A Phase 1/2 clinical trial conducted by biotech company uniQure is offering new hope for patients with Huntington’s disease, a devastating neurodegenerative disorder. Huntington’s disease is caused by a genetic mutation that leads to cognitive and physical symptoms starting in middle age. Currently, there is no treatment that can stop or reverse the progression of the disease.
The trial, which is the first for a one-time gene therapy for Huntington’s disease, aims to target the genetic source of the disease. uniQure’s drug, known as AMT-130, is a one-time infusion into the brain that contains a microRNA (miRNA) designed to target the mutant huntingtin gene. The miRNA directs cellular machinery to reduce the amount of mutant protein accumulating in the neurons, with the goal of preserving cognitive and motor function.
Unlike previous attempts to target mutant huntingtin, uniQure’s gene therapy takes a different approach by using miRNA. This approach has the potential to be more effective in reducing the production of the disruptive mutant huntingtin protein. Additionally, AMT-130 is a one-time infusion, which means that patients would only need to undergo the procedure once in their lifetime.
The clinical trial is currently in progress, with interim data showing promising results. However, there is still a long road ahead to determine if the drug has a lasting benefit. If successful, AMT-130 could potentially be the first treatment for Huntington’s disease.
While the current trial is focused on early-stage Huntington’s disease, researchers believe that it could lay the groundwork for future treatments in later stages of the disease. By targeting the core pathogenic mechanisms in early-stage patients, researchers hope to determine if meaningful deflection or even reversal of the disease’s progression is possible.
The trial is still relatively small, but the researchers are hopeful that enrolling more patients will provide a clearer picture of AMT-130’s safety and effectiveness. The ultimate goal is to provide a new treatment option for the thousands of patients and their families who are affected by Huntington’s disease.
Sources:
– Original article: Retrieved from [source]
– Definition of Huntington’s disease: Retrieved from [source]
– Definition of gene therapy: Retrieved from [source]
– Definition of microRNA (miRNA): Retrieved from [source]
