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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

00:28:28

Forging a Faster Path for Gene Therapies

RARECast ·

2024/04/11

Courtney Silverthorn, vice president of strategic alliances and innovation for the Foundation for the National Institutes of Health; and Sharon King, manager of advocacy and community engagement for Aldevron and founder and president of Taylor’s Tale; discuss the Bespoke Gene Therapy Consortium, its new playbook, and how it will help gene therapy developers get their medicines to patients faster.

en

00:25:25

Determining the Value of Rare Disease Therapies

RARECast ·

2024/04/04

Rick Chapman, chief scientific officer of the Innovation and Value Initiative, discusses the challenges of assessing the value of rare disease therapies, the role qualitative data should play in value assessments, and the recommendations from the report.

en

00:28:00

Buying and Building a Gene Therapy Presence

RARECast ·

2024/03/28

Ha Tran, medical head of cell and gene therapy for Astellas Pharma, discusses the company’s vision for cell and gene therapies, its efforts to restart trials for its X-linked myotubular myopathy gene therapy, and how it is looking to other programs beyond that.

en

00:44:08

A Clinical Trial Failure Derails a Promising Technology

RARECast ·

2024/03/21

Neal Sondheimer, outgoing head of clinical for Synlogic and adjunct associate professor of pediatrics and molecular genetics for The Hospital for Sick Children in Toronto, discusses PKU, the treatment options for people with the condition, and the consequences of the surprising results from the Synlogic study.

en

00:32:19

Helping People with Undiagnosed Rare Diseases Find...

RARECast ·

2024/03/14

Michele Herndon and F. Sessions Cole of the Undiagnosed Disease Network Foundation discuss the diagnostic odyssey, the work of the Undiagnosed Disease Network and the Undiagnosed Disease Network Foundation, and how the UDNF’s new Patient Navigator program works.

en

00:50:47

Speeding and Scaling the Development of Genome...

RARECast ·

2024/03/07

Fyodor Urnov, director of technology and translation for the Innovative Genomics Institute and director of the new Danaher-IGI Beacon for CRISPR Cures center, discusses the evolution of gene editing technology, the challenges of a platform approach to gene editing, and how the organization plans to share what it learns.

en

00:42:47

Getting a Next-Generation Genome Editing Therapy for...

RARECast ·

2024/02/29

Matthew Porteus, co-founder and CEO of Kamau Therapeutics, discusses the company’s genome editing technology, what’s now understood about the serious adverse event that occurred in a Graphite Bio clinical trial of the company’s lead experimental therapy, and the development path forward for the therapy.

en

00:16:52

Trying to Break a Leg in a Wheelchair

RARECast ·

2024/02/22

Actress Shannon DeVido and creative director Adam Pryor, about the spinal muscular atrophy musical comedy “Most Likely Not to…,” living with SMA, and DeVido’s pursuit of a career as a performing artist despite having the condition.

en

00:32:58

A Drug Developer that Makes Pediatric Cancers a Priority

RARECast ·

2024/02/15

Samuel Blackman, head of research and development for Day One Biopharmaceuticals, discusses childhood cancers, the need for precisions therapies, and Day One’s business model that makes targeting childhood cancers a priority.

en

00:35:50

Harnessing the Body’s Natural RNA Machinery to Treat...

RARECast ·

2024/02/08

Ram Aiyar, president and CEO of Korro Bio, discusses the company’s RNA editing platform technology, how it works, and its initial focus on applying its approach to treat a rare liver disease.

en

00:24:39

Targeting the Leading Cause of Death in Friedreich’s...

RARECast ·

2024/02/01

R. Nolan Townsend, CEO of Lexeo Therapeutics, discusses Friedreich’s ataxia, his company’s gene therapy in development, and its pursuit of gene therapies for both rare and common diseases.

en

00:24:33

How Inhaled mRNA May Help Rare Disease Patients...

RARECast ·

2024/01/25

Thomas Langenickel, chief medical officer of Ethris, discusses how the company’s technology overcomes existing challenges for the therapeutic use of mRNA, its ability to deliver treatments directly to the lung through inhalation, and its pipeline of therapies in development.

en

00:34:34

Changing What’s Possible with Cell and Gene Therapies

RARECast ·

2024/01/18

Rahul Kakkar, president and CEO of Tome Biosciences, discusses the company’s genome editing technology, how it works, and its potential to change what is possible with gene and cell therapies.

en

00:43:02

Why Flying with a Wheelchair Is a Civil Rights Issue

RARECast ·

2024/01/11

Global Genes’ Director of Community Engagement Daniel DeFabio and Founder and President of The Jansen’s Foundation Neena Nizar, discuss the challenges disabled airline passengers face, what the law says, and why this is a civil rights issue.

en

00:28:42

Mapping Spheres of Influence

RARECast ·

2024/01/04

Sandra Shpilberg, co-founder and CEO of Adnexi, discusses the role KOLs and DOLs play, how Adnexi identifies and measures their influence, and what companies need to consider when engaging them.

en

00:32:58

Bringing Precision to the Treatment of Rare Cancers

RARECast ·

2023/12/28

David Hysong, founder and CEO of Shepherd Therapeutics, discusses his company’s use of AI to analyze individual patient’s tumor RNA, its efforts to match rare cancer patients to their best therapeutic options, and how it is using data captured from the transcriptome to develop new therapies for people with rare cancers.

en

00:27:03

The Challenges of Developing a Therapy for Pregnant...

RARECast ·

2023/12/21

Katie Abouzahr, vice president of the autoantibody portfolio and maternal fetal disease area leader for Johnson and Johnson, discusses hemolytic disease of the fetus and newborn, the challenges of developing a therapy for a rare condition in pregnant women, and why its experimental therapy nipocalimab has the potential to be a pipeline in a product.https://globalgenes.org/raredaily/the-challenges-of-developing-a-therapy-for-pregnant-women-with-a-rare-condition/

en

00:24:42

How One Drug Developer Is Addressing Health Inequities

RARECast ·

2023/12/14

Robert Blum, CEO of Cytokinetics, discusses hypertrophic cardiomyopathy, how the company has worked to build relationships in the black community to expand participation in clinical trials, and how it is thinking ahead to issues of access and affordability as it advances the drug towards the market.

en

00:19:15

Accelerating the Development of Genetic Medicines...

RARECast ·

2023/12/07

David Del Bourgo, CEO of WhiteLab Genomics, discusses its AI-platform technology, the data it uses, and its role in a consortium to develop highly specific vectors for genetic medicines.

en

00:35:57

Leveraging AI to Address the Neuropsychiatric...

RARECast ·

2023/11/30

Neil Inala, CEO of Nobias Therapeutics, discusses how AI is transforming drug discovery, the data Nobias is able to use to fuel its drug discovery, and the company’s experimental therapy for the neuropsychiatric conditions associated with 22q11.2 deletion syndrome.

en

566 results

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