Sano blog | rare disease

precision medicine patient recruitment rare disease genetic testing

AATD gene editing race hinges on patient identification

In September 2026, three in vivo gene editing programs reported progress against the same rare disease, aimed at the same mutation. On September 7, YolTech repo...

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Genetic eligibility shows up earlier in the pipeline than most plans assume

During enrollment planning, genetic eligibility is often treated as a therapy area question, settled once a program knows which organ system it targets. Genetic...

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FDA approves first therapy for Sanfilippo Type A

On 17 September 2026, the FDA granted standard full approval to FAYUVI (rebisufligene etisparvovec-hopf), a single-dose intravenous AAV9 gene therapy from Ultra...

precision medicine patient recruitment rare disease genetic testing protocol design

FDA draft guidance expands single-trial effectiveness pathway

In June 2026, the FDA released a revised draft guidance, Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products, that clarif...

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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...

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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...

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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...

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