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Clinical research blog

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

What two FDA gene therapy reversals in one week means for rare disease sponsors

In the space of five days, the FDA reversed its position on two rare disease gene therapies it had previously rejected. On June 17, the agency agreed that UniQu...
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Podcast recap: Sarah Marzi on how genetics, environment, and cell state shape neurodegenerative disease

In the most recent episode of The Genetics Podcast, Patrick Short speaks with Dr. Sarah Marzi, Senior Lecturer at King’s College London and Group Leader at the ...
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Podcast recap: Nicky Whiffin on the discovery of ReNU syndrome and what it unlocks for genetic medicine

The majority of rare disease discovery has been linked to protein coding genes, where changes are easier to interpret and easier to capture in routine testing. ...
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Podcast recap: Arabella Bouzigues on building a global research infrastructure for frontotemporal dementia

In a recent episode of The Genetics Podcast, host Patrick Short talked with Dr. Arabella Bouzigues, postdoctoral researcher and coordinator of the Genetic Front...
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How spatial omics is reshaping biomarker development in neuroscience

Spatial omics have revolutionized biomedical research in recent years. The use of spatial techniques unlocks cell-specific understanding of disease, which suppo...
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Podcast recap: Wanda Smith on turning a family diagnosis into CureGRN

In last week’s episode of The Genetics Podcast, Patrick Short sat down with Wanda Smith, founder of CureGRN and long-time advocate for families affected by prog...
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Decentralized clinical trials: At-home patient screening

Precision medicine trials increasingly depend on genetic or biomarker testing to determine eligibility. Each additional test introduces operational complexity: ...
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Podcast recap: Xin Jin on decoding brain disorders with in vivo Perturb-seq

In the last episode of the Genetics Podcast, Patrick speaks with Dr. Xin Jin, a pioneering neuroscientist at Scripps Research, about her team’s cutting-edge met...
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Podcast recap: Heiko Runz on population genomics and drug discovery

In the latest episode of The Genetics Podcast, we spoke with Dr. Heiko Runz, SVP of Neuroscience at insitro. With a background spanning clinical genetics, acade...
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Podcast recap: Frank Gentile on harnessing autophagy to treat neurodegenerative diseases

In a recent episode of The Genetics Podcast, we talked to Frank Gentile, Chief Executive Officer of Casma Therapeutics, to explore how autophagy could be a valu...
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