Health & Safety Industry Today

Global Huntington’s Disease Market is Expanding in Gene and RNA-Based Therapies – BIS Research

Huntington’s disease (HD) is a rare, inherited neurodegenerative disorder caused by a mutation in the HTT gene, leading to progressive breakdown of nerve cells in the brain. HD is characterized by motor dysfunction, cognitive decline, and psychiatric symptoms. The disease typically manifests in mid-adulthood and gradually worsens, causing significant disability and, in many cases, death within 10 to 30 years of diagnosis. Market growth is driven by advancements in genetic testing, novel therapies, and increasing patient awareness.
Published 23 October 2025

What is Huntington’s Disease? 

Huntington’s disease is confirmed through genetic testing and currently has no cure. Treatments are focused on symptom management, including motor symptoms, cognitive decline, and psychiatric complications. Recent innovations, such as gene therapies and antisense oligonucleotides, are designed to target the underlying genetic cause, offering potential disease-modifying benefits. 

What is the Current Market Outlook for Huntington’s Disease? 

The global huntington’s disease market is expanding due to increasing prevalence, improved diagnostics, and the development of targeted treatments. Gene therapies, RNA-based therapies, and neuroprotective agents are entering clinical development, while symptomatic treatments such as tetrabenazine and deutetrabenazine remain widely used. Early detection through advanced genetic testing and imaging supports better patient outcomes, further driving demand for specialized therapies. 

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What is the key Innovation & Trends in Huntington’s Disease Market? 


  • Gene Therapies: Treatments like AMT-130 aim to reduce the mutant huntingtin gene expression, addressing the root cause of HD. 
  • RNA-Based Therapies: Antisense oligonucleotides such as tominersen are being developed to lower production of the mutant protein. 
  • Symptomatic Treatments: Tetrabenazine and deutetrabenazine are used to manage chorea, while antidepressants and antipsychotics address psychiatric symptoms. 
  • Neuroprotective Agents: Investigational therapies focus on slowing neurodegeneration and improving patient quality of life. 


How Does This Report Help Organizations Drive Strategic Growth? 


  • Benchmark Offerings: Track gene therapies, RNA-based treatments, and symptomatic drug options. 
  • Monitor Innovations: Focus on clinical trials, biomarker development, and gene-silencing research. 
  • Identify Opportunities: Leverage unmet needs in rare neurodegenerative disorders and emerging patient populations. 
  • Evaluate Competition: Assess strategies for leading players such as Prilenia Therapeutics B.V., Novartis AG, and Bausch Health Companies Inc. 


What Are the Demand Driver and Challenges in Huntington’s Disease Market? 

Drivers

  • Rising prevalence of Huntington’s disease 
  • Advancements in genetic testing and imaging for early diagnosis 
  • Approval of gene therapies and disease-modifying treatments 
  • Increased awareness among patients and healthcare professionals 

Challenges

  • High cost of novel therapies limiting access, particularly in low- and middle-income regions 
  • Limited awareness and expertise leading to delayed diagnosis 
  • Absence of universally accepted treatment protocols 
  • Regulatory hurdles for approval of innovative gene- and RNA-based therapies


What is the Market Segmentation in Huntington’s Disease Market? 

By Drug Class 

o    Antidepressants 

o    Antipsychotic Drugs 

o    Deutetrabenazine 

o    Deutetrabenazine 

o    RNA-based Therapy 

o    Tetrabenazine 

By the Region 

o    North America 

o    Europe   

What Is the Competitive Landscape in Huntington’s Disease Market? 

Strategic Initiatives 

  • Prilenia Therapeutics B.V., Novartis AG, and Bausch Health Companies Inc. are leading R&D in gene- and RNA-based therapies. 
  • Emerging biotech companies are developing next-generation gene-silencing and neuroprotective treatments. 
  • Collaborations between pharmaceutical companies, research institutions, and healthcare providers focus on advancing personalized medicine and improving patient outcomes. 

Here Are Some Case Studies and Success Stories in Huntington’s Disease Market 

  • AMT-130 Gene Therapy: Clinical trials demonstrate potential in reducing mutant huntingtin protein and slowing disease progression. 
  • Tominersen Antisense Therapy: RNA-based therapy under investigation for lowering mutant protein production and modifying disease courses. 


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