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Clinical research blog

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

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...
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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...
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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 Precision medicine programs benefitted from new draft guidance, r...
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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...
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3 recent signals that hint at the next phase of rare disease development

Recent activity in the US and UK offers important signals for how personalized and rare disease therapies may evolve. Scientific teams are building structured e...
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Cell and gene therapy: Systems gaps shaping next scale

At the recent Evolution of the Cell and Gene Therapy (CGT) Sector Workshop by the Alliance for Regenerative Medicine (ARM), speakers from across industry, acade...
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Podcast recap: Ananth Sridhar and Sun-Gou Ji on building a sustainable model for rare disease innovation

On the latest episode of The Genetics Podcast, we spoke with Ananth Sridhar, Chief Operating Officer for cardiorenal programs at BridgeBio, and Sun-Gou Ji, the ...
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Podcast recap: Justin Porcano on speeding up treatments for Usher syndrome type 1B

In the last episode of The Genetics Podcast, Patrick spoke with Justin Porcano, co-founder and executive director of Save Sight Now, a nonprofit accelerating th...
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Scaling the promise of gene editing: Why progress still falls short of potential

Gene editing has progressed significantly over the past decade, evolving from an experimental technology that was associated with safety and efficacy limitation...
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Podcast recap: Chris Hopkins on developing a one-time cell therapy for Fabry disease

On this episode of The Genetics Podcast, Patrick spoke with Chris Hopkins, CEO of Glafabra Therapeutics, a company developing a one-time autologous cell therapy...
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