Hamburg, Germany – October 29, 2026 — During the 33rd Annual Congress of the European Society of Gene & Cell Therapy (ESGCT), Byongen Therapeutics presented new preclinical data on its central nervous system (CNS)-targeted AAV capsid, SynCAP™-BY368, in a poster presentation.
BY368 is an AAV capsid displaying a human Neuroligin-3-derived peptide, isolated through three rounds of non-human primate (NHP) intrathecal (IT) screening. It was developed to address a core bottleneck in CNS gene therapy: achieving efficient, widespread neuronal transduction across broad brain regions and deep parenchyma via the minimally invasive, clinically established intrathecal route.
Key Preclinical Data Highlights:
⭐Superior CNS Transduction Efficiency with Significantly Reduced Peripheral Toxicity Risk
In rhesus macaque models, BY368 achieved approximately 36-fold higher brain mRNA expression and 11-fold higher spinal cord expression on average compared with AAV9, while markedly reducing expression in peripheral tissues such as liver and heart.
RNA-seq analysis shows that after intrathecal delivery, BY368 delivers substantially higher mRNA expression than AAV9 across brain and spinal cord regions, with markedly reduced expression in peripheral tissues. Y-axis: Log2 fold change versus AAV9.
⭐Deep Penetration and Broad Neuronal Targeting
In dual-species models (rhesus and cynomolgus macaques), BY368 efficiently penetrated cortex, cerebellum, thalamus, hippocampus, and other deep brain regions, with widespread colocalization with the mature neuronal marker NeuN.
Immunofluorescence images confirm that after intrathecal delivery, BY368 achieves widespread colocalization with the mature neuronal marker NeuN across spinal cord, cerebellum, brainstem, cortex, thalamus, and hippocampus.
⭐High Cross-Species Consistency
The above NHP findings were further corroborated in mouse intracerebroventricular (ICV) delivery models, demonstrating strong translational potential.
Byongen Therapeutics is actively seeking strategic partners to advance gene therapy programs based on SynCAP™-BY368. To access the full dataset, download the original ESGCT poster (PDF) at https://www.byongen.com, or contact: info@byongen.com.
About Byongen Therapeutics
Byongen Therapeutics is a biotechnology company focused on original innovation in AAV delivery. Leveraging its proprietary DynEvoLib® platform — which integrates AI, rational design, and high-throughput directed evolution — the company drives the precision evolution of next-generation AAV capsids. Its research spans neuromuscular, CNS, and cardiovascular indications.
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