Key Takeaways:
• Over 60 gene therapies and ASO drugs are now FDA-approved, with 1,200+ in active clinical trials. 88% of 2024 approvals were for rare/orphan diseases.
• Patient-founded organizations are driving some of the most important breakthroughs.
• 74% of FDA gene therapy rejections stem from manufacturing quality issues, not scientific failure — making manufacturing expertise critical for any therapy development program.
• For nano-rare diseases where pharma won't invest, individualized ASOs offer the most mature and established pathway to treatment.
