RARECast

512 Episodes

1. Creating a Toolkit to Accelerate the Development of Gene Editing Therapies

   Published: 4/29/2021
2. PTC Looks to Advanced Therapies

   Published: 4/22/2021
3. Going to Extreme Lengths

   Published: 4/15/2021
4. Everything’s up to Date in Kansas City, at Least When It Comes to Genomics

   Published: 4/8/2021
5. Choosing the Right Viral Vector for a Gene Therapy

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

    Published: 12/17/2020