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Clinical research blog

Explore our blog for insights into the big questions in precision medicine and clinical research.

OptiPrime speeds prime editing but patient identification lags

On 12 August 2026, the David Liu lab at the Broad Institute published OptiPrime, a machine-learning model that predicts how well prime-editing guide RNAs (pegRN...
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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...
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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...
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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...
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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...
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Podcast recap: Christopher Walsh on how somatic mosaicism rewrites the story of neurodegeneration

For decades, the dominant framework for understanding neurodegenerative disease has centered on inherited risk genes. A growing body of single-cell genomics res...
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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 ...
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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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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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