Data on pan-neuronal AAV capsid variant demonstrate widespread CNS transduction and neuronal targeting in non-human primates
Novel kidney-targeting capsid variants achieve substantially improved transduction of disease-relevant cells in glomeruli and tubules in preclinical studies compared with conventional AAVs
Additional preclinical data highlighting pipeline programs for Huntington’s disease (LTS-201) and CLN2 Batten disease (LTS-101) also presented
PHILADELPHIA & BOSTON--(BUSINESS WIRE)--Latus Bio, Inc. (Latus), a biotechnology company engineering gene therapies at scale, today announced new preclinical data presented at the European Society of Gene and Cell Therapy (ESGCT) 33rd Annual Congress, held October 27-30, 2026, in Hamburg, Germany. The posters showcased the ability of Latus’ integrated discovery engine to generate differentiated, engineered AAV gene therapies for central nervous system (CNS) and peripheral tissues, including new preclinical data on proprietary pan-neuronal and kidney-targeting capsid variants with superior performance relative to AAV9. The poster presentations also highlighted investigational gene therapy programs for Huntington's disease (HD) (LTS-201) and CLN2 Batten disease (LTS-101) that are enabled by previously disclosed engineered capsids.
Latus' proprietary engineered AAV pan-neuronal (AAV-PN) capsid variant is designed to achieve broad and efficient neuronal delivery throughout the brain and spinal cord while limiting off-target expression in peripheral tissues, a central goal in CNS gene therapy. Following low-dose intracerebroventricular (ICV) administration in adult non-human primates (NHPs), AAV-PN demonstrates:
- Widespread CNS biodistribution and transgene expression
- Approximately 40-60% neuronal transduction across the cortex and up to 40% transduction of spinal cord motor neurons based on native fluorophore expression
- Markedly higher CNS transgene expression than either ICV or intravenous (IV)-administered AAV9, with substantially lower off-target expression in peripheral tissues, including the dorsal root ganglia and liver
Latus' engineered kidney-targeting capsid (AAV-Kd) variants demonstrate broad transduction across glomerular and tubular compartments following low-dose IV administration in adult NHPs. These data show the fully characterized AAV-Kd-1 capsid variant achieves a biodistribution and transduction profile that is substantially superior to IV-administered AAV9:
- Transduction of approximately 80% of glomeruli in both kidneys
- Greater than 30% transduction of podocytes, glomerular endothelial cells and mesangial cells by cell-specific quantification
- Up to approximately 30% transduction of proximal tubules and >2x higher transduction of distal tubules and collecting ducts than AAV9
- Ongoing studies support promising profiles for emerging capsids, including additional AAV-Kd variants with superior kidney targeting and liver detargeting
A third poster highlighted preclinical data on LTS-201, Latus’ investigational AAV gene therapy for HD. LTS-201 combines AAV-DB-3, a previously described capsid engineered for efficient targeting of striatal medium spiny neurons (MSNs) and layer-5/6 cortical projection neurons, with an artificial microRNA designed to selectively lower expression of the DNA mismatch repair protein MSH3. MSH3 is the key pathogenic driver of somatic instability and causally linked to HD onset and progression. The poster presented the first computational model of HD pathogenesis built entirely from human data. The model quantitatively predicts the benefits of targeting MSH3, and therefore somatic instability, in HD, by linking levels of MSH3 suppression and percentages of targeted MSNs to the number of potential years of delay in clinical HD progression. The research integrates model-predicted thresholds for therapeutic activity with validating preclinical data. In NHPs, LTS-201 achieved deep, dose-dependent reductions in MSH3 mRNA (up to 94% in >90% of MSNs); in a mouse model of HD, it produced dose-dependent reductions in striatal somatic instability. As previously announced on October 28, 2026, these data were published by Simpson et al. in Nature Commun.
Preclinical data from an NHP GLP toxicology study of LTS-101, Latus’ investigational AAV gene therapy for CLN2 Batten disease, were featured in a fourth poster. LTS-101 leverages the engineered Ep+ capsid variant to deliver a human tripeptidyl peptidase 1 (TPP1) transgene. Through six months after administration, LTS-101 was well tolerated and sustained potentially therapeutic levels of TPP1 protein in the cerebrospinal fluid of NHPs. LTS-101 holds Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for the treatment of CLN2 Batten disease. Latus’ Investigational New Drug (IND) application for LTS-101 has been cleared by the FDA.
About Latus Bio
Latus is a biotechnology company dedicated to addressing devastating CNS and peripheral diseases via innovative and scalable gene therapies. The Company is advancing a broad therapeutics pipeline based on novel AAV capsid variants with potency and specificity. Latus is powered by a diverse team of visionary scientists, experienced clinicians, and leading industry executives. The Company has offices in Philadelphia, PA and in the Seaport in Boston, MA. For more information, visit www.latusbio.com.
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