Latus Bio: engineered AAV capsids reach up to 80% of kidney glomeruli in primates

Latus Bio Inc., a Philadelphia biotechnology company, reported preclinical results on gene delivery to brain and kidney cells at the 33rd Annual Congress of the European Society of Gene and Cell Therapy in Hamburg, Germany, held Oct. 27–30, 2026, according to mychesco.com mychesco.commychesco.commychesco.comOpen source ↗. The data come from non-human primates and describe engineered adeno-associated virus (AAV) capsids compared with the conventional AAV9 vector.

The work addresses a central problem of gene therapy: delivering genetic material to specific tissues while limiting exposure in organs where treatment is not intended.

Brain and kidney capsids

Latus reported that its pan-neuronal capsid AAV-PN reached about 40% to 60% of neurons across the cerebral cortex and up to 40% of spinal cord motor neurons after low-dose administration into the ventricular system of the brain. Gene expression throughout the central nervous system was substantially higher than with AAV9, which was tested by intracerebroventricular and intravenous routes. Unintended expression in the liver and dorsal root ganglia was lower.

For the kidney, the AAV-Kd-1 capsid reached about 80% of glomeruli in both kidneys after low-dose intravenous administration in adult non-human primates mychesco.commychesco.commychesco.comOpen source ↗. Transduction exceeded 30% in podocytes, glomerular endothelial cells and mesangial cells, and about 30% of proximal tubular cells were reached. In distal tubules and collecting ducts, transduction was more than twice that of AAV9. Other kidney capsid variants, including ones designed to reduce distribution to the liver, are under investigation.

Huntington's and Batten disease candidates

The Huntington's disease candidate LTS-201 combines the AAV-DB-3 capsid with an artificial microRNA that lowers expression of the DNA mismatch repair protein MSH3. MSH3 contributes to somatic instability, a change in repetitive DNA sequences linked to the onset and progression of the disease. In non-human primates, LTS-201 reduced MSH3 messenger RNA in a dose-dependent manner, by up to 94% in more than 90% of medium spiny neurons. In a mouse model, somatic instability in the striatum also decreased dose-dependently.

Latus also presented a computational model built entirely on human data that relates the degree of MSH3 suppression and neuronal targeting to the projected delay of clinical progression. The findings were also published in Nature Communications by Simpson and colleagues, according to the company's Oct. 28 announcement.

LTS-101 delivers a functional TPP1 gene via the Ep+ capsid for CLN2 Batten disease. In a Good Laboratory Practice toxicology study in non-human primates it was well tolerated through six months and maintained TPP1 protein in cerebrospinal fluid at levels the company characterized as potentially therapeutic mychesco.commychesco.commychesco.comOpen source ↗. The U.S. FDA has granted Fast Track, Orphan Drug and Rare Pediatric Disease designations and cleared the Investigational New Drug application for LTS-101.