Therapeutic targeting of Huntington's disease: Molecular and clinical approaches

Huntington's disease (HD) is an autosomal dominant ailment that affects a larger population. Due to its complex pathology operating at DNA, RNA, and protein levels, it is regarded as a protein-misfolding disease and an expansion repeat disorder. Despite the availability of early genetic diagnostics, disease -modifying treatments are still missing. Importantly, potential therapies are starting to make their way through clinical trials. Still, clinical trials are ongoing to discover potential drugs to relieve HD symptoms. However, now being aware of the root cause, the clinical studies are focused on molecular therapies to target it. The road to success has not been without bumps since a big phase III trial of tominersen was unexpectedly discontinued due to exceeding risks than drug's benefit to the patients. Although the trial's conclusion was disappointing, there is still cause to be optimistic about what this technique may achieve. We have reviewed the present disease-modifying therapies in clinical development for HD and examined the current landscape of developing clinical therapies. We further investigated the pharmaceutical development of Huntington's medicine in the pharma industries and addressed the existing challenges in their therapeutic success. (c) 2023 Elsevier Inc. All rights reserved.

Automated summary

Huntington's disease is a common autosomal dominant disease that affects a large number of patients. Despite early genetic diagnostics, effective disease-modifying treatments are still lacking. Ongoing clinical trials are exploring potential drugs to alleviate symptoms by targeting the root cause at the molecular level. Although a major phase III trial was unexpectedly discontinued, there is still optimism about the potential of this technique.