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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.

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All Episodes

00:25:30

Part 4-Gene Therapy: A Researcher’s Perspective

RARECast ·

2020/11/13

P.J. Brooks, program director in the Office of Rare Disease Research at the National Center for Advancing Translational Sciences, discusses the PaVe-GT program, the potential for developing a toolkit of plug-and-play vectors, and how this can alter the cost of developing gene therapies for ultra-rare and individual patients.

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00:23:03

Part 3-Gene Therapy: An Industry Perspective

RARECast ·

2020/11/06

Janet Lambert, CEO of the Alliance for Regenerative Medicine, discusses the state of the gene therapy industry, the challenges developers face in advancing therapies to the market, and emerging pricing approaches to make them accessible to the patients who need them.

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00:27:28

Part 2-Gene Therapy: A Patient Advocate's Perspective

RARECast ·

2020/10/29

Kim Nye, founder and president of the TESS Research Foundation, discusses her own journey to becoming a rare disease advocate, how a gene therapy fit into her organization’s research agenda, and the considerations she is weighing before dosing her children with an experimental gene therapy.

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00:19:42

Part 1-Gene Therapy: A Genetic Counselor’s Perspective

RARECast ·

2020/10/23

In this first of our four-part series on gene therapies, genetic counselor Stephanie Gandomi, discusses the role genetic counselors play in working with rare disease patients, and considerations for patients who may want to pursue a gene therapy through a clinical trial.

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00:24:12

An App to Make Patients the Experts in their Own...

RARECast ·

2020/10/15

Nell Meosky Luo, founder and CEO of Folia Health, discusses spoke to Luo about how patients are using the platform, its potential to gather real-world data, and how the data it gathers could be used to advance the understanding of rare diseases.

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00:20:04

Pursuing a New Target for Autoimmune Diseases

RARECast ·

2020/10/08

Noreen Henig, chief medical officer for Kezar Life Sciences, discusses lupus nephritis, the company’s first-in-class candidate to target the condition, and why it may have application in a range of autoimmune conditions.

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00:19:10

Finding a Rare Friend

RARECast ·

2020/10/02

Ben Munoz, co-founder of Ben’s Friends, discusses his own experience with a rare condition, the role support from someone with the same condition has played for him, and his efforts to create a forum where patients with specific conditions can connect and provide support to each other.

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00:28:38

Industrializing Individual Therapies for People with...

RARECast ·

2020/09/25

Timothy Yu, a neurologist and attending physician in the Division of Genetics and Genomics at Boston Children’s Hospital Yu, discusses his work to advance individualized therapies for rare disease patients, the potential to industrialize the process, and the challenges that need to be addressed.

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00:33:57

Rare Disease Advocate Turned Filmmaker Turns Lens on...

RARECast ·

2020/09/17

Clinton Moore, Cystinosis Research Network and co-executive producer of the film Walk in My Shoes, on his son’s rare disease journey, his own emergence as an advocate, and how he came to recognize the power an individual has to make a difference.

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00:25:33

Inhibiting Signaling that Drives Growth of Rare...

RARECast ·

2020/09/10

Thom Rossi, CEO of Venthera, discusses the company’s lead experimental therapy, its first-in-man-trial, and how it is leveraging the resources of BridgeBio.

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00:17:52

Readying a Rare Disease Community for the...

RARECast ·

2020/09/03

Joslyn Crowe, executive director of the National Niemann-Pick Disease Foundation, discusses how her organization is preparing the patient community for the availability of the first therapies for the condition, the issues people will need to consider, and how families may need to think differently about their futures.

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00:25:07

Forging a Rare Disease Research Agenda

RARECast ·

2020/08/27

Cara O’Neill, chief scientific officer of Cure Sanfilippo Foundation, discusses Sanfilippo syndrome, her journey from physician to advocate, and her efforts to craft a research agenda.

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00:19:53

Delivering Injectable Drugs Orally

RARECast ·

2020/08/20

Raj Kannan, CEO of Chiasma, discusses the company’s recently approved therapy for acromegaly, the company’s drug delivery technology, and the value of having oral alternatives to injectable therapies.

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00:36:50

What Young Adults with Rare Conditions Need to Know...

RARECast ·

2020/08/14

Colleen Huysman, a clinical social worker with the Bridges Adult Transition Program at Boston Children’s Hospital, and Sneha Dave, founder and executive director of the Health Advocacy Summit, discuss the challenges young adults with rare and chronic condition face as they seek their own health insurance for the first time.

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00:22:24

Mirum Prepares to Seek Approval for Rare Liver...

RARECast ·

2020/08/07

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00:23:36

Payer Perceptions of Fairness in Pricing Rare Disease...

RARECast ·

2020/08/04

Charles Rivers Associates’ Andrew Parece and Matthew Majewski discuss how payers view the pricing of rare disease therapies, how context changes perceptions around pricing, and why they began their questioning of payers around the issue of fairness.

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00:21:15

Improving Narcolepsy Therapy with Drug Delivery...

RARECast ·

2020/07/23

Narcolepsy is a rare neurological sleep disorder with limited treatment options. Avadel is working on a controlled-release formulation of the standard of care, sodium oxybate, that allows for a once nightly dose of the drug. We spoke to Greg Divis, CEO of Avadel, about the company’s experimental therapy for narcolepsy, how it improves on the existing standard of care, and how it breaks with the company’s existing base of business. Editor's note: We recorded this podcast in late June. During the

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00:26:04

Fighting the Persistent Cough in a Rare and Deadly...

RARECast ·

2020/07/15

Idiopathic pulmonary fibrosis is a family of lung diseases characterized by scarring and thickening of lung tissue leading to an irreversible loss of lung function and reduced life expectancy. In normal times, the dry and persistent cough the condition can cause, can have a big impact on a person’s quality of life, but in the midst of a pandemic where coughing is a sign of infection, it can be particularly isolating. Respivant Sciences is developing an experimental therapy that treats IPF patients

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00:21:02

Targeting Huntington's Disease with Antisense

RARECast ·

2020/07/08

Ionis Pharmaceuticals has been a pioneer of antisense therapies, which target RNA to either disrupt production of a disease-causing protein or upregulate needed proteins in people who are deficient. The company is in late stage development of a promising therapy to treat Huntington’s disease, a rare and fatal neurodegenerative condition. But the company is also working on advancing a new generation of antisense therapies to improve the safety and efficacy of these treatments. We spoke to Eric

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00:20:12

Assembling a Pipeline for Rare GI Disorders

RARECast ·

2020/07/01

Short Bowel Syndrome is a life-threatening rare disease caused by a significant shortening of the gastrointestinal tract. Because of difficulties people with short bowel syndrome have properly absorbing nutrients they rely on receiving them through intravenous infusions. This has an impact on the quality of life of people with the condition and comes with a high risk of systemic infections. 9 Meters Biopharma is focused on rare gastrointestinal conditions. It is advancing an experimental therapy for

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568 results

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