AAV-DB-3-miRNA / Latus Bio - LARVOL DELTA

Targeting MSH3 for Huntington's Disease: Preclinical Validation of AAV-DB-3-miRNA to Prevent Somatic CAG Repeat Expansion (ASGCT 2025) - "In conclusion, human genetics and preclinical proof-of concept data in HD mouse models support the therapeutic hypothesis that depletion of MSH3 protein using a miRNA expressed by Latus' novel AAV-DB-3 delivery vector, may significantly delay symptom onset in HD through prevention of somatic CAG repeat expansions in the HTT gene. Disease Focus of Abstract:Inherited Neurological Disorders"

Preclinical • CNS Disorders • Gene Therapies • Huntington's Disease • Movement Disorders • MSH3

Latus Bio Announces Data Presentations in Support of its Preclinical Huntington’s Disease Gene Therapy Program (Businesswire) - "Latus Bio, Inc...is pleased to announce data presentations in support of its preclinical drug candidate at the 20th Annual CHDI Huntington’s Disease (HD) Therapeutics Conference in Palm Springs, California, which is being held from February 24-27, 2025...One presentation will focus on a novel computational model derived by Latus that is aimed at predicting the effects of knockdown of the DNA repair enzyme MSH3 on reducing somatic instability and the potential outcomes that could result for HD patients. A second presentation will share new preclinical data, demonstrating MSH3 knockdown via an artificial miRNA packaged in our novel AAV-DB-3 capsid, in a mouse model of HD. The knockdown achieved in this study resulted in reductions in somatic instability at levels that are predicted from the model to yield potential clinical benefits."

Preclinical • Huntington's Disease