and the therapy its...

The Genetics Podcast featuring Jagesh Shah

podcast recap

Podcast recap: Jagesh Shah on why delivery is genetic medicine's real bottleneck

In genetic medicine, we increasingly know what to deliver. The unsolved problem is getting it to the right place in the body. That was the central thesis in the...

precision medicine rare disease protocol design patient engagement

Split verdicts: What HOPE-3 exposes about rare disease evidence

On 29 July 29, 2026, the same clinical trial data produced opposite verdicts. The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee voted 9-3 that a...

precision medicine screen failure patient recruitment protocol design

Precision medicine's execution deficit is an accountability problem

A genetically-defined trial can be designed correctly and still stall. The protocol names the right molecular subtype, the endpoints are sound, and the therapeu...

screen failure patient recruitment rare disease genetic testing patient engagement

Why precision medicine trials fail on the way from protocol to patient

Precision medicine clinical trials are designed by some of the most capable scientists in drug development. The biomarker hypotheses are well-reasoned, the endp...

patient recruitment protocol design health equity

Decentralized trials expand access and diversity at Mayo Clinic

On July 20, 2026, Mayo Clinic reported peer-reviewed evidence that decentralization changes who takes part in clinical research. In a study published in JAMA Ne...

Clinical research blog