RARECast

The ability to target the underlying cause of a disease and make a lasting correction makes gene therapy an attractive approach to treating neurodegenerative conditions. The advent of Zolgensma, a gene therapy for the treatment of the rare neurodegenerative condition spinal muscular atrophy, serves as a model for this approach. A recent review article in Nature Neuroscience looks at the advances in development of gene therapies for neurodegenerative disease and considers the challenges and promises. We spoke to article co-author Subhojit Roy, professor in the departments of Pathology and Neuroscience at the University of California, San Diego, about the pace of activity in this area, why he believes it is so promising, and its potential extend beyond monogenic diseases. This podcast is part of our ongoing Platforms of Hope series that explore advances in gene Thanks to Pfizer, Inc., Bluebird, and Novartis Gene Therapies for their support of this podcast article, part of our Platforms of Hope: Advances in Gene Therapy and Gene Editing series.therapy and gene editing. Thanks to Pfizer, Inc., Bluebird, and Novartis Gene Therapies for their support of this podcast, part of our Platforms of Hope: Advances in Gene Therapy and Gene Editing series.

Eric Ostertag, CEO of Poseida, discusses the company’s platform technology, its gene therapy pipeline, and its efforts to move beyond viral vectors.

Robert Iannone, executive vice president of research and development and chief medical officer of Jazz Pharmaceuticals, discusses the company’s neuroscience pipeline, its growing family of commercial products, and how its preparing to maintain its leadership in the treatment of the rare sleep disorder narcolepsy as new competitors move toward market.

David Liu, director of the Merkin Institute for Transformative Technologies at the Broad Institute, discussing a recent study of base editing to treat the rare disease progeria, how it works, and why it may offer the potential to treat a wide range of rare diseases.

Arya Singh discusses the surgeries and clinical trials she endured as a child, her new children’s book Courageous Calla about participating in a clinical child, and her interest in humanizing medicine.

Marc Hedrick, CEO of Plus Therapeutics, discusses the company’s nanoliposome technology, how it works, and why the company is focusing on rare cancers.

Nicole Boice, co-founder and executive director of RARE-X, discusses the problem RARE-X is seeking to address, the technology and expertise the organization has been able to bring together, and why data sharing is essential to accelerating the diagnosis or rare diseases and development of new treatments to treat them.

Ted Love, president and CEO of Global Blood Therapeutics, discusses the changing landscape of sickle cell disease, Oxbryta and the company’s pipeline behind it, and how the company is approaching the global need for the drug given the higher prevalence of the disease in parts of the world.

David Meeker, CEO of Rhythm, discusses genetic obesities, the company’s drug Imcivree, and his plans to expand the use of the drug beyond the initial approved indications.

Bobby Gaspar, founder and CEO of Orchard Therapeutics, discusses the company’s sharpened focus, its gene therapy in development for metachromatic leukodystrophy, and where he sees the greatest opportunities for Orchard going forward