blog-icon

Clinical research blog

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

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...
Continue reading

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...
Continue reading

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...
Continue reading

Redefining accessibility in cell and gene therapies

Cell and gene therapy continues to advance across a growing number of rare genetic conditions. As more of these therapies move closer to the clinic, a central t...
Continue reading

The infrastructure gaps biopharma misses in rare and genetic trials

Rare and genetic programs increasingly depend on healthcare systems that are still adapting to the demands of precision medicine. Many of the constraints that a...
Continue reading

The forces shaping regulatory expectations in genomics-driven pharma

Regulatory expectations in genomics-driven drug development are evolving alongside scientific practice, clinical implementation, and policy. While formal guidan...
Continue reading

Podcast recap: Lisa Gurry on scaling early genetic diagnosis at GeneDx

In the latest episode of The Genetics Podcast, Patrick spoke with Lisa Gurry, Chief Business Officer at GeneDx. The conversation focuses on how GeneDx has built...
Continue reading

Podcast recap: Jeffrey Chamberlain on four decades of progress in gene therapy for muscular dystrophy

In the latest episode of The Genetics Podcast, Patrick spoke with Dr. Jeffrey Chamberlain, Professor at the University of Washington School of Medicine and Dire...
Continue reading

Top five regulatory signals sponsors can act on now

The FDA’s activity in 2025 reflected a mix of scientific progress and operational instability. Precision medicine programs benefitted from new draft guidance, r...
Continue reading

Podcast recap: Rachel Salzman on vectorized RNAi for neuromuscular disease

In the last episode of The Genetics Podcast, Patrick spoke with Dr. Rachel Salzman, CEO of Armatus Bio, a biotech company developing vectorized RNA interference...
Continue reading