Sano blog | rare disease

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...

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...

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...

precision medicine rare disease patient engagement

Who tells patients when a gene therapy program pauses?

When a gene therapy sponsor pauses a clinical program, the operational machinery responds within hours. The medical monitor convenes a safety review. Regulatory...

precision medicine patient recruitment rare disease genetic testing patient engagement

Operation Trailblazer and the clinical trial access gap

On June 23, 2026, the US Department of Health and Human Services launched Operation Trailblazer, a cross-agency initiative coordinated across the FDA, NCI, NCAT...

Huntington's disease patient recruitment rare disease genetic testing neurodegenerative disease

What two FDA gene therapy reversals in one week means for rare disease sponsors

In the space of five days, the FDA reversed its position on two rare disease gene therapies it had previously rejected. On June 17, the agency agreed that UniQu...

The Genetics Podcast featuring Timothy Yu

precision medicine rare disease podcast recap

Podcast recap: Tim Yu on the rise of N-of-1 genetic medicines

Most genetic medicine is built for populations large enough to support a clinical trial. N-of-1 medicine is built for the opposite case: a single patient whose ...

precision medicine rare disease

The rare disease blueprint: What precision medicine can learn from the hardest trials

Rare disease exposes the limits of current clinical development models. Small, fragmented populations make patient identification difficult. Heterogeneous biolo...

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