22/09/2026
The Rise of In Vivo Gene Editing: New Opportunities in Therapeutic Innovation
In vivo gene editing is reshaping the operating model of genetic medicine by moving the editing process from the manufacturing facility directly into the patient.
Key Highlights:
• In vivo editing can shift genetic medicine from bespoke procedures toward scalable pharmaceutical products
• Delivery remains one of the field’s biggest scientific and development challenges
• Liver-directed approaches are advancing while extrahepatic delivery remains more complex
• Irreversible genomic changes require rigorous safety assessment and long-term follow-up
• Batch manufacturing could improve scalability compared with patient-specific ex vivo approaches
• One-time therapies create new challenges for pricing, reimbursement, and long-term value assessment
Why It Matters: In vivo gene editing could expand access to genetic medicines by transforming a highly personalized treatment process into a scalable therapeutic model. But its long-term potential depends on solving delivery, safety, regulatory, manufacturing, and reimbursement challenges with the same rigor as the underlying editing science.
🔗 https://www.pharmafocuseurope.com/articles/the-rise-of-in-vivo-gene-editing
In vivo gene editing is advancing therapeutic innovation through scalable manufacturing, targeted delivery and evolving regulatory strategies for genetic medicines.
