When Trace Neuroscience announced on June 22 that first patients had been dosed in the LAUNCH ALS trial, it felt like the right moment to revisit our original p...
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On June 23, 2026, the US Department of Health and Human Services launched Operation Trailblazer, a cross-agency initiative coordinated across the FDA, NCI, NCAT...
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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On June 2, 2026, the FDA released draft guidance that could reshape how gene therapies reach patients.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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