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Clinical research blog

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

Podcast recap: Ryan Dhindsa and Caleb Lareau on using biobank genomics to measure EBV persistence

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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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: David Dismuke and Steven Gray on scaling AAV gene therapy

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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Barriers to long-term follow-up in cell and gene therapy

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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Podcast recap: Daniel Chung on the evolution of retinal gene therapy

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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From click to randomization: What truly predicts trial success?

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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Rethinking drug approval for ultra-rare disease: Inside the UK’s n-of-1 ASO trial

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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Podcast recap: Zornitza Stark on real-world impact from newborn genomic screening 

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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Understanding the gap between feasibility estimates and patient availability

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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Podcast recap: Michelle Werner and Michael Severino on moving engineered tRNA and gene writing into the clinic

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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