RARECast

548 Episodes

1. Choosing the Right Viral Vector for a Gene Therapy

   Published: 4/1/2021
2. A Software Platform to Give Patients the Tools to Build Treatments for Rare Genetic Diseases

   Published: 3/25/2021
3. Side Effects: The Toll a Rare Disease Can Take on a Family in Pursuit of a Cure

   Published: 3/19/2021
4. Realizing the Potential of CRISPR Gene Editing

   Published: 3/12/2021
5. Understanding the Economic Toll of Rare Disease in the United States

   Published: 3/4/2021
6. The Promise of Gene-Based Therapies for Neurodegenerative Conditions

   Published: 2/25/2021
7. Moving Beyond Viral Vectors for Gene Therapies

   Published: 2/19/2021
8. Acquisitions Help Jazz Build Toward Commercial Crescendo

   Published: 2/12/2021
9. Powerful Gene Editing Approach Offers the Promise of Correcting a Range of Rare Diseases

   Published: 2/5/2021
10. A Child's-Eye-View of Clinical Trials

    Published: 1/29/2021
11. Using Nanoliposomes to Make Cancer Therapies Safer and More Effective

    Published: 1/21/2021
12. Accelerating Treatments for Rare Disease through Data Sharing

    Published: 1/14/2021
13. Treating the Root Cause of Sickle Cell Disease

    Published: 1/8/2021
14. Targeting a Common Pathway in Genetic Forms of Obesity

    Published: 12/30/2020
15. After Pruning Orchard, Gaspar Focuses on High Value Opportunities

    Published: 12/23/2020
16. Writing a New Chapter of Genetic Medicine

    Published: 12/17/2020
17. How an Ultra-Rare Disease Patient Organization Drove Research to a Treatment

    Published: 12/10/2020
18. With a New Identity, Travere Thearpeutics Seeks to Find Its True Path

    Published: 12/4/2020
19. Making Gene Therapies Accessible to Patient with Ultra-Rare Conditions

    Published: 11/26/2020
20. Using RNA Therapies to Target Inherited Retinal Diseases

    Published: 11/20/2020