Latus Bio Presents New Preclinical Gene Therapy Data for CNS and Kidney-Targeting AAV Capsids at ESGCT 2026
Latus Bio, Inc., a biotechnology company focused on engineering and developing advanced gene therapies at scale, has announced new preclinical findings demonstrating the potential of its proprietary adeno-associated virus (AAV) capsid engineering platform. The latest data were presented through four scientific posters at the European Society of Gene and Cell Therapy (ESGCT) 33rd Annual Congress, held October 27–30, 2026, in Hamburg, Germany.
The presentations highlighted the capabilities of Latus’ integrated discovery engine to develop differentiated AAV-based gene therapies targeting both the central nervous system (CNS) and peripheral tissues. The findings included new preclinical evidence from proprietary pan-neuronal and kidney-targeting capsid variants, which demonstrated improved performance compared with conventional AAV9 in several important measures of tissue delivery and transgene expression.
In addition to its capsid engineering research, Latus presented further preclinical findings supporting two investigational gene therapy programs: LTS-201 for Huntington’s disease (HD) and LTS-101 for CLN2 Batten disease. Both programs use previously disclosed engineered AAV capsids designed to improve the delivery of therapeutic genetic material to specific tissues and cell populations.
The four posters collectively demonstrated the company’s efforts to overcome some of the major challenges associated with gene therapy development, particularly the need for more efficient tissue targeting, improved cellular specificity and reduced unintended exposure in non-target tissues.
The findings also reinforce Latus’ strategy of developing engineered AAV vectors capable of delivering therapeutic genes to specific organs and cell types while potentially improving the overall efficiency and precision of gene therapy treatments.
Engineered Pan-Neuronal Capsid Demonstrates Broad CNS Delivery
One of the major presentations focused on Latus’ proprietary engineered AAV pan-neuronal (AAV-PN) capsid variant, which has been developed to achieve broad and efficient neuronal delivery throughout the brain and spinal cord.
Delivering therapeutic genetic material across the CNS remains a significant challenge in gene therapy development. Different regions of the brain and spinal cord contain diverse neuronal populations, and achieving sufficient distribution across these areas is important for treating neurological disorders that affect multiple parts of the nervous system.
At the same time, limiting unintended gene expression in peripheral tissues is an important consideration for improving the precision and safety profile of investigational gene therapies.
Latus designed its AAV-PN capsid to address these challenges by facilitating widespread neuronal transduction while reducing off-target expression outside the CNS.
The newly presented preclinical findings were generated following low-dose intracerebroventricular (ICV) administration in adult non-human primates (NHPs). ICV administration involves delivering a therapeutic agent into the cerebrospinal fluid through the brain’s ventricular system, providing a route for distributing gene therapy vectors throughout the CNS.
The study demonstrated several important characteristics of the engineered capsid.
AAV-PN achieved widespread biodistribution and transgene expression throughout the CNS. Native fluorophore expression indicated approximately 40% to 60% neuronal transduction across the cortex. The capsid also achieved transduction of up to approximately 40% of spinal cord motor neurons.
These findings suggest that the engineered capsid can efficiently reach multiple neuronal populations in different anatomical regions of the nervous system following low-dose administration.
Importantly, AAV-PN demonstrated markedly higher CNS transgene expression compared with AAV9 administered through either the ICV or intravenous (IV) route.
The engineered capsid also showed substantially lower off-target expression in peripheral tissues, including the dorsal root ganglia and liver.
The dorsal root ganglia contain sensory neurons that can be exposed to certain AAV vectors during systemic or CNS-directed administration. Reducing unintended vector distribution and transgene expression in these tissues is an important consideration in the development of neurological gene therapies.
By demonstrating improved neuronal delivery alongside reduced peripheral tissue expression, AAV-PN could provide a differentiated platform for developing gene therapies targeting a range of CNS disorders.
The findings support continued investigation of the capsid’s potential applications in diseases requiring widespread delivery of therapeutic genetic material across the brain and spinal cord.
Kidney-Targeting AAV Capsids Show Improved Tissue Transduction
A second major area of research presented at ESGCT 2026 focused on Latus’ proprietary kidney-targeting AAV capsid variants, collectively referred to as AAV-Kd.
These engineered capsids are designed to improve gene delivery to different cellular compartments within the kidneys, including glomerular and tubular structures.
The kidneys contain several specialized cell types responsible for filtering blood, regulating fluid balance and maintaining electrolyte levels. Effective gene therapy for kidney disorders may require the ability to reach multiple cellular populations rather than targeting only one specific region.
Conventional AAV vectors, including AAV9, may have limitations in achieving efficient and widespread gene delivery across the different structures of the kidney. Latus is seeking to address these challenges through capsid engineering aimed at improving organ-specific targeting and cellular transduction.
The newly presented preclinical findings demonstrated that AAV-Kd variants achieved broad kidney transduction following low-dose intravenous administration in adult NHPs.
