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Clinical research blog

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

What the FDA's new potency guidance means for rare disease gene therapy trials

On November 2, 2026, the FDA issued its final guidance, Potency Assurance for Cellular and Gene Therapy Products, outlining a risk-based approach to ensuring th...
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Podcast recap: Patrick Short on why verification speed will decide how fast AI transforms biology

AI systems have started producing results on open problems in mathematics, and mathematicians are paying attention. In the most recent episode of The Genetics P...
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FDA's new genome editing guidance: Why patient identification matters earlier in development

The FDA's draft guidance on individualized human genome editing products, issued on 27 October 2026, proposes a pathway that could allow a single biologics lice...
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Podcast recap: Sek Kathiresan on developing gene editing for cardiovascular disease

In the latest episode of The Genetics Podcast, host Patrick Short speaks with Dr. Sekar (Sek) Kathiresan, CEO of Verve Therapeutics and SVP, Head of Obesity Res...
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Population genomic screening is scaling. Is clinical trial infrastructure keeping up?

New findings presented at ASHG 2026 show how population genomic screening is identifying genetic risk at scale and changing preventive care. For sponsors develo...
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Podcast recap: Luke Pilling on why a genotype is only the starting point

In the latest episode of The Genetics Podcast, Patrick Short is joined by Dr. Luke Pilling, Senior Lecturer in Genetic Epidemiology at the University of Exeter....
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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...
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Podcast recap: James Zou on what 37,000 AI agents learned about why drug trials fail

In the latest episode of The Genetics Podcast, host Patrick Short speaks with Dr. James Zou, Associate Professor of Biomedical Data Science at Stanford Universi...
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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...
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Cardiovascular genetic medicine will need a different patient-finding infrastructure

Genetic medicines are changing who cardiovascular trials need to find. Eligibility is increasingly defined by a genotype or a biomarker level, and the people wh...
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