The Huntington's Disease pipeline report provides detailed information of products from the initial phase of development until its commercialization market.

Huntington's Disease Pipeline Insight, Pipeline Drugs 

DelveInsight&rsquo;s, &ldquo;Huntington&rsquo;s Disease - Pipeline Insight, 2025&rdquo; report provides comprehensive insights about 22+ companies and 25+ pipeline drugs in Huntington&rsquo;s Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
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<h2>Geography Covered</h2>
<ul>
<li>Global coverage</li>
</ul>
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<h2>Huntington&rsquo;s Disease: Understanding</h2>
Huntington&rsquo;s Disease: Overview
Huntington's disease (HD) is a neurodegenerative disorder caused by a genetic mutation in the Huntingtin (HTT) gene, leading to the expansion of CAG repeats on chromosome 4. This results in abnormