How many organs could you donate and remain alive? How many planet Earths could fit inside the Sun? How high is a giraffe's blood pressure? Why is the sea blue? To find out, Ask The Naked Scientists!
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EP 212: A hub-and-spoke model for accelerating rare disease drug development with Ananth Sridhar and Sun-Gou Ji of BridgeBio
Manage episode 517985624 series 2631947
Content provided by Sano Genetics. All podcast content including episodes, graphics, and podcast descriptions are uploaded and provided directly by Sano Genetics or their podcast platform partner. If you believe someone is using your copyrighted work without your permission, you can follow the process outlined here https://podcastplayer.com/legal.
Summary:
This week on The Genetics Podcast, Patrick is joined by BridgeBio’s Ananth Sridhar, Chief Operating Officer of Cardiorenal Programs, and Sun-Gou Ji, Vice President of Computational Genomics. They discuss the hub-and-spoke model for de-risking and accelerating rare disease drug development, the role of human genetics in target discovery, lessons from their autosomal dominant hypocalcemia type 1 (ADH1) program, and how portfolio design and predictive genomics are shaping the future of precision medicine.
Show Notes:
0:00 Intro to The Genetics Podcast
00:59 Welcome to Ananth & Sun-Gou
01:57 How BridgeBio’s hub-and-spoke model de-risks and accelerates rare disease drug development
06:45 How programs move from the hub to the spokes in target discovery and development
09:10 Translating a target into a therapy for autosomal dominant hypocalcemia type 1 (ADH1)
12:28 Challenges in ADH1 drug development and using population genetics to identify patients with gain-of-function variants
18:08 Under-diagnosis and incomplete penetrance in rare disease and quantifying genetic versus phenotypic prevalence
20:52 Balancing first-in-class innovation with risk management in rare disease drug development
24:24 Evaluating rare disease programs using risk-adjusted net present value (NPV) instead of peak sales
27:20 Key factors that can make rare disease programs faster and cheaper to develop, and why modality agnosticism is important
32:00 Sun-Gou’s experience in computational genetics and building data-driven infrastructure for discovery
36:44 Ananth’s lessons from Regeneron and applying patient-centered principles to rare disease drug development
39:00 Sun-Gou on the power of newborn sequencing and personal lessons from early diagnosis
43:36 Ananth’s views on making predictive medicine more personal and human-centered
44:51 Closing remarks
Find out more
BridgeBio (https://bridgebio.com/unlocking-rare-diseases-medicine)
Hub-and-spoke model (https://www.youtube.com/watch?v=gRURL49QsX4)
ADH1 publication (https://www.cell.com/ajhg/fulltext/S0002-9297(25)00244-7)
Please consider rating and reviewing us on your chosen podcast listening platform!
https://drive.google.com/file/d/1Bp2_wVNSzntTs_zuoizU8bX1dvao4jfj/view?usp=share_link
215 episodes
Share
Manage episode 517985624 series 2631947
Content provided by Sano Genetics. All podcast content including episodes, graphics, and podcast descriptions are uploaded and provided directly by Sano Genetics or their podcast platform partner. If you believe someone is using your copyrighted work without your permission, you can follow the process outlined here https://podcastplayer.com/legal.
Summary:
This week on The Genetics Podcast, Patrick is joined by BridgeBio’s Ananth Sridhar, Chief Operating Officer of Cardiorenal Programs, and Sun-Gou Ji, Vice President of Computational Genomics. They discuss the hub-and-spoke model for de-risking and accelerating rare disease drug development, the role of human genetics in target discovery, lessons from their autosomal dominant hypocalcemia type 1 (ADH1) program, and how portfolio design and predictive genomics are shaping the future of precision medicine.
Show Notes:
0:00 Intro to The Genetics Podcast
00:59 Welcome to Ananth & Sun-Gou
01:57 How BridgeBio’s hub-and-spoke model de-risks and accelerates rare disease drug development
06:45 How programs move from the hub to the spokes in target discovery and development
09:10 Translating a target into a therapy for autosomal dominant hypocalcemia type 1 (ADH1)
12:28 Challenges in ADH1 drug development and using population genetics to identify patients with gain-of-function variants
18:08 Under-diagnosis and incomplete penetrance in rare disease and quantifying genetic versus phenotypic prevalence
20:52 Balancing first-in-class innovation with risk management in rare disease drug development
24:24 Evaluating rare disease programs using risk-adjusted net present value (NPV) instead of peak sales
27:20 Key factors that can make rare disease programs faster and cheaper to develop, and why modality agnosticism is important
32:00 Sun-Gou’s experience in computational genetics and building data-driven infrastructure for discovery
36:44 Ananth’s lessons from Regeneron and applying patient-centered principles to rare disease drug development
39:00 Sun-Gou on the power of newborn sequencing and personal lessons from early diagnosis
43:36 Ananth’s views on making predictive medicine more personal and human-centered
44:51 Closing remarks
Find out more
BridgeBio (https://bridgebio.com/unlocking-rare-diseases-medicine)
Hub-and-spoke model (https://www.youtube.com/watch?v=gRURL49QsX4)
ADH1 publication (https://www.cell.com/ajhg/fulltext/S0002-9297(25)00244-7)
Please consider rating and reviewing us on your chosen podcast listening platform!
https://drive.google.com/file/d/1Bp2_wVNSzntTs_zuoizU8bX1dvao4jfj/view?usp=share_link
215 episodes
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Similar to The Genetics Podcast
How many organs could you donate and remain alive? How many planet Earths could fit inside the Sun? How high is a giraffe's blood pressure? Why is the sea blue? To find out, Ask The Naked Scientists!
…
continue reading
I discuss a variety of topics in both the natural and social sciences, exploring the many fascinating insights that the scientific method yields about the world around us.
…
continue reading
Whether the topic is popcorn or particle physics, you can count on BrainStuff to explore -- and explain -- the everyday science in the world around us.
…
continue reading
The Science Show gives Australians unique insights into the latest scientific research and debate, from the physics of cricket to prime ministerial biorhythms.
…
continue reading
The kickass science and technology radio show that delivers an irreverent look at the week in science and technology.
…
continue reading
When you hear the words Alzheimer's disease, what do you think of? The truth is, the picture most of us have of the disease is incomplete. Alzheimer's disease doesn't start when someone starts to lose their memory. It actually starts years – sometimes decades – earlier. The Rethinking Alzheimer's Disease Podcast is an engaging, narrative-style podcast miniseries for those curious or motivated to learn about Alzheimer’s disease. Perhaps you have a family member with Alzheimer’s disease, or ca ...
…
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Flash Forward is a show about possible (and not so possible) future scenarios. What would the warranty on a sex robot look like? How would diplomacy work if we couldn’t lie? Could there ever be a fecal transplant black market? (Complicated, it wouldn’t, and yes, respectively, in case you’re curious.) Hosted and produced by award winning science journalist Rose Eveleth, each episode combines audio drama and journalism to go deep on potential tomorrows, and uncovers what those futures might re ...
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continue reading
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continue reading
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