LTS-101 / Latus Bio - LARVOL DELTA

LTS-101: An Intracerebroventricular Delivered AAV Gene Therapy Using a Novel Capsid Variant for the Treatment of CLN2 Batten Disease (ESGCT 2026) - No abstract available

Gene therapy • CNS Disorders • Gene Therapies

A 3 and 6 month GLP toxicology study in cynomolgus monkeys of a single intracerebroventricular dose of LTS-101 to support development as an AAV gene therapy candidate for treatment of CLN2 Batten disease (ASGCT 2026) - "While bi-monthly infusions of recombinant human TPP1 (cerliponase alfa) to the brain have provided benefit to patients, significant disease burden remains and the therapy requires families to relocate in proximity to infusion centers for the frequent, hours long, infusions...All animals were immunosuppressed with prednisone and tacrolimus...Clinical development planning is progressing in collaboration with world-leaders in CLN2 disease. LTS-101 is a promising gene therapy candidate with the potential to treat CLN2 Batten disease patients with a one-time ICV administration providing stable, long-term expression of TPP1 while eliminating the caregiver and patient burden of repeated ERT treatments."

Gene therapy • CNS Disorders • Gene Therapies • Ophthalmology • TPP1