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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

00:43:37

Using AI to De-Risk Rare Disease Drug Development

RARECast ·

2025/01/16

Tim Guilliams, co-founder and CEO of Healx, discusses the origin of the company, its lead asset in development for neurofibromatosis type 1, and how he views the company’s progress to date.

en

00:30:39

Addressing a Growing Demand for Plasma-Derived Therapies

RARECast ·

2025/01/09

Joerg Schuettrumpf, chief scientific innovation officer at Grifols, discusses the changing landscape for primary immunodeficiency disorders, the challenges of ensuring a reliable supply of plasma-derived therapies, and how treatment options may evolve.

en

00:43:19

Empowering Patients with Data to Drive Drug Development

RARECast ·

2025/01/02

Citizen Health Co-Founders Farid Vij and Nasha Fitter discuss the evolution of the rare patient data platform Citizen Health, what it will enable, and how it is helping shift power towards patients and their caregivers in the pursuit of new therapies.

en

00:27:06

How Advocates Are Advancing a Treatment for an...

RARECast ·

2024/12/26

Dan Williams, co-founder and CEO of SynaptixBio, discusses the ultra-rare and deadly leukodystrophy H-ABC, how the company’s experimental therapy works, and the role patient advocates have played in the company’s efforts to advance its experimental therapy.

en

00:31:53

Engineering B cells to Produce Therapeutic Proteins

RARECast ·

2024/12/19

Sean Ainsworth, CEO of Immusoft, about MPS I, the limits of existing enzyme replacement therapies, and the potential benefits of using engineered B cells to treat people with the rare lysosomal storage disorder and other conditions.

en

00:28:15

A Pipeline Win as BridgeBio Learns to Balances Theory...

RARECast ·

2024/12/12

Neil Kumar, co-founder and CEO of BridgeBio, discusses the approval of its ATTR-CM therapy Attruby, the company’s late-stage pipeline, and what he’s learned about its business model in the process.

en

00:36:05

Considering What It Will Take for Payers to Embrace...

RARECast ·

2024/12/05

Tomi Pastinen, director of the Center for Pediatric Genomic Medicine at Children’s Mercy Kansas City, discusses the success of the Genomic Answers for Kids program to date, reimbursement challenges, and the future of the technology.

en

00:38:07

Finding Strength

RARECast ·

2024/11/27

Friedreich's ataxia patient advocate Anthony Monaco discusses coming to terms with his diagnosis, how he was forced to abandon some dreams, and how he was able to find new ones to pursue.

en

00:35:17

A Non-Profit Works to Advance a DMD Gene Therapy

RARECast ·

2024/11/21

Frederic Revah, CEO of Genethon, discusses Duchenne muscular dystrophy, the organization’s efforts to complete clinical development of its gene therapy for the condition, and how it might commercialize the treatment.

en

00:47:17

Accepting What We Can’t Understand

RARECast ·

2024/11/14

James Robinson, author of More than We Expected: Five Years with a Remarkable Child, discusses his experience as the father of a child with a rare disease, life in hospital wards across two continents, and the feeling of pride in his son that remains.

en

00:49:03

A Gene Therapy Company Born from a Father’s Efforts...

RARECast ·

2024/11/07

Noam Baumatz, co-founder and CEO of Noga Therapeutics, discusses his experience with a daughter born with a rare immunodeficiency, how that experience led to the founding of the gene therapy company Noga Therapeutics, and the company’s business decision to pursue both rare and common diseases.

en

00:26:54

Lowering Barriers to Clinical Trial Participation...

RARECast ·

2024/10/31

en

00:31:32

Scaling N-of-1 Therapies to Viability

RARECast ·

2024/10/24

Kent Rogers, CEO of EveryONE Medicine, discusses the challenges of building a sustainable business model for the development of individualized therapies, the regulatory hurdles it may face, and what it will take to get payers to embrace such medicines.

en

00:40:08

Building Gene Therapies that Address Gain and Loss of...

RARECast ·

2024/10/17

Guang Qu, chief operating officer and co-founder of NGGT, discusses the company’s dual-functional vectors that simultaneously remove harmful, mutated genes and replace them with normal, healthy genes to restore cellular function. https://globalgenes.org/raredaily/building-gene-therapies-that-address-gain-and-loss-of-function-simultaneously/

en

00:31:30

How One Woman Climbed from Patient to Advocate

RARECast ·

2024/10/10

Allison Freedman discusses her multiple myeloma journey through diagnosis and treatment, her recovery that culminated in her scaling Mount Kilimanjaro in Tanzania, and why she went from not wanting to talk about her condition to being a patient advocate.

en

00:27:55

Shortening the Diagnostic Odyssey

RARECast ·

2024/10/03

Katherine Stueland, CEO of GeneDx, discusses the state of genetic testing, what its 2022 acquisition by the AI-drive genomics company SEMA4 has meant to it, and what she thinks it will take to make meaningful change to the diagnostic odyssey.

en

00:22:00

Making Exomes More Revealing

RARECast ·

2024/09/26

Brigette Tippin Davis, chief operating officer for Ambry Genetics, discusses the diagnostic odyssey for people with a rare disease, Ambry’s new ExomeReveal test, and what people can do to accelerate their path to a diagnosis.

en

00:34:27

How One Patient Organization Catalyzed Drug Development

RARECast ·

2024/09/19

Monica Coenraads diseases her experience with Rett syndrome as a mother of a daughter with the condition, how she crafted a scientific agenda for the advocacy organizations she founded, and what other rare disease organizations can learn from her experience.

en

00:39:56

Enabling On-Demand Therapies

RARECast ·

2024/09/16

Chris Hart, co-founder, president and CEO of Creyon Bio; discusses the proof-of-concept achieved with its recent N-of-1 therapy, the business model for Creyon, and the potential for its approach to reduce the time and cost of drug development.

en

00:30:48

After Driving an N-of-1 Therapy for Her Son, an...

RARECast ·

2024/09/05

Yiwei She, founder of the TNPO2 Foundation, discusses how her family was able to treat her son Leo with an experimental ASO with relative speed, the work the TNPO2 Foundation is doing to accelerate the diagnosis of other children with ultra-rare conditions, and its efforts to find accessible and affordable pathways to treatments for others.

en

566 results

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