Clinical research blog
Explore our blog for insights into the big questions in precision medicine and clinical research.
In a recent episode of The Genetics Podcast, Patrick Short spoke with Dr. Ryan Dhindsa, Assistant Professor at Baylor College of Medicine and Investigator at th...
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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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Adeno-associated virus (AAV) gene therapy has moved from proof of concept to approved products. As more programs enter the clinic and target larger patient popu...
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Cell and gene therapy (CGT) programs are committing to 10-15 years of follow-up – often without the infrastructure to support it. FDA guidance for certain gene ...
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In the latest episode of The Genetics Podcast, we spoke with Dr. Daniel Chung, Chief Medical Officer at Beacon Therapeutics. The conversation focused on how ret...
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Digital recruitment has become a standard part of clinical trial execution. Sponsors invest heavily in online outreach, patient referrals, and awareness campaig...
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Last week, the rare disease field celebrated another breakthrough. A teenage girl with an ultra-rare genetic neurodegenerative disease was treated with a bespok...
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As genetic medicine has continued to advance, experts have consistently highlighted the importance of newborn genomic screening as a practical avenue for early ...
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Patient availability is often overestimated during trial planning. This usually starts at feasibility, where sites are asked to report how many patients they ha...
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At the JPM Healthcare Conference 2026, the Genetics Podcast recorded a special episode from the Flagship Pioneering studio, bringing together two leaders advanc...
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