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PODCAST

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.

business

All Episodes

00:22:00

Expanding Access to Whole Genome Sequencing Across...

RARECast ·

2022/01/06

Ryan Taft, vice president of scientific research for Illumina, discusses the growing case for expanded use of genome sequencing as a diagnostic tool, the iHope Genetic Health program, and its efforts to expand use of the technology in low- and middle-income communities around the globe.

en

00:37:36

A First-in-Class Approach to Treating a Rare and...

RARECast ·

2021/12/31

Sujal Shah, president and CEO of CymaBay Therapeutics, discusses the rare and chronic livers disease primary biliary cholangitis, Seladelpar, and why this first-in-class therapy has promise for addressing a condition with high unmet needs.

en

00:28:41

A New Approach to Treating a Rare Endocrine Disorder

RARECast ·

2021/12/24

David Katz, founder and chief scientific officer of Sparrow Pharmaceuticals, discusses the company’s efforts to develop approach to treating Cushing’s syndrome, how it works, and why this has the potential to address the unmet needs of patients with endogenous Cushing’s syndrome.

en

00:29:01

Detecting Genetic Disease Prior to Birth

RARECast ·

2021/12/16

Paul Billings, chief medical officer for Natera, discusses the state of non-invasive prenatal testing, the growing use of these tests, and the range of conditions they can detect.

en

00:44:56

Turning Words into Action: Equity, Diversity, and...

RARECast ·

2021/12/10

Eve Dryer, vice president of patient advocacy for Travere Therapeutics, discusses why Travere has focused on equity, diversity, and inclusion issues; the work it is doing in this area, and why it is such a critical issue for the rare disease community.

en

00:26:39

Chasing Cures

RARECast ·

2021/12/03

Gaurav Shah, CEO of Rocket, discusses his company’s approach to gene therapy, the conditions it’s targeting, and how it determines what gene therapies it will pursue.

en

00:24:52

A Map to Navigate A Rare Disease

RARECast ·

2021/11/24

TSC Alliance CEO Kari Rosbeck and TSC Alliance Director of Medical Affairs Ashley Pounders, discuss the organization’s TSC Navigator, the thinking behind it, and why it could serve as a model for other patient organizations to follow.

en

00:20:58

A Popular Cancer Target Is Eyed for a Rare Immune...

RARECast ·

2021/11/19

Anurag Relan, chief medical officer of Pharming, discusses the rare immune condition APDS, the role PI3K inhibitors can play in treating it, and why a potential drug in development to treat the condition may have broader use in autoimmune and inflammatory diseases.

en

00:45:49

Leveraging a Gene Therapy Approach to Maximize Speed...

RARECast ·

2021/11/12

R.A. Session II, founder and CEO of Taysha Gene Therapies, discusses the origins of his company, its unique relationship with gene therapy innovator Steve Gray and UT Southwestern, and the critical role patient organization have played in the process.

en

00:37:03

Programing the Body to Make Its Own Medicine with...

RARECast ·

2021/11/04

Ken Mills, CEO of RegenxBio, discusses wet-age macular degeneration, the company’s collaboration with AbbVie, and the potential to alter the way patients with this and other eye conditions are treated by using a gene therapy that gets the body to produce a VEGF inhibitor.

en

00:18:48

Developing in Vivo Gene Editors that Target Liver...

RARECast ·

2021/10/28

Joe Truitt, CEO of iECURE, discusses his company’s in vivo gene editing therapies, its focus on liver diseases, and how it’s leveraging its partnership with the University of Pennsylvania’s Gene Therapy Program.

en

00:31:12

A Find-and-Replace Approach to Fixing the Mutation...

RARECast ·

2021/10/21

Josh Lehrer, CEO of Graphite Bio, discusses his company’s experimental sickle cell gene editing therapy, how it works, and what makes it a next-generation gene editor. @globalgenes #RARECast

en

00:21:28

A Bet on a Unique Set of Vectors

RARECast ·

2021/10/15

Arthur Tzianabos, CEO of Homology Medicines, discusses the company’s genetic therapies, its program in PKU, and how it pairs its vectors and approach to meet the needs of a given condition. @globalgenes #RARECast

en

00:50:21

How a Rare Diagnosis Impacts a Family

RARECast ·

2021/10/07

Jon and Shirley Dicks discuss the diagnostic odyssey for their daughter Elle, how having a diagnosis changed care for her, and the challenges they have faced caring for a child with a rare condition.

en

00:30:50

Transforming the Rare Disease Landscape with Data

RARECast ·

2021/10/01

Nancy Yu, co-founder and CEO of AllStripes, discusses the growing efforts around the collection of patient data, where AllStripes fits into this emerging landscape, and how data can transform the outlook for diagnosing and treating people with rare diseases.

en

00:32:46

Creating a Playbook for Bespoke Gene Therapies

RARECast ·

2021/09/24

P.J. Brooks, deputy director of the Office for Rare Diseases Research at NCATS, discusses the newly created Bespoke Gene Therapy Consortium, why it is looking beyond the translational science challenges of gene therapies to include manufacturing and regulation, and how it hopes to accelerate the development of gene therapies for rare diseases.

en

00:25:16

Finding Answers for Undiagnosed Patients with Rare...

RARECast ·

2021/09/16

Matthew Bainbridge, principal investigator and associate director of clinical genomics at Rady Children’s Institute for Genomic Medicine, discusses his organization’s collaboration with Pacific Biosciences to use long-read whole genome sequencing to diagnose patients with rare diseases, how the technology differs from traditional whole genome sequencing, and why this is helping to find answers for undiagnosed patients with rare genetic diseases.

en

00:22:39

How a Familiar Face Can Lead to a Rare Disease Diagnosis

RARECast ·

2021/09/10

Marius George Linguraru, who led a Children's National Hospital research team that developed digital biometric analysis software to help diagnose rare disease patients, discusses the diagnostic tool he developed, how it works, and a deal with a newly formed company to commercialize the technology.

en

00:27:24

Bringing the Trial to the Patient

RARECast ·

2021/09/02

Jonathan Cotliar, chief medical officer of Science 37, discusses the move toward decentralized clinical trials, how technology is changing the types of data that can be gathered, and how it is forever reshaping clinical studies.

en

00:23:33

Targeting Rare and Chronic Kidney Diseases

RARECast ·

2021/08/26

Tom Frohlich, chief operating officer of Chinook Therapeutics, about rare kidney diseases, the company’s lead therapeutic candidate atrasentan for IgA nephropathy, and the company’s plans for commercialization.

en

568 results

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