A biotechnology firm working to correct an underlying cause of Huntington’s disease will announce positive data this week on its ongoing gene therapy development.
Latus Bio aims to address mutations in the HTT gene which lead to expansion of >39 CAG trinucleotide repeats which cause Huntington’s. A CAG repeat is a sequence of DNA in which the three nucleotides “cytosine, adenine, and guanine” are repeated multiple times in a row within a gene.
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 utilise its engineered microRNA to prospectively reduce expression of MSH3 in the cells primarily affected in HD, aiming to slow or to halt HD progression.
The gene therapy is delivered via adeno-associated virus (AAV), a small, non-pathogenic virus that is widely used as a vector to deliver genetic material (genes) into cells.
This week at the annual CHDI Huntington’s Disease (HD) Therapeutics Conference in California Latus will announce data presentations in support of its preclinical drug candidate.
It will focus on a novel computational model 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 its novel AAV-DB-3 treatment, 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.
Jang-Ho Cha, CMO of Latus Bio, says: “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.”
Latus was founded by technology from Professor Beverly Davidson’s lab at the Children’s Hospital of Philadelphia.
