PHILADELPHIA, PA — Latus Bio is advancing experimental gene therapies for Huntington’s disease and CLN2 Batten disease while developing delivery technology intended to improve how genetic treatments reach brain, spinal cord and kidney cells, according to preclinical research scheduled for presentation at a European scientific conference next week.
The Philadelphia biotechnology company will present four research posters at the European Society of Gene and Cell Therapy’s 33rd Annual Congress, scheduled for October 27-30 in Hamburg, Germany.
The presentations cover two investigational treatments and engineered adeno-associated virus (AAV) capsids, the protein shells used to deliver genetic material into cells. Improving the ability of these delivery systems to reach specific tissues is a central component of Latus’ development strategy.
One presentation will examine a proprietary AAV pan-neuronal capsid variant engineered to deliver genetic material broadly throughout the central nervous system while preferentially targeting neurons.
The company will also present preclinical findings involving an engineered kidney-targeting capsid designed to deliver genetic material across multiple kidney cell types relevant to renal disease.
The therapeutic research includes LTS-201, an investigational gene therapy for Huntington’s disease that seeks to address somatic instability, a biological process associated with changes in the length of repeated DNA sequences.
LTS-201 is designed to reduce expression of MSH3, a gene involved in DNA mismatch repair. The treatment combines an engineered AAV-DB-3 capsid with an artificial microRNA intended to selectively lower MSH3 expression.
The capsid is designed to target neuronal populations implicated in Huntington’s disease, including neurons in the basal ganglia and cerebral cortex.
Latus will present computational modeling and preclinical evidence supporting the treatment’s development. The findings do not establish clinical effectiveness in patients.
A separate presentation will address LTS-101, an investigational gene therapy for CLN2 Batten disease, a rare inherited neurological disorder.
LTS-101 uses the company’s AAV-Ep+ capsid variant to deliver the human TPP1 gene. The therapy is being developed for administration directly into the brain’s ventricular system.
The research program is intended to address the underlying genetic deficiency associated with CLN2 disease, although the company has not provided clinical efficacy results in the announcement.
The four presentations are scheduled as follows:
- October 28, 1:30–3:30 p.m. CET: LTS-101 gene therapy for CLN2 Batten disease, Poster P0479.
- October 29, 2–4 p.m. CET: Computational modeling and preclinical evidence for LTS-201 in Huntington’s disease, Poster P0480.
- October 29, 2–4 p.m. CET: Engineered neuronal-targeting AAV capsids for brain and spinal cord delivery, Poster P0486.
- October 29, 2–4 p.m. CET: Engineered kidney-targeting AAV capsids for renal gene therapy, Poster P0050.
The presentations will provide additional preclinical information on Latus’ delivery platform and its potential applications across neurological and kidney disorders.
The accepted abstracts are available online to registered congress participants.
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