At Latus, we are advancing a one-time gene therapy that aims to correct Huntington's disease at the start, promising to slow or even halt disease progression.
HD is caused by mutations in the huntingtin (HTT) gene leading to >39 CAG expansions. Human genetic data reveal that over time the CAG expansions grow to >150 CAGs, which causes neuronal death and leads to HD symptoms. The expansion is caused by a protein called MSH3. At Latus, we aim to reduce MSH3 expression to target HD at it's start.
Huntington’s disease is fatal within ~10-20 years of symptom onset
Progressive onset of additional symptoms at typically 2 years after diagnosis
We have unveiled a novel computational model 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.
Our preclinical data disclosures support deep MSH3 knockdown with our investigational gene therapy, yielding reductions in somatic instability in a mouse model of HD.
“I really hope we can find a way to slow down this disease. No parent wants to see their kid go through endless hospital visits, the pain of treatments, or spend their days in a hospital bed. We just want more happy, normal days at home, more laughter, and less tears—more time being a family without the constant stress of medical issues.”
“Huntngton's Disease remains a devastating, incurable genetic brain disease, for which we desperately need effective interventions. A breakthrough would be transformative for affected patients and families worldwide.”