Among the evaluated candidates, the fully characterized AAV-Kd-1 variant demonstrated a biodistribution and transduction profile substantially superior to intravenously administered AAV9.
The study reported that AAV-Kd-1 achieved transduction of approximately 80% of glomeruli across both kidneys. This finding highlights the capsid’s ability to reach a substantial proportion of the kidney’s filtering structures.
Further cell-specific quantification demonstrated greater than 30% transduction of several important glomerular cell populations, including:
- Podocytes, which play an essential role in maintaining the kidney’s filtration barrier.
- Glomerular endothelial cells, which line the blood vessels within the glomeruli.
- Mesangial cells, which provide structural support and contribute to the regulation of glomerular function.
The ability to reach these different cell populations is particularly relevant for developing genetic medicines targeting kidney diseases involving multiple cellular compartments.
AAV-Kd-1 also demonstrated encouraging results in tubular structures. The capsid achieved up to approximately 30% transduction of proximal tubules.
Additionally, transduction of distal tubules and collecting ducts was more than two times higher than that observed with AAV9.
These findings indicate that the engineered capsid may provide improved access to both glomerular and tubular regions of the kidneys.
Latus also reported that ongoing research is supporting promising profiles for additional emerging AAV-Kd candidates. These include other engineered variants demonstrating superior kidney targeting and liver detargeting characteristics.
Reducing liver exposure while improving delivery to the intended organ is an important objective in the development of systemic gene therapies. The company’s continued capsid engineering efforts are intended to improve tissue selectivity and support the development of more targeted genetic medicines.
The kidney-targeting findings expand the potential applications of Latus’ AAV discovery platform beyond neurological disorders and demonstrate its broader capabilities in engineering vectors for peripheral organs.
LTS-201 Advances as an Investigational Gene Therapy for Huntington’s Disease
A third poster presented additional preclinical research supporting LTS-201, Latus’ investigational AAV gene therapy candidate for Huntington’s disease.
Huntington’s disease is a progressive inherited neurological disorder characterized by the gradual deterioration of nerve cells in the brain. The condition can affect movement, cognition and mental health, eventually interfering with a person’s ability to perform everyday activities.
The disease is associated with a genetic mutation in the huntingtin (HTT) gene. One important feature of Huntington’s disease is somatic instability, a process in which the disease-associated genetic expansion can increase in certain cells over time.
Research has identified DNA mismatch repair pathways as important contributors to this process. Among the proteins involved, DNA mismatch repair protein MSH3 has emerged as a key pathogenic driver associated with somatic instability and causally linked to the onset and progression of Huntington’s disease.
LTS-201 has been designed to address this mechanism by selectively reducing MSH3 expression.
The investigational therapy combines AAV-DB-3, a previously described engineered capsid, with an artificial microRNA designed to selectively lower MSH3 expression.
AAV-DB-3 was engineered to efficiently target striatal medium spiny neurons (MSNs) and layer-5/6 cortical projection neurons. These neuronal populations are particularly relevant to Huntington’s disease because of their involvement in neurological functions affected by the condition.
By combining targeted delivery with an artificial microRNA approach, LTS-201 is intended to reduce the expression of MSH3 in relevant neuronal populations and potentially limit the biological processes contributing to disease progression.
Human-Based Computational Model Supports LTS-201 Development
An important component of the third poster was the presentation of the first computational model of Huntington’s disease pathogenesis built entirely from human data.
The model was developed to quantitatively predict the potential benefits of targeting MSH3 and reducing somatic instability in patients with Huntington’s disease.
It establishes relationships between the level of MSH3 suppression, the percentage of targeted medium spiny neurons and the potential number of years by which clinical disease progression could be delayed.
This approach is intended to provide a more quantitative framework for evaluating the potential therapeutic activity of LTS-201.
The computational model integrates human-derived information with preclinical findings to identify thresholds that may be relevant to therapeutic effectiveness.
By connecting the degree of MSH3 suppression with neuronal targeting, the model provides a way to assess how changes in treatment activity could influence the potential impact on disease progression.
The research also integrates model-predicted thresholds for therapeutic activity with validating preclinical data, providing additional support for the development strategy behind LTS-201.
In non-human primates, LTS-201 achieved deep, dose-dependent reductions in MSH3 messenger RNA (mRNA). The findings demonstrated reductions of up to 94% in more than 90% of medium spiny neurons.
These results indicate that the investigational therapy can achieve substantial target suppression in the neuronal population relevant to Huntington’s disease.
Further preclinical findings were generated in a mouse model of Huntington’s disease, where LTS-201 produced dose-dependent reductions in striatal somatic instability.
Together, the findings provide evidence supporting the candidate’s proposed mechanism of action and demonstrate the potential of combining engineered AAV delivery with targeted gene regulation.
The research was published by Simpson et al. in Common Nature, as previously announced by Latus on October 28, 2026.
