Sano blog

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...

The Genetics Podcast featuring Danny Miller

podcast recap

Podcast recap: Danny Miller on making long-read sequencing the first test every patient receives

Most patients with a suspected genetic condition still move through testing one layer at a time. A panel checks a set of genes, a separate assay looks for a rep...

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FDA draft guidance expands single-trial effectiveness pathway

In June 2026, the FDA released a revised draft guidance, Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products, that clarif...

podcast recap

Podcast recap: Brent Richards on how genetic evidence can de-risk drug targets

In the latest episode of The Genetics Podcast, Patrick Short sits down with Dr. Brent Richards, CEO and founder of 5 Prime Sciences and Professor of Human Genet...

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...

The Genetics Podcast featuring Andrew Jackson

podcast recap

Podcast recap: Andrew Jackson on how the same DNMT3A mutations behind dwarfism drive an accelerated aging syndrome

In the latest episode of The Genetics Podcast, Patrick Short speaks with Dr. Andrew Jackson, Programme Leader at the MRC Human Genetics Unit within the Universi...

patient recruitment rare disease patient engagement

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...

Clinical research blog

Rare disease recruitment problems start earlier than most sponsors think

Autoimmune cell therapy pauses expose stratification gaps

Podcast recap: Danny Miller on making long-read sequencing the first test every patient receives

FDA draft guidance expands single-trial effectiveness pathway

Podcast recap: Brent Richards on how genetic evidence can de-risk drug targets

Genomic newborn screening: The operational challenge ahead

Podcast recap: Andrew Jackson on how the same DNMT3A mutations behind dwarfism drive an accelerated aging syndrome

Long-term follow-up in clinical trials is a compounding asset for rare disease programs

OptiPrime speeds prime editing but patient identification lags

Podcast recap: Stevie Ringel on building operational capacity for rare disease drug development

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