Sano blog | precision medicine

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...

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...

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...

precision medicine patient recruitment health equity

Why polygenic risk scores miss diverse populations

On 20 July 2026, The New York Times reported that the genetic risk tools entering clinical use do not perform equally well across populations. Polygenic risk sc...

precision medicine protocol design population health health equity

The All of Us milestone shows precision medicine’s next infrastructure challenge

The NIH’s All of Us Research Program has become the world’s largest integrated genomic and electronic health record database. Its June 30 data release includes ...

precision medicine neurodegenerative disease podcast recap

Podcast recap: Yentli Soto Albrecht on turning a fatal diagnosis into a cure strategy

The C9orf72 hexanucleotide repeat expansion is the most common genetic cause of both ALS and frontotemporal dementia (FTD). Carriers face roughly a 50% chance o...

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 neurodegenerative disease podcast recap

From discovery to dosing: Trace Neuroscience's UNC13A bet is now in the clinic

When Trace Neuroscience announced on June 22 that first patients had been dosed in the LAUNCH ALS trial, it felt like the right moment to revisit our original p...

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...

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