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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

00:25:33

Putting Rare Disease Organization in the Business of...

RARECast ·

2024/08/29

Chris Hopkins, CEO of DevineBio, discusses how the company works with rare disease patient organizations to discover and develop therapies, how far it will advance programs, and its exit strategy.

en

00:31:02

Understanding the Many Questions Gene Therapies Raise

RARECast ·

2024/08/22

Avery McIntosh, director at Pfizer, and Alex Sverdlov, senior director of statistical analysis at Novartis, discuss their new book "Development of Gene Therapies," how a pair of biostatisticians view the challenges they raise, and why these therapies don’t easily fit into existing models.

en

00:46:30

A Test to Identify People Likely to Develop ALS

RARECast ·

2024/08/15

Paul Wicks, vice president of neuroscience for Sano Genetics, discusses its ALS diagnostic test, how it works, and why some healthy people might want to take advantage of it. @global genes #RARECast

en

00:27:57

How an Ultra-Rare Disease Patient Foundation Advanced...

RARECast ·

2024/08/08

United MSD Foundation Executive Director Sarah Cortell Vandryspen and UT Southwestern Gene Therapy Core Director Steven Gray, discuss United MSD Foundation’s research strategy, what enabled it to advance a gene therapy as fast as it did, and what other patient organizations can learn from its success.

en

00:21:35

Making the $10 Genome a Reality

RARECast ·

2024/08/01

Johan Strömqvist, CEO and founder of Single Technologies and Bob Kain, advisor to the company, discuss Single’s 3-D Sequencing, how it works, and how this can open up expansive use of the technology.

en

00:28:01

A No-Nonsense Effort to Develop a Therapy that Works...

RARECast ·

2024/07/25

Michelle Werner, CEO of Alltrna, discusses nonsense mutations, how the company’s transfer RNA therapies work, and why the approach has the potential to address so many diseases at once.

en

00:26:57

Mapping a Perilous Journey with Humor

RARECast ·

2024/07/18

Effie Parks discusses her experience with the diagnostic odyssey for her son Ford, her version of the map to a genetic diagnosis, and what organizations usually get wrong. @globalgenes #RARECast

en

00:21:54

Restoring Vision in Inherited Retinal Disease

RARECast ·

2024/07/11

Brian Strem, CEO of Kiora Pharmaceuticals, discusses inherited retinal diseases, how Kiora’s experimental therapy works to treat these conditions, and a recent collaboration that will help drive clinical development of its lead experimental therapy.

en

00:27:51

Bringing Genome Sequencing to Rural Populations

RARECast ·

2024/07/04

Ana Cohen, assistant director of molecular genetics at Children’s Mercy Research Institute, about its direct-to-provider model, how providing local support to patients at their regular clinics allowed them to bypass bottlenecks, and how the approach can shorten the time to a diagnosis for people with rare diseases in rural communities.

en

00:23:20

Addressing a Blind Spot in Treatments for the Cornea

RARECast ·

2024/06/27

Clarke Atwell, CEO of Claris Bio, discusses the cornea, the role hepatocyte growth factor plays in its healing, and the rare and chronic condition neurotrophic keratitis that it is focusing on as its lead indication.

en

00:38:53

Taking a Different Approach to Rare Epilepsies

RARECast ·

2024/06/20

Meg Alexander, chief strategy officer of Ovid Therapeutics, discusses rare epilepsies, how the company’s experimental therapy soticlestat works, and the potential to apply the approach to other CNS conditions.

en

00:20:00

A Rare Disease Drug Developer Tries to Earn Its Stripes

RARECast ·

2024/06/13

Neil McFarlane, president and CEO of Zevra Therapeutics, discusses the rare lysosomal storage disorder Niemann-Pick type C disease, the FDA’s upcoming decision on whether to approve the drug, and its broader efforts to build itself into a rare disease therapeutics company. @globalgenes #RARECast

en

00:15:01

Using Plasma to Treat Rare Diseases

RARECast ·

2024/06/06

Giles Platford, president of the Plasma-Derived Therapies Business Unit at Takeda, discusses its work in plasma-derived therapies, its recently approved therapy for the rare neuromuscular condition CIDP, and what issues need to be addressed to ensure an adequate supply of human plasma for therapeutic applications.

en

00:25:47

Differentiating Gene Therapies through Regulatory...

RARECast ·

2024/05/30

Salvador Rico, chief medical officer of Encoded Therapeutics, discusses the company’s lead program in Dravet syndrome, its efforts to develop gene therapies with optimized regulatory elements to target specific organs, and why he believes its approach is a point of differentiation for the company.

en

00:29:05

How One Patient Organization Leverages Research...

RARECast ·

2024/05/23

Steve Roberds, chief scientific officer of the TSC Alliance, discusses the organization's success with crafting a research agenda, how it’s been able to invest in ways that catalyze research, and what it’s done to facilitate drug development by industry.

en

00:21:11

Charging into the Storm

RARECast ·

2024/05/16

Sunitha Malepati, founder of the Buffalo Initiative, discusses how she grew frustrated with the drug development landscape, how the Buffalo Initiative plans to fund patient advocacy organizations drug development efforts, and what the initiative is doing to reduce the time and cost of developing a therapy.

en

00:30:20

A Gene Therapy Developer that Embraces Different...

RARECast ·

2024/05/09

Frederic Revah, CEO of the non-profit Genethon, discusses the limits of licensing out its discoveries to biopharma, the different development strategies it pursues, and how it determines the best path for a particular development program.

en

00:31:08

Using Directed Evolution to Develop New Vectors for...

RARECast ·

2024/05/02

Alan Cohen, senior vice president of clinical development and therapeutic area head of pulmonology for 4DMT, discusses the limitations of existing vectors for genetic medicines, 4DMT’s directed evolution platform technology, and its programs in cystic fibrosis and Fabry disease.

en

00:35:18

Addressing a Shortage of Genetic Counselors with AI

RARECast ·

2024/04/25

Doron Behar, co-founder and CEO of Igentify, discusses the company’s AI-powered Digital Genetic Assistant, how it works, and why it will allow genetic counselors to handle a much larger volume of patients.

en

00:23:55

A Small Molecule Therapy to Regenerate Muscle in...

RARECast ·

2024/04/18

Frank Gleeson, co-founder and CEO of Satellos, discusses its regenerative therapy for Duchenne, how it works, and why it may provide benefits to patients with other conditions that result in muscle degeneration.

en

566 results

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