Former FDA Commissioner Dr. Scott Gottlieb on finding cures for rare diseases

Summary

Former FDA Commissioner Scott Gottlieb discusses rare disease research and the regulatory path for personalized gene therapies. He highlights advances in AAV vectors, lipid nanoparticles, synthetic capsids, and base editing, and notes that FDA approval rates for cell and gene therapies are rising. Gottlieb argues regulation, not technology, is now the main roadblock and calls for congressional action to scale N-of-1 trials. He names Beam and Verve as companies advancing base editing.

• CNBC Cures initiative focuses on rare diseases and treatment gaps.
• Gottlieb recounts early N-of-1 pathway work for Batten disease.
• Gene therapy delivery has advanced through AAV, lipid nanoparticles, synthetic capsids, and base editing.
• FDA has not yet created a reproducible framework for N-of-1 trials.
• Congress could give FDA targeted authorities and resources.
• Cell and gene therapy approvals have risen toward a 10-per-year run rate.
• Beam and Verve are cited for progress in base editing.