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May 18, 2023

SwanBio Unveils Research in Next Generation CNS Capsids at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting

PHILADELPHIA, May 18, 2023SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, inherited neurological conditions, presented preliminary data from primate studies of AAV capsids that outperform the commonly used AAV9 capsid. The findings illuminate SwanBio’s vector engineering capabilities and ongoing pursuit of novel capsids that deliver enhanced transgene expression, biodistribution, and safety profiles.

“Our vector engineering platform, along with our proprietary insights into delivery and biodistribution, and expertise in cassette design, form the three pillars of how we approach gene therapy,” said Karen Kozarsky, co-founder and chief scientific officer, SwanBio. “This research, conducted with our partners at the Mass General Research Institute’s Maguire Lab, indicates the potential of our innovative approach to capsid optimization. We are excited by the potential to incorporate these next-gen capsids into our pipeline products and future portfolio.”

Key findings presented at the 2023 ASGCT Annual Meeting include:

  • Screening of a capsid library delivered intrathecally in primates resulted in the identification of a pool of 13 new capsid candidates, which displayed enhanced biodistribution compared to AAV9 at all levels of the spinal cord, ranging from 2- to 265-fold.
  • Four of seven candidate capsids demonstrated enhanced transgene expression in the spinal cord (up to 2.4-fold).
  • Several capsids displayed significant reduction in off-target distribution, including lower leakage to the liver (up to 1,250-fold) compared to AAV9.

Additional research in mice will evaluate transduction performance of the most promising capsids following intrathecal delivery, for application in new disease models.

SwanBio is developing a pipeline of gene therapy candidates designed to be delivered intrathecally to targets in both the central and peripheral nervous systems, with an initial focus on spinal cord-related disorders. The company’s lead candidate, SBT101, is currently being investigated for the treatment of adrenomyeloneuropathy (AMN) in a Phase 1/2 clinical trial, PROPEL.

SwanBio is also presenting a poster at ASGCT showcasing a recent healthcare resource use study of men and women living with AMN. The research indicates that AMN imposes a substantial and previously under-recognized health burden, pointing to the value of developing targeted genetic therapies.

To view SwanBio’s final 2023 ASGCT posters, visit


Chelcie Lister


Lara Furst