We Share Findings from Investigational Gene Therapy Program at ASGCT Annual Meeting Scientific Symposium.

NEW ORLEANS, LA — May 14, 2026 — Dr. Jude Samulski today presented clinical trial data on the development of a next-generation adeno-associated virus (AAV) vector for spinal muscular atrophy (SMA) during the Innovative Modalities in Cell and Gene Therapy: From Editing to Delivery scientific symposium at the American Society of Gene and Cell Therapy (ASGCT)

The presentation, delivered on May 14, 2026, highlighted advances in AAV vector engineering aimed at improving the safety, efficacy, and durability of gene replacement therapy for SMA — a devastating neuromuscular disease caused by mutations in the SMN1 gene.

Key topics covered in the presentation include:

• Engineering rational and preclinical validation of the next-generation AAV capsid for central nervous system (CNS) targeting
• Clinical trial design, patient selection criteria, and dosing strategy of NKG001
• Interim safety and biomarker data from enrolled SMA patients
• Comparative analysis with first-generation AAV9-based SMA gene therapies
• Perspectives on scalable manufacturing and global access

  “SMA gene therapy has transformed the treatment landscape, but the field continues to push boundaries — improving transduction efficiency, reducing immunogenicity, and extending therapeutic durability,” said Dr. Jude Samulski. “Our clinical data represents an important step toward a next-generation solution for SMA patients worldwide.”