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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

00:26:10

Using CRISPR to Modulate Gene Expression

RARECast ·

2025/05/29

Amber Salzman, CEO of Epicrispr Biotechnologies, discusses the company’s one-and-done therapies work to target the epigenome, its lead program in FSHD, and the broader applications for its therapeutic approach.

en

00:20:14

Satisfying the Hunger for a Prader-Willi Therapy

RARECast ·

2025/05/22

Anish Bhatnagar, CEO of Soleno Therapeutics, discusses Prader-Willi syndrome, the company’s therapy to treat the condition, and the significance of having a medicine that can address hyperphagia associated with the disorder.

en

00:22:50

How an Academic Medical Center Helped Change the...

RARECast ·

2025/05/15

Bart Duell, a professor of medicine at Oregon Health Sciences University, discusses the rare metabolic disease CTX, the role OHSU played in developing a diagnostic and advancing a treatment for CTX, and the critical role academic medical centers play in working with patient advocates and drug developers to address the needs of people with rare diseases.

en

00:22:09

Improving Outcomes for People with a Set of Rare Cancers

RARECast ·

2025/05/08

Rachna Shroff, associate director of clinical investigations of the gastrointestinal clinical research team at the University of Arizona Cancer Center, discusses biliary tract cancers, the importance of biomarker testing, and what can be done to improve outcomes for people with these rare cancers.

en

00:27:32

An Effort to Build a Better DMD Gene Therapy

RARECast ·

2025/05/02

Curran Simpson, CEO of Regenxbio, discusses the company’s platform technology, the advantages he sees with its experimental DMD gene therapy, and how a partnership announced at the start of 2025 focused on a pair of other gene therapies provided a welcome alternative to tapping the capital markets.

en

00:32:48

A Once-Failed Pain Therapy Shows Potential in a...

RARECast ·

2025/04/24

Bruce Leuchter, president and CEO of GRIN Therapeutics, discusses GRIN-related disorders, how the company came to develop radiprodil, and how it represents an effort by Neurvati Neurosciences to bring a portfolio approach to developing novel treatments for neurological and psychiatric disorders.

en

00:25:12

Advancing a Cutting-Edge Therapy for a Rare,...

RARECast ·

2025/04/17

Paul Romness, president and CEO of OS Therapies, discusses why osteosarcomas have been difficult to treat, the company’s experimental therapy for the condition, and the promising results that have been seen to date.

en

00:29:50

Learning to Take His Vitamins

RARECast ·

2025/04/10

Paul Biderman, chief operating officer and patient liaison for the ABL+ Foundation, discusses living with ABL, the challenges of obtaining adequate nutrition, and the impact becoming a patient advocate has had on him.

en

00:24:16

The First Treatment for a Rare Neurodegenerative...

RARECast ·

2025/04/03

Melissa Beiner, senior medical director at Biohaven, discusses spinocerebellar ataxia, the company’s therapy under priority review at the FDA to treat the condition, and why it may have the potential to benefit a number of other neurological conditions as well.

en

00:28:27

Engineering Skin Bacteria to Be Live Biotherapeutics

RARECast ·

2025/03/27

Travis Whitfill, co-founder and chief operating officer of Azitra, discusses Netherton syndrome, the company’s platform technology for engineering live biotherapeutic products, and why it takes more than simply inserting a gene into a desired bacteria to produce these treatments.

en

00:33:58

Seeing the Gene and Cell Therapy Translational Divide...

RARECast ·

2025/03/20

Nathan Yozwiak, head of research at the Mass General Brigham Gene and Cell Therapy Institute, about the need it is seeking to address, how the institute operates, and what it might do to enable the development of bespoke therapies for ultra-rare diseases.

en

00:27:47

My Mother, Myself, and ALS

RARECast ·

2025/03/13

Raziel Green, an ALS patient advocate, discusses her experience with the condition, her decision to enroll in a clinical trial, and her health more than eight years after her diagnosis.

en

00:24:11

From Immovable Object to Advocacy Force

RARECast ·

2025/03/06

Russ Walter, liaison and board member of the GBS-CIDP Foundation International, discusses his recovery from Guillian-Barre syndrome, the changing medical landscape for the condition, and why his experience led him to become a patient advocate.

en

00:23:56

A Rapid and Scalable Approach for Screening...

RARECast ·

2025/02/27

Scott Younger, director of disease gene engineering at Children Mercy Kansas City’s Genomic Medicine Center, discusses a process he developed to rapidly and inexpensively create organoids from a person’s induced pluripotent stem cells, how the process was used to match three children with Duchenne muscular dystrophy to an antisense oligonucleotide therapy, and the potential impact this may have on developing customized therapies for people with rare genetic diseases.

en

00:25:52

Addressing the Disease Mechanism of a Rare Kidney...

RARECast ·

2025/02/20

Marshall Fordyce, founder and CEO of Vera Therapeutics, discusses IgA nephropathy, the company’s experimental therapy atacicept, and its potential in IgAN other B cell-mediated diseases.

en

00:38:42

The Mother of a Son with SCD, Applies Direct...

RARECast ·

2025/02/13

Katrina Rice, chief delivery officer of biometrics services at the clinical trial data management and analytics company eClinical Solutions, discusses her experience as a mother of a child with a rare disease has informed her professional life, and how she has used that to educate her colleagues and clinical trial sponsors about how to better engage with rare disease clinical trial participants.

en

00:23:07

A First for Rett Syndrome with More in the Pipeline

RARECast ·

2025/02/06

Liz Thompson, executive vice president and head of research and development for Acadia, discusses Rett syndrome, how Daybue has changed the treatment landscape for people with the condition, and the company’s advancing pipeline to treat it.

en

00:30:13

A Heart Attack that Led to a Rare Disease Diagnosis

RARECast ·

2025/01/30

Patty Keating, executive director of the HypoPARAthyroidism Association, discusses her experience with hypoparathyroidism, why it can be challenging to diagnose, and the changing treatment landscape.

en

00:23:33

Targeting Rare Liver Diseases with Gene Editing...

RARECast ·

2025/01/23

Joe Truitt, CEO of iECURE, discusses the company’s experimental in vivo gene-editing therapy for OTC, how it works, and its the complete response seen in the first infant treated with the medicine.

en

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

565 results

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