Sano blog | Lisa Conroy, MPH

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...

press patient recruitment neurodegenerative disease patient engagement

Parkinson’s patients first to benefit from Sano Genetics and PKB’s new partnership

Sano Genetics and Patients Know Best (PKB) are excited to announce a new partnership to streamline and accelerate clinical trial recruitment processes, launchin...

patient recruitment rare disease genetic testing neurodegenerative disease

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...

Rare disease recruitment problems start earlier than most sponsors think

Rare disease trials have a recruitment problem, but the problem often begins well before recruitment starts.

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FDA draft guidance expands single-trial effectiveness pathway

In June 2026, the FDA released a revised draft guidance, Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products, that clarif...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

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