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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

00:18:11

Improving Rare Disease Care through Partnerships

RARECast ·

2025/10/16

Partnerships between biopharmaceutical companies and patient organizations can play a critical role in improving access and outcomes for people with rare diseases. However, to do so, companies must listen to patient communities and address the unique challenges they face. We spoke to Laura Russo, U.S. patient engagement lead for Pfizer, discusses how the company’s patient-facing teams work to bridge health systems and patient communities, how they help people with rare diseases navigate the

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00:27:21

A Call on the FDA for Timely Reviews of Life-Saving...

RARECast ·

2025/10/09

Hunter syndrome is caused by the body’s inability to produce a critical enzyme needed to break down cellular waste. The condition can cause damage to organs throughout the body as well as to the brain. A new generation of therapies in development, including a gene therapy currently under review by the U.S. Food and Drug Administration, that has the potential to address the neurological symptoms of the disease. Still, patient advocates have been frustrated by regulatory delays and are seeking to push

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00:31:50

The Need to Look Beyond Seizures When Treating DEEs

RARECast ·

2025/10/02

Developmental and epileptic encephalopathies are a group of rare disorders that are characterized by frequent seizures that often don’t respond to existing medications. These are complex conditions that involve progressive cognitive and behavioral manifestations that can pose significant burdens on patients and their families. In both clinical practice and within the biopharmaceutical industry, there has been a tendency to focus on seizure control, while often overlooking the non-seizure burdens of

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00:23:06

Taking On Big Competitors with an Oral Therapy to...

RARECast ·

2025/09/25

Achondroplasia is the most common form of dwarfism. Beyond short stature, people living with achondroplasia can experience serious health complications, including compression of the brainstem and upper spinal cord due to impaired development of the skull. Tyra Biosciences is developing a next-generation medicine to precisely target FGFR3, an overactive growth factor that causes achondroplasia. We spoke to Todd Harris, CEO of Tyra Biosciences, about the company’s experimental once-daily, oral

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00:28:00

Going Viral to Fight Brain Cancer

RARECast ·

2025/09/18

Recurrent high-grade glioblastoma is a rare and aggressive brain tumor, which today is generally treated with surgery and chemotherapy. Outcomes are poor, with survival ranging from three to nine months and five-year survival rates less than 10 percent. Candel Therapeutics is developing viral immunotherapies that both kill tumor cells directly and enlist the patient’s own immune system in the fight against cancer. It’s experimental therapy CAN-3110 uses a modified herpes simplex virus that carries a

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00:29:22

Changing the Playbook on Childhood Cancers

RARECast ·

2025/09/11

While there has been enormous innovation in the treatment of cancer over the past two decades, much of this has been focused on adult cancers. Despite the advent of targeted therapies and immunotherapies, the treatment of childhood cancers relies largely on chemotherapy and radiation, both of which can create lifelong side effects in developing bodies. And cancer remains the leading cause of death by disease in children in the United States and the United Kingdom. C-Further, an international

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00:32:56

Sometimes the Hoofbeats Do Belong to Zebras

RARECast ·

2025/09/04

Avion was a healthy and athletic 15-year-old who became critically ill when he was admitted into a pediatric intensive care unit. For Robin Williams, assistant professor of pediatric hematology/oncology at the University of Minnesota Masonic Children's Hospital, Avion offered a medical puzzle she couldn’t crack on her own. His immune system was on overdrive and it was attacking healthy cells and organs within his body. Though testing ruled out blood cancers, it was only when Williams consulted a

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00:29:51

Breathing Easier with a Breakthrough Immunotherapy

RARECast ·

2025/08/28

Recurrent respiratory papillomatosis is a potentially life-threatening disease of the upper and lower respiratory tract caused by chronic infection with human papillomavirus type 6 or type 11. In the absence of approved therapies, people with the condition often undergo repeated surgeries to clear their airways. The U.S. Food and Drug Administration in August approved Precigen’s Papzimeos, an immunotherapy that targets the underlying cause of the RRP, as the first approved therapy to treat the

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00:28:19

Using AI Open Innovation to Tackle ALS

RARECast ·

2025/08/21

Tris Dyson founded Challenge Works to incentivize innovators to solve societal problems. Dyson, who was diagnosed with amyotrophic lateral sclerosis, is now using the platform to find new treatments for the progressive neurodegenerative disease. The $10 million Challenge Works' Longitude Prize on ALS harnesses AI, open collaboration, and big data to find new treatments for the condition. We spoke to Dyson, managing director of Challenge Works, about his diagnosis of ALS, the case for using a prize

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00:40:24

Recovering What’s Lost in Translation

RARECast ·

2025/08/14

When Daniel Fischer’s daughter Natasha was diagnosed with the rare genetic epilepsy, Dravet syndrome, his search for treatments eventually led him to tRNA therapies, an emerging area of genetic medicines that work to correct so-called nonsense mutations. Nonsense mutations prematurely cause the translation of a gene to stop before a protein is fully formed. What’s particularly compelling about the approach is that a single therapy has the potential to correct any nonsense mutation, regardless of the

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00:25:43

Targeting Hunger Rather than Appetite in Prader-Willi...

