x

75% of people globally want unbiased news (Pew Research) but are concerned they're not getting that.

Get balanced coverage with Symby. See how stories are covered across diverse outlets and spot when one side covers a story the other skips.

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

00:35:18

Addressing the Delivery Challenges of Genetic Medicines

RARECast ·

2021/08/19

James Dahlman, associate professor in the Department of Biomedical Engineering at Georgia Tech and Emory University, discusses the delivery challenges of genetic medicines, how nanoparticles compare to viral vectors, and what it takes to develop new vectors that can deliver genetic medicines to where they need to go.

en

00:21:22

Targeting Tissues Throughout the Body with RNA Therapies

RARECast ·

2021/08/12

Arthur Suckow, co-founder and CEO of DTx Pharma, discusses the delivery challenge of RNA therapies, how DTx’s platform technology addresses these, and how a $100 million financing from earlier this year will be used to fuel its growth.

en

00:20:54

Delivering Rare Disease Therapies to Patients in Need

RARECast ·

2021/08/05

Bill Sibold, executive vice president of Sanofi Genzyme and president of Sanofi North America, discusses the company’s Humanitarian Aid Program, how it works; and how it overcomes the regulatory, infrastructure, and medical challenges of delivering these treatments to patients around the globe.

en

00:23:44

An Advocate's Journey through Diagnosis, Loss, and Hope

RARECast ·

2021/07/29

Ashley Walker, an XLMTM patient advocate, discusses her own rare disease journey ahead of an externally-led XLMTM Patient-Focused Drug Development meeting, what life with the progressive neuromuscular condition is like, and her hopes that new treatments are not far away.

en

00:20:25

Using Model Systems to Find Drugs to Repurpose for...

RARECast ·

2021/07/22

James Doyle, CEO of Modelis, discusses the use of model systems, how his company creates genetic avatars of patients; and how it works with rare disease drug developers, patient organizations, and patients to identify candidates for repurposing.

en

00:23:54

How a Small and Young Foundation Catalyzed Research...

RARECast ·

2021/07/16

Susan Ruediger, founder and CEO of CMT Research Foundation, discusses her organization’s efforts to catalyze drug development for Charcot-Marie Tooth disease, what it’s done to address obstacles, and some of the partnerships it has established to advance the development of treatments and potentially a cure.

en

00:30:35

Overcoming the Limitations of Conventional Cell...

RARECast ·

2021/07/08

Murat Kalayoglu, president and CEO of Cartesian Therapeutics, discusses the company’s RNA-engineered cell therapies, how they work, and how the company is pushing the use of cell therapies beyond cancer.

en

00:20:20

Experimental Therapy for Rare Endocrine Disorder...

RARECast ·

2021/07/02

Richard King, CEO of Spruce Biosciences, discusses the rare endocrine disorder CAH, how his company’s experimental therapy tildacerfont works, and what the company is doing to build a pipeline of other rare endocrine therapies behind it.

en

00:18:49

Forging Gene Therapy Capacity and a Pipeline at the...

RARECast ·

2021/06/24

Tim Miller, co-founder and CEO of Forge Biologics, discusses the company’s hybrid business model, how it hopes to differentiate itself through its shared experience with its customers, and its emerging pipeline of gene therapies to treat rare diseases.

en

00:26:34

Using A Natural DNA Repair Process to Improve Genetic...

RARECast ·

2021/06/18

Daniel Gruskin, chief medical officer of LogicBio, discusses the company’s platform technology, the advantages it provides, and the company’s lead experimental therapy for the rare metabolic condition methylmalonic acidemia.

en

00:22:40

Empowering Rare Disease Patients with their Own...

RARECast ·

2021/06/10

Nasha Fitter, vice president of rare disease for Ciitizen, about her own experience as a mother of a child with a rare neurologic condition, her work as a rare disease advocate, and a collaboration between a group of rare neurologic disease advocacy organizations to build a natural history study on the Ciitizen platform.

en

00:40:40

Targeting CNS Disease with Gene Therapies

RARECast ·

2021/06/03

Bruce Goldsmith, CEO of Passage Bio, discusses the company’s relationship with Penn’s Gene Therapy Program, its focus on CNS conditions, and the company’s lead program in the rare lysosomal storage disorder GM1 gangliosidosis.

en

00:21:15

A New Therapy Offers A Different Approach to...

RARECast ·

2021/05/28

Cedric Francois, co-founder and CEO of Apellis, discusses the recent FDA approval of the PNH therapy Empaveli, what advantages it may provide over existing therapies, and other indications the company will pursue for this medicine.

en

00:22:28

Bringing Aberrant Proteins Back into the Fold

RARECast ·

2021/05/20

Eric Richman, CEO of Gain Therapeutics, discusses its platform technology, how it works, and why it offers a compelling alternative to other approaches to treating lysosomal storage disorders.

en

00:33:34

A Cat who Curates Rare Mice

RARECast ·

2021/05/13

Cat Lutz, senior director of mouse repository and in vivo pharmacology genetic resource science at The Jackson Laboratory, discusses the role mouse models play in rare disease research, how new gene editing technologies are changing the development of mouse models, and why new technologies are unlikely to displace the critical role they play anytime soon.

en

00:22:58

Putting the Patient at the Center of Rare Disease...

RARECast ·

2021/05/06

Scott Schliebner, senior vice president and head of the Center for Rare Diseases at PRA Health Sciences, discusses designing patient-centric clinical trials, why it matters, and what sponsors can do to stay focused on patients. @globalgenes #RARECast

en

00:32:50

Creating a Toolkit to Accelerate the Development of...

RARECast ·

2021/04/29

Erik Sontheimer, co-chair of the National Institutes of Health’s Somatic Cell Genome Editing Consortium's steering committee, about the program, the challenges it is seeking to address, and it potential to accelerate the development of a new generation of genetic medicines.

en

00:22:03

PTC Looks to Advanced Therapies

RARECast ·

2021/04/22

Matthew Klein, chief development officer for PTC Therapeutics, disc usses the company’s work in SMA, its move into regenerative medicine, and its efforts to win approval in Europe and the United States for its first gene therapy. @globalgenes #RARECast

en

00:49:28

Going to Extreme Lengths

RARECast ·

2021/04/15

Chandler Crews, founder and president of the nonprofit patient organization The Chandler Project, discusses her experiences living with the achondroplasia, her decision to undergo surgery to lengthen her limbs, and how she views new therapies working their way through clinical development that seek to promote growth in people with achondroplasia.

en

00:27:40

Everything’s up to Date in Kansas City, at Least When...

RARECast ·

2021/04/08

Tom Curran, executive director of the Children’s Mercy Research Institute, about the research program, its ambitious efforts, and it focus on translational research.

en

568 results

Similar Podcasts

TaxTalent.com The Golden Rule Podcast

Adam Golden

TaxTalent.com Podcast

Tony Santiago

Handelsblatt Crime - spannende Streitfälle der deutschen Wirtschaft

Ina Karabasz, Solveig Gode, Sönke Iwersen

Deffner und Zschäpitz – Der Wirtschafts-Talk von WELT

WELT

Black Box - La scatola nera della finanza

Guido Brera e Gian Luca Comandini -...