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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

00:29:19

BridgeBio Advances Therapy for Limb-Girdle Muscular...

RARECast ·

2023/02/24

Doug Sproule, chief medical officer of ML Bio Solutions, discusses limb girdle muscular dystrophy type 2i, the company’s experimental therapy to treat the condition, and the power of rare disease patient families to shape drug development.

en

00:36:53

Translating Urgency into Science

RARECast ·

2023/02/16

Joni Rutter, director of the National Center for Advancing Translational Sciences, discusses NCATS’ priorities under her leadership, the challenges of translational science, and where she sees the biggest opportunities for accelerating the discovery and development of therapies for rare diseases.

en

00:33:28

Empowering Ultra-Rare Disease Patients to Pursue the...

RARECast ·

2023/02/10

Casey McPherson, chief innovation officer of Everlum Bio, discusses what led him to create the company, its “rare-disease-lab-as-a-service model, and how he is working to change the discovery landscape for ultra-rare disease therapies.

en

00:20:03

Why a Topical Cannabidiol Gel May Help Treat the...

RARECast ·

2023/02/02

Co-director of the molecular diagnostics section of the Genetic Laboratory at Rush Medical College Elizabeth Berry-Kravis and Zynerba Chairman and CEO Armando Anido, discuss Fragile X, the company’s experimental therapy Zygel, and why the topical cannibidol gel may hold promise for treating the behavioral symptoms of the condition.

en

00:25:49

An Ultra-Rare Disease Drug Developer Tries to...

RARECast ·

2023/01/26

Reenie McCarthy, CEO of Stealth Biotherapeutics, discusses the challenges the company has faced in seeking FDA approval for its experimental therapy to treat the rare and life-threatening condition Barth syndrome, the lack of consistency the company found from division to division within the agency, and why this could have a chilling effect on the development of ultra-rare disease therapies if left unaddressed.

en

00:16:51

Powering Cells in People with Rare Mitochondrial...

RARECast ·

2023/01/19

Greg Flesher, president and CEO of Reneo Pharmaceuticals, discusses mitochondrial myopathies, the company’s efforts to develop an experimental therapy to treat these conditions, and how it works.

en

00:27:25

Powering Weakened and Stressed Cells in ALS to...

RARECast ·

2023/01/12

Rob Etherington, president and CEO of Clene Nanomedicine, discusses the neurodegenerative condition ALS, the role that the compromised ability of cells to produce energy play in the disorder, and why the company believes its gold nanocrystal therapy has the potential to improve function in people with the condition.

en

00:29:21

Developing a New Class of Therapies Based on a...

RARECast ·

2023/01/05

Tony de Fougerolles, CEO of Evox Therapeutics, discusses exosomes, the company’s platform technology to load these natural transport vehicles with therapeutic cargo, and how it is using this approach to target a range of rare diseases.

en

00:36:05

Reaching Beyond the Limits of Enzyme Replacement...

RARECast ·

2022/12/29

Geoff MacKay, president and CEO of Avrobio, discusses cystinosis, the company’s gene therapy platform, and how it's leveraging its technology to develop therapies across a range of rare diseases.

en

00:26:09

One Woman’s Journey as a Caregiver to a Husband with...

RARECast ·

2022/12/22

Deb Scharper, a frontotemporal dementia caregiver and advocate, discusses the impact of the condition on her family, her experience as a caregiver, and why she has become an advocate who organizes support groups for other caregivers of people with the condition.

en

00:23:38

Treating Rare Endocrine Disorders with Therapeutic...

RARECast ·

2022/12/15

Mark Sumeray, chief medical officer of Amolyt, discusses what it’s like for people living with the hypoparathyroidism, why it is difficult to manage with current medical approaches, and why therapeutic peptides offer a compelling approach for hypothyroidism and other endocrine disorders.

en

00:33:29

Tackling the Pricing Challenges for Advanced...

RARECast ·

2022/12/08

Alice Valder Curran, partner with Hogan Lovells, discusses the challenges of value-based pricing for gene therapies, some of the pricing approaches developers are employing, and how the existing policy landscape complicates matters.

en

00:16:37

Addressing the Current Limitations of AAV Gene Therapies

RARECast ·

2022/12/01

Joseph La Barge, CEO of Apertura, about its platform technologies, how they work, and the potential for next-generation gene therapies to transcend the limits of first-generation AAV therapies.

en

00:29:33

Examining the Legislative Landscape for Rare Disease...

RARECast ·

2022/11/24

Amanda Malakoff, executive director of the Rare Disease Company Coalition, discusses the policy landscape for rare disease therapies, unfinished business from the recent passage of a lean Prescription Drug User Fee Act, and policy priorities for 2023.

en

00:35:09

How a Drug Setback Became a Patient Community’s Gain

RARECast ·

2022/11/18

Ovid CEO Jeremy Levin and Angelman Syndrome Foundation CEO Amanda Moore discuss the LADDER database, Ovid’s decision to contribute its data to it, and why the two believe other drug developers should take similar steps to share their data with patients and researchers to advance the understanding of rare diseases.

en

00:25:08

Helping Regulators and Drug Developers Understand the...

RARECast ·

2022/11/10

Jack Johnson, co-founder of the Fabry Support and Information Group, discusses his experience with the condition, his journey into advocacy, and a recent externally-led Patient-Focused Drug Development meeting to help regulators and drug developers understand the need for new therapies to address the challenges of living with the disease.

en

00:32:57

Addressing Racial Disparities in a Rare Blood Cancer

RARECast ·

2022/11/03

Kyowa Kirin Vice President of Public Affairs Lauren Walrath and Co-Leader of the Immune Cell Regulation and Targeting Program at the Sidney Kimmel Cancer Center at Jefferson Health Pierluigi Porcu, discuss cancer cutaneous T-cell lymphoma, the disparities in care and outcomes for African Americans with the condition, and what they are doing to address that.

en

00:27:04

How New Sequencing Technology Promises to Alter the...

RARECast ·

2022/10/27

Tomi Pastinen, director of the Genomic Medicine Center at Children’s Mercy Kansas City, discusses the Genomic Answers for Kids program, how new sequencing technology is allowing it to diagnose rare disease patients who previously were undiagnosable, and how it has the potential to alter the diagnostic odyssey for patients with rare, genetic diseases.

en

00:25:39

Leveraging Technology to Empower Patients and...

RARECast ·

2022/10/20

Gabriele Brambilla, CEO and co-founder of Alira Health, about how Health Storylines platform to provide patients with greater control over their own health, how it is using the technology to drive decentralized clinical trials and the integration of real-world evidence in the drug development process, and the potential to leverage the platform to answer research questions outside of a traditional clinical study.

en

00:22:43

Equipping The Next-Generation Rare Disease Patient...

RARECast ·

2022/10/13

Charlene Son Rigby, who will take the helm of Global Genes following its merger with RARE-X, discusses the integration of the two organizations, the convergence of her personal and professional lives, and how the combination of the two organization will provide next-generation rare disease advocates the tools and resources they need to accelerate their drive for treatments.

en

566 results

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