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Clinical research blog

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

Podcast recap: Ellen Reisinger on gene therapy for hereditary deafness

In the last episode of The Genetics Podcast, we spoke with Dr. Ellen Reisinger, Professor at the University of Tübingen. Ellen has spent nearly twenty years stu...
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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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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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From fragmentation to fusion: How AI is unlocking the value of multi-omic data in drug discovery

In drug discovery, data is the key to success. Although more and more large-scale data is being generated every year, much of it is inaccessible or fragmented. ...
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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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Innovation in AAV: Breaking through bottlenecks in yield, safety, and cost

At this year’s European Society of Gene & Cell Therapy (ESGCT) meeting, one theme resonated across sessions: adeno-associated viruses (AAVs) are entering a ...
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Podcast recap: The state of AAV gene therapy and where it’s headed

On the most recent episode of The Genetics Podcast, host Patrick Short took a solo deep dive into gene therapy, with a focus on therapies based on delivery via ...
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Podcast recap: Sonya Dumanis and Andy Singleton on building a global genetics engine for Parkinson’s

On the most recent episode of The Genetics Podcast, we hosted Sonya Dumanis and Andy Singleton to unpack GP2, the Global Parkinson’s Genetics Program. GP2 is a ...
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