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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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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Rare disease trials are among the most challenging to design and execute. Patient populations are small and hard to reach, and personal and disease-related burd...
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On the most recent episode of The Genetics Podcast, we hosted Terry Pirovolakis, founder and CEO of Elpida Therapeutics. Terry’s journey began the day his young...
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Natural history studies are central to rare disease research. Rare diseases are defined as conditions that affect less than 1 in 200,000 people in the US or les...
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On the latest episode of The Genetics Podcast, Patrick speaks with Max Bronstein, founder and CEO of Aviva Strategies, a health policy firm dedicated to support...
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Genetic medicines have been advancing at an extraordinary pace. Following the first wave of adeno-associated virus (AAV)–based gene therapies, the toolbox for g...
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In rare disease, families are often forced into roles that extend far beyond caregiving. When faced with diseases for which no treatment exists, parents have of...
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In recent years, the therapeutic landscape for genetic hearing loss (GHL) has advanced rapidly, with several programs reaching clinical development. However, tr...
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In the latest episode of The Genetics Podcast, we sat down with Dr. Benny Sorenson, CEO of Hemab Therapeutics, to explore a relatively overlooked therapeutic ar...
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