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

18 May 2023
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Orphan DrugGene TherapyFast TrackClinical Study
PHILADELPHIA--(BUSINESS WIRE)-- SwanBio 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 pro “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 swanbiotx.com/investors-and-media/events-and-presentations/. About Adrenomyeloneuropathy Adrenomyeloneuropathy (AMN) is a progressive and debilitating neurodegenerative disease caused by mutations in the ABCD1 gene that disrupt the function of spinal cord cells and other tissues. AMN is characterized by loss of mobility in adulthood, incontinence, pain, and sexual dysfunction, which all affect quality of life. Patients often experience adrenal gland dysfunction as well. Between 8,000-10,000 men in the United States and EU5 (France, Germany, Italy, Spain, and the United Kingdom) are living with AMN. There are no approved therapies for the treatment of the disease; current standard of care is limited to symptom management. About SBT101 SBT101 is the first clinical-stage adeno-associated virus (AAV)-based gene therapy candidate for people with adrenomyeloneuropathy (AMN). In preclinical studies, treatment with SBT101 demonstrated dose-dependent improvement of disease markers and functional improvement in AMN mouse models. SBT101 was also shown to be well-tolerated in non-human primates through six months post-treatment. The ongoing clinical program for SBT101 builds on this positive preclinical data, plus SwanBio’s deep understanding of the underlying pathophysiology of AMN and the AMN patient experience, including new insights being gathered in a proprietary natural history study, CYGNET. SBT101 has been granted Fast Track and Orphan Drug Designation from the U.S. Food and Drug Administration and Orphan Drug Designation from the European Medicines Agency. About SwanBio Therapeutics SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, inherited neurological conditions. SwanBio is advancing a pipeline of gene therapies, designed to be delivered intrathecally, that can address targets within both the central and peripheral nervous systems. This approach has the potential to be applied broadly across three disease classifications – spastic paraplegias, monogenic neuropathies, and polygenic neuropathies. SwanBio’s lead program is being advanced toward clinical development for the treatment of adrenomyeloneuropathy (AMN). SwanBio is supported by long-term, committed investment partners, including its primary investors Syncona, Ltd. (lead investor and majority shareholder) and Mass General Brigham Ventures. For more information, visit SwanBioTx.com. View source version on businesswire.com: Contacts Media: Lara Furst +1-703-946-0183 media@swanbiotx.com Investor: Jan Case investors@swanbiotx.com Source: SwanBio Therapeutics View this news release online at:
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