Emerging Therapies for Huntington’s Disease: A Review of Promising Treatments

Gene Therapy:
uniQure's AMT-130, a microRNA gene therapy, has shown promising results in slowing disease progression in a Phase I/II trial, with a dose-dependent slowing of disease progression and significant reduction in neurofilament light chain levels3 4.

Antisense Oligonucleotides:
Wave Life Sciences' WVE-003, an allele-specific silencing therapy, has demonstrated significant and selective reduction in mutant HTT protein levels in a Phase Ib/IIa trial, with a 46% drop in mutant HTT levels in patients' cerebrospinal fluid3 4.

Stem Cell Therapy:
Research into stem cell therapies, including the use of induced neural stem cells and cerebral organoids, is ongoing, offering potential for future treatments1.

Disease-Modifying Treatments:
Several therapies, including AMT-130 and WVE-003, are nearing the R&D finish line, aiming to be the first disease-modifying treatments for Huntington's disease4.

Current Treatments:
Current treatments focus on managing symptoms with medicines such as tetrabenazine (Xenazine), deutetrabenazine (Austedo), and valbenazine (Ingrezza) for movement disorders, and antidepressants and antipsychotics for mental health conditions2 5.

Regulatory Progress:
uniQure has scheduled a Type B meeting with the FDA to discuss the potential for an accelerated development pathway for AMT-130, and Wave Life Sciences has initiated engagement with regulators regarding a clinical development path that could support accelerated approval for WVE-0033 4.