Shots:
- The US FDA has approved the IND application of NVC-001 to treat LMNA-related dilated cardiomyopathy (LMNA DCM), enabling a P-I/II trial to start in early 2026
- Preclinical studies showed that NVC-001 significantly improved survival and cardiac function. They planned a P-I/II trial, a 52-week, open-label, multicenter ascending-dose study to assess the safety, tolerability, and preliminary efficacy of one-time IV NVC-001 in adults with LMNA-DCM
- NVC-001 is an AAV-based gene therapy for LMNA-DCM that reduces abnormal mechanical stress on the nucleus to restore nuclear envelope integrity, addressing the disease’s root cause
Ref: Prnewswire| Image: Prnewswire| Press Release
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