PHILADELPHIA--(BUSINESS WIRE)--#AAV--Latus Bio, Inc. (Latus), a biotechnology company pioneering advances in AAV gene therapy, 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.
Latus will share progress toward the development of a novel, investigational, AAV drug candidate that is intended to address somatic instability that drives the CAG repeat expansion in HD. 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.
“As someone who has devoted my career to Huntington’s disease (HD), I am tremendously excited about the advancements we’ve made in developing a gene therapy that targets the somatic instability underlying disease progression. With Latus’ game-changing capsid, AAV-DB-3, we plan to develop a one-time treatment that aims to deliver potentially definitive therapy to the regions of the brain that are most affected in HD,” says Jang-Ho Cha, MD, PhD, CSO/CMO of Latus Bio.
The data presentations are as follows:
Abstract Title: A Computational Model Predicts Treatable Huntington’s Disease Patient Populations and Minimum Therapeutic Efficacy Requirements for Gene Therapies Targeting Somatic Instability
Presenter: Paul T. Ranum, PhD
Day: Wednesday, February 26, 2025
Abstract Title: Targeting MSH3 for Huntington’s Disease: Preclinical Validation of AAV-DB-3-miRNA to Prevent Somatic CAG Repeat Expansion
Presenter: George Yohrling, PhD
Day: Wednesday, February 26, 2025
About Huntington’s Disease
Huntington’s disease is a rare, progressive neurodegenerative disorder caused by genetic mutation in the huntingtin (HTT) gene, leading to the death of nerve cells (neurons) in the brain. Symptoms include motor dysfunction, cognitive decline, as well as psychiatric disturbances that worsen over time. Currently, there is no cure and treatment options are limited to symptom management.
Targeting MSH3 with AAV Gene Therapy
Latus’ investigational AAV gene therapy is being developed to potentially correct the underlying cause of Huntington’s disease (HD). Mutations in the HTT gene, leading to expansion of >39 CAG trinucleotide repeats, cause HD. In some cells in the brain, the CAG repeats continue to expand over decades, resulting in neuronal death. Human genetic studies reveal that MSH3 is a primary driver of CAG repeat expansion. Latus aims to utilize its novel AAV-DB-3 capsid variant to deliver an engineered microRNA to prospectively reduce expression of MSH3 in the cells primarily affected in HD, aiming to slow or to halt HD progression.
About Latus Bio (Latus)
Latus is a biotechnology company dedicated to addressing devastating CNS and peripheral diseases via gene therapy. The Company is advancing an innovative therapeutics pipeline based on novel AAV capsid variants with unprecedented potency, specificity, and tropism. Latus was founded by technology from Professor Beverly Davidson’s lab at the Children’s Hospital of Philadelphia and is powered by a diverse team of visionary scientists, experienced clinicians, and leading industry executives.
For more information, visit www.latusbio.com and follow on LinkedIn.
Contacts
danielle.sliter@latusbio.com
215-995-0890