The combination of human-based computational modeling, NHP findings and disease-model data represents an important component of the company’s strategy to translate genetic and biological insights into potential treatments for progressive neurological disorders.
LTS-101 Demonstrates Sustained TPP1 Expression in CLN2 Batten Disease Research
The fourth poster presented preclinical findings from a good laboratory practice (GLP) toxicology study in non-human primates evaluating LTS-101, Latus’ investigational AAV gene therapy for CLN2 Batten disease.
CLN2 Batten disease is a rare, inherited neurodegenerative disorder associated with deficiencies in the enzyme tripeptidyl peptidase 1 (TPP1). Insufficient TPP1 activity results in the accumulation of cellular waste materials, contributing to progressive neurological damage and significant disease burden.
LTS-101 is being developed as a gene replacement therapy designed to deliver a functional human TPP1 transgene to address the underlying enzyme deficiency.
The investigational treatment uses Latus’ engineered Ep+ capsid variant to deliver the genetic material responsible for producing human TPP1.
The latest GLP toxicology findings demonstrated that LTS-101 was well tolerated in NHPs through six months following administration.
The study also showed sustained levels of TPP1 protein in the cerebrospinal fluid throughout the six-month observation period. The reported protein levels were potentially therapeutic, supporting the candidate’s ability to deliver and maintain expression of the intended enzyme.
Sustained TPP1 expression is an important objective for a gene replacement approach targeting CLN2 Batten disease because the condition is associated with an underlying deficiency in this essential enzyme.
The preclinical findings provide additional information about the investigational therapy’s tolerability and its ability to maintain TPP1 protein levels over an extended observation period.
LTS-101 has also received three important regulatory designations from the US Food and Drug Administration (FDA):
- Fast Track Designation, intended to facilitate the development and expedite the review of therapies addressing serious conditions with unmet medical needs.
- Orphan Drug Designation, which supports the development of medicines for rare diseases and may provide regulatory and development incentives.
- Rare Pediatric Disease Designation, which recognizes qualifying therapies targeting serious or life-threatening diseases that primarily affect children.
In addition to these designations, Latus announced that its Investigational New Drug (IND) application for LTS-101 has been cleared by the FDA.
The IND clearance represents an important regulatory milestone, allowing the company to proceed with clinical investigation of the candidate under the applicable regulatory requirements.
The combination of sustained TPP1 expression, six-month tolerability findings and regulatory progress supports the continued advancement of LTS-101 as a potential gene therapy for CLN2 Batten disease.
Latus Strengthens Its Engineered AAV Gene Therapy Platform
The four scientific presentations at ESGCT 2026 highlight the breadth of Latus’ integrated AAV discovery and development capabilities.
The company’s research encompasses engineered capsids designed for specific tissue targets, as well as investigational gene therapy programs addressing serious neurological and genetic diseases.
The AAV-PN findings demonstrate the potential for improved neuronal delivery throughout the brain and spinal cord while limiting peripheral exposure. Meanwhile, AAV-Kd-1 and related kidney-targeting variants have shown the ability to reach multiple glomerular and tubular cell populations with improved transduction compared with AAV9.
The LTS-201 program illustrates how engineered capsids can be combined with genetic regulation technologies to target disease-associated biological mechanisms. Its development incorporates human-based computational modeling and preclinical evidence supporting MSH3 suppression as a potential approach to reducing somatic instability in Huntington’s disease.
Similarly, LTS-101 demonstrates the application of engineered AAV technology to enzyme replacement through gene delivery, with preclinical findings supporting sustained TPP1 expression in the context of CLN2 Batten disease.
These programs reflect the company’s broader objective of improving the precision, efficiency and tissue selectivity of AAV-based gene therapies.
Advancing Potential Treatments for Serious Genetic Diseases
The latest preclinical data represent an important development milestone for Latus as it continues to expand its engineered AAV platform across CNS and peripheral tissue applications.
The findings presented at the ESGCT 33rd Annual Congress provide additional evidence of the potential advantages of proprietary capsid engineering compared with conventional AAV9 delivery in selected preclinical settings.
The company’s immediate research priorities include further evaluating its pan-neuronal and kidney-targeting capsids while advancing its investigational programs for Huntington’s disease and CLN2 Batten disease.
With LTS-101’s IND application cleared by the FDA and its existing Fast Track, Orphan Drug and Rare Pediatric Disease designations, the company has reached an important regulatory milestone in its CLN2 development program.
Meanwhile, LTS-201 continues to build on preclinical findings demonstrating substantial MSH3 suppression and reductions in somatic instability.
Although additional clinical research will be necessary to establish the safety and effectiveness of these investigational therapies, the latest findings provide further support for Latus’ approach to engineering AAV vectors for targeted genetic medicine development.
Through its integrated discovery engine, proprietary capsid technologies and disease-focused research programs, Latus aims to advance a new generation of gene therapies designed to address the underlying biological mechanisms of serious genetic disorders.
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 .