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RARECast
RARECast
577 episodes
4 days ago

RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.

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Business
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All content for RARECast is the property of RARECast and is served directly from their servers with no modification, redirects, or rehosting. The podcast is not affiliated with or endorsed by Podjoint in any way.

RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.

Show more...
Business
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Developing a New Approach to Cystic Fibrosis to Improve Outcomes
RARECast
29 minutes 31 seconds
3 weeks ago
Developing a New Approach to Cystic Fibrosis to Improve Outcomes
There have been great advances in the treatment of cystic fibrosis, but nevertheless it remains a progressive, life‑shortening genetic disease as many patients still don’t reach normal function and continue to face infections, exacerbations, and impaired quality of life. Siona Therapeutics is building a new generation of small‑molecule therapies that directly stabilize the region most affected by the most common mutation underlying the disease for the roughly 90 percent of patients who carry the defect. We spoke to Mike Cloonan, CEO of Sionna, about its experimental therapy that stabilizes the misfolded protein at the heart of the disease, its exploration of combinations with existing and proprietary therapies, and its $219 million IPO in February, despite the difficult financing environment for development-stage biotechs.
RARECast

RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.