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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Most genetic medicine is built for populations large enough to support a clinical trial. N-of-1 medicine is built for the opposite case: a single patient whose ...
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On June 2, 2026, the FDA released draft guidance that could reshape how gene therapies reach patients. The document, "Leveraging Prior Knowledge in the Developm...
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Bipolar disorder has long been recognized as highly heritable, yet the biology underlying the condition has remained difficult to resolve. The genetics are comp...
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Finding patients who qualify for a clinical trial has always been one of the hardest parts of running one. In liver disease, particularly MASLD, it is especiall...
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More than three decades after the Huntington's disease gene was cloned in 1993, there is still no approved therapy that alters the course of the disease. Recent...
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When a clinical trial misses its enrollment targets, the instinct is often to look at recruitment through more channels, more outreach, and a bigger advertising...
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In the most recent episode of The Genetics Podcast, host Patrick Short speaks with Dr. Paul Valdmanis, Associate Professor at the University of Washington, abou...
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What four episodes of The Genetics Podcast reveal about the future of Alzheimer’s precision medicine
Alzheimer’s research is entering a new phase. For decades, the field has been shaped by the biology of amyloid plaques and tau tangles. Those remain central to ...
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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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