Sano blog | precision medicine

precision medicine patient recruitment rare disease genetic testing

What BEAM-301 reveals about the next challenge in precision medicine trials

Beam Therapeutics' early clinical results highlight the promise of mutation-specific therapies. But as treatments become more genetically targeted, they also ra...

precision medicine patient recruitment rare disease protocol design

FDA's rare disease framework could center patient identification

An FDA notice seeking public input is under review at the Office of Management and Budget, according to a Citeline report on October 12, 2026. The notice sugges...

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...

precision medicine thought leadership industry report rare disease

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...

biomarkers precision medicine patient recruitment

Autoimmune cell therapy pauses expose stratification gaps

Novartis and Bristol Myers Squibb have paused multiple trials of their autoimmune cell therapies following inflammatory side effects. Novartis placed holds on i...

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FDA draft guidance expands single-trial effectiveness pathway

In June 2026, the FDA released a revised draft guidance, Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products, that clarif...

precision medicine public health patient recruitment genetic testing population health

Genomic newborn screening: The operational challenge ahead

A Nature News Feature published on 19 August 2026 synthesized the global wave of genomic newborn screening programs and opened with a concrete result. In the GU...

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...

Clinical research blog

What BEAM-301 reveals about the next challenge in precision medicine trials

FDA's rare disease framework could center patient identification

AATD gene editing race hinges on patient identification

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

Autoimmune cell therapy pauses expose stratification gaps

FDA draft guidance expands single-trial effectiveness pathway

Genomic newborn screening: The operational challenge ahead

OptiPrime speeds prime editing but patient identification lags

Inherited genetics linked to CAR-T toxicity and expansion

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

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