RARECast ·

2025/08/07

Prader-Willi syndrome is a rare and complex genetic condition, the hallmark of which is hyperphagia, an intense and insatiable hunger. Hunger and appetite, though, are different things, particularly from a biological perspective. Aardvark Therapeutics is developing an experimental therapy to treat Prader-Willi syndrome by targeting hunger as opposed to appetite. We spoke to Tien Lee, CEO of Aardvark Therapeutics, about Prader-Willi syndrome, the company’s experimental therapy to treat the condition,

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00:42:29

A Rare-to-Common Business Model

RARECast ·

2025/07/31

Rare disease advocates have long made the case that studying rare diseases can provide insights into more common ones. Actio Biosciences has turned that into a business model. The company is leveraging genetics and precision medicine to develop drugs for rare diseases in the hopes of expanding the indications for them to include more common disease that share underlying biology. We spoke to David Goldstein, founder and CEO of Actio Biosciences, about the company’s Rare Disease Target Atlas, how it

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00:34:47

Leading a Rare Youth Movement

RARECast ·

2025/07/24

When people with a rare disease accomplish a lot, someone might say they did so “despite their condition.” In the case of Khartik Uppalapati, it might be more appropriate to say “because of his conditions.” Uppalapati, a 16-year-old Virginia high school student, is a co-founder of the RareGen Youth Network, an organization designed to give voice to young people affected by rare diseases. He’s also an entrepreneur, scientific researcher, and international rights activist, all stemming from his

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00:37:10

Developing Gene Therapies that Work Across Mutations

RARECast ·

2025/07/17

One of the challenges for developing gene therapies for inherited eye diseases is that a large number of individual mutations to a gene can drive the same disease. That makes conventional gene replacement therapy a difficult approach to treat a large number of patients. Ocugen is developing gene therapies that target master regulators of genetic networks and can work across different mutations. We spoke to Arun Upadhyay, chief scientific officer and head of research and development at Ocugen, about

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00:40:04

Eyeing A Challenge to a Blockbuster

RARECast ·

2025/07/10

Steve Mahoney, CEO of Viridian Therapeutics, discusses the complexities of thyroid eye disease, the challenges of living with the condition, and why veligrotug has the potential to provide a new alternative for people living with the condition.

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00:28:14

Like Mother, Like Child

RARECast ·

2025/07/03

Ben Davies and his mother Traci Davies discuss their experiences with primary immunodeficiency, what it's like to be both a caregiver and patient, and why they have both become patient advocates.

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00:24:08

Transforming from Grandmother to Mother to Advocate

RARECast ·

2025/06/26

Melanie Kandzierski discusses how she has learned to care for a child with Dravet syndrome, care for herself, and to advocate not only for the child but others with complex medical conditions.

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00:16:57

Overcoming a Delivery Challenge of Retinal Gene...

RARECast ·

2025/06/19

Patrick Ritschel, CEO of Atsena Therapeutics, discusses the delivery challenges of gene therapies for inherited retinal diseases, how the company’s unique vector technology addresses this, and how it allows for safer and more effective delivery of gene therapies to the retina.

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00:41:28

A Gene Editing First Augurs an Era of Bespoke Therapies

RARECast ·

2025/06/12

P.J. Brooks, deputy director of the Office of Rare Disease Research at the National Institutes of Health’s National Center for Advancing Translational Sciences, discusses the recent treatment of an infant with a bespoke gene editing therapy, how the researchers were able to move from diagnosis to treatment with great speed, and what it would take to scale such an approach.

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00:49:19

How One Patient Organization Drives Drug Development

RARECast ·

2025/06/05

Annette Bakker, CEO of the Children's Tumor Foundation, discusses the complexities of a group of rare genetic conditions that cause tumors to grow on nerves throughout the body, the foundation's role in advancing research and drug development, and what other patient organizations can learn from its strategic approach.

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565 results

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