PHILADELPHIA, PA — Latus Bio Inc. has reported preclinical advances in delivering gene therapies to brain and kidney cells, demonstrating improved targeting compared with conventional viral delivery technology as the Philadelphia biotechnology company develops treatments for neurological and renal diseases.
The findings, presented at the European Society of Gene and Cell Therapy’s 33rd Annual Congress in Hamburg, Germany, Oct. 27-30, 2026, showed that engineered adeno-associated virus, or AAV, capsids could deliver genetic material to a substantial proportion of targeted cells in non-human primates.
The research addresses a central obstacle in gene therapy development: delivering therapeutic genetic material to specific tissues while limiting exposure in organs where treatment is not intended.
Latus reported that its experimental pan-neuronal capsid, AAV-PN, reached approximately 40% to 60% of neurons across the cerebral cortex and up to 40% of spinal cord motor neurons following low-dose administration into the brain’s ventricular system.
The company also reported substantially greater gene expression throughout the central nervous system than was achieved using AAV9, a conventional viral vector evaluated through both intracerebroventricular and intravenous administration.
AAV-PN demonstrated lower unintended gene expression in peripheral tissues, including the liver and dorsal root ganglia, according to the preclinical findings.
The results suggest that the engineered capsid could improve the distribution of gene therapies intended to treat neurological conditions affecting multiple regions of the brain and spinal cord.
Latus separately reported advances in kidney-targeted gene delivery, an area where conventional AAV vectors have faced limitations in reaching disease-relevant cells.
Its engineered AAV-Kd-1 capsid reached approximately 80% of glomeruli in both kidneys following low-dose intravenous administration in adult non-human primates.
Glomeruli are structures within the kidneys responsible for filtering blood, making them important targets for therapies addressing certain renal disorders.
Cell-specific measurements showed transduction rates exceeding 30% in podocytes, glomerular endothelial cells and mesangial cells, all of which contribute to kidney filtration and structural function.
The capsid also reached approximately 30% of proximal tubular cells and achieved more than twice the transduction of distal tubules and collecting ducts compared with AAV9.
Latus reported that additional kidney-targeting capsid variants are under investigation, including candidates designed to improve delivery to kidney tissues while reducing distribution to the liver.
The brain and kidney findings reflect the company’s strategy of engineering viral capsids to improve the delivery of genetic therapies to particular cell populations.
AAV capsids are protein structures that package and transport genetic material into cells. Their ability to reach specific tissues can influence the effectiveness and safety of experimental gene therapies.
Beyond its delivery technology, Latus presented additional findings from two investigational treatments targeting inherited neurological diseases.
Its Huntington’s disease candidate, LTS-201, combines an engineered AAV capsid known as AAV-DB-3 with an artificial microRNA designed to reduce expression of the DNA mismatch repair protein MSH3.
Huntington’s disease is an inherited neurological disorder associated with progressive deterioration in movement, cognition and behavior.
MSH3 contributes to somatic instability, a process involving changes in repetitive DNA sequences that has been linked to the onset and progression of Huntington’s disease.
Latus is investigating whether suppressing MSH3 in vulnerable neurons could slow that process and delay disease progression.
The company’s engineered capsid is designed to target medium spiny neurons in the striatum and projection neurons in deeper layers of the cerebral cortex, cell populations relevant to Huntington’s disease.
In non-human primates, LTS-201 produced dose-dependent reductions in MSH3 messenger RNA, reaching as much as 94% suppression in more than 90% of medium spiny neurons.
Separate experiments in a mouse model of Huntington’s disease showed dose-dependent reductions in somatic instability within the striatum.
The company also presented a computational model developed entirely from human data that estimates how different levels of MSH3 suppression and neuronal targeting could affect the timing of clinical disease progression.
The model connects the extent of gene suppression with projected delays in disease progression, providing a framework for evaluating the potential therapeutic effects of LTS-201.
The findings were also published in Nature Communications in research by Simpson and colleagues, according to Latus’ Oct. 28 announcement.
The company presented a fourth set of findings involving LTS-101, its investigational gene therapy for CLN2 Batten disease, a rare inherited neurological disorder.
LTS-101 uses an engineered capsid known as Ep+ to deliver a functional human tripeptidyl peptidase 1, or TPP1, gene.
The treatment is intended to address the underlying enzyme deficiency associated with CLN2 Batten disease.
In a non-human primate toxicology study conducted under Good Laboratory Practice standards, LTS-101 was well tolerated through six months following administration.
The treatment also maintained levels of TPP1 protein in cerebrospinal fluid that the company characterized as potentially therapeutic.
LTS-101 has received Fast Track, Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration.
The FDA has also cleared the company’s Investigational New Drug application for LTS-101, allowing the treatment to advance into clinical investigation.
The regulatory designations recognize the treatment’s development for a rare pediatric condition and provide access to certain regulatory development pathways.
Together, the findings provide additional preclinical evidence supporting Latus’ engineered gene delivery technology and its investigational neurological treatments.
The next stage of development will determine whether the delivery efficiency and biological effects observed in laboratory and animal studies can translate into meaningful clinical benefits for patients.
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