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Clinical research blog

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

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...
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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...
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Podcast recap: Inigo Martincorena on somatic evolution in healthy tissue

Every cell in the body picks up changes to its DNA over a lifetime. For most of the history of cancer genomics, researchers could only read those changes once a...
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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...
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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...
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Podcast recap: Marleah Dean Kruzel on reframing hereditary cancer uncertainty

In the latest episode of The Genetics Podcast, Patrick sits down with Dr. Marleah Dean Kruzel, Professor of Communication at the University of South Florida. He...
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Podcast recap: Dave Hallett on cutting through the AI hype in drug discovery

In the most recent episode of The Genetics Podcast, Patrick Short sat down with Dr. Dave Hallett, Chief Scientific Officer at Recursion. Dave has spent nearly t...
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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.
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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...
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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...
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