Latus Bio Nabs $97 Million to Continue Neurodegenerative Gene Therapy Pipeline
The funding will support Latus Bio’s lead gene therapy programs in Huntington’s and CLN2 disease while advancing its low-dose AAV delivery platform designed to make gene therapies safer, more scalable, and accessible to broader patient populations.
Key Points
- Latus Bio has raised $97 million in Series A financing to advance its lead gene therapy programs in Huntington’s disease and CLN2 disease.
- The company’s platform uses engineered AAV capsids and low-dose delivery approaches that it says could improve safety, manufacturability, and scalability.
- LTS-201 is on track for an IND submission in the third quarter of 2026, while LTS-101 is expected to generate initial clinical data by the end of 2026.
Latus Bio is developing gene therapies for central nervous system and peripheral nervous system diseases using proprietary, engineered AAV capsids paired with what it describes as optimized clinical delivery routes, and it just closed a $97 million Series A financing to support that mission.
Platform and pipeline
- LTS-201: An investigational AAV gene therapy for Huntington’s disease designed to reduce expression of MSH3, a DNA repair enzyme linked to somatic instability. The company says preclinical work showed strong targeting of medium spiny neurons in the deep brain. Latus expects to submit an IND in the third quarter of 2026.
- LTS-101: A program for CLN2 disease, a rare and fatal neurodegenerative disorder with no curative solutions. The candidate has received IND clearance, Orphan Drug Designation, Rare Pediatric Disease Designation, and Fast Track Designation from the FDA. An investigator-initiated first-in-human trial is expected to begin in the third quarter of 2026, with initial safety, biomarker, and clinical results expected by year-end.
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Beyond CNS disorders, Latus said it is developing capsid variants for potential use in kidney, eye, heart, and muscle diseases.
P. Peter Ghoroghchian, M.D., Ph.D., CEO of Latus Bio, said: “By combining proprietary and engineered AAV capsids with optimal routes for clinical delivery, we aim to achieve robust cell- and tissue-specific transduction at low doses, which we believe is critical to improving safety, efficacy, manufacturability, and costs. This approach enables a repeatable model for developing therapies across multiple underserved indications with significant unmet need. We are advancing LTS-201 toward a first-in-human study in Huntington’s disease, which is our first step into large-rare CNS indications. In parallel, LTS-101 for CLN2 disease will advance towards initial clinical data in late 2026 through an investigator-initiated trial.”
More information is available at latusbio.com.
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