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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In June 2026, the FDA released a revised draft guidance, Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products, that clarif...
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In the latest episode of The Genetics Podcast, Patrick Short sits down with Dr. Brent Richards, CEO and founder of 5 Prime Sciences and Professor of Human Genet...
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A Nature News Feature published on 19 August 2026 synthesized the global wave of genomic newborn screening programs and opened with a concrete result. In the GU...
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In the latest episode of The Genetics Podcast, Patrick Short speaks with Dr. Andrew Jackson, Programme Leader at the MRC Human Genetics Unit within the Universi...
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Rare disease trials demand years of investment to identify, genetically screen, enroll, and engage patients. When the study closes, that effort disperses. Commu...
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On August 12, 2026, the David Liu lab at the Broad Institute published OptiPrime, a machine-learning model that predicts how well prime-editing guide RNAs (pegR...
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Rare and ultra-rare disease research has produced real scientific breakthroughs over the past decade: antisense oligonucleotides, gene therapies, and gene editi...
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Cell and gene therapy has solved problems that seemed intractable a decade ago. The science of editing genomes, replacing defective genes, and engineering patie...
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CAR-T therapies carry meaningful safety risks, and toxicity is known to vary with factors including disease characteristics, patient factors and the therapy its...
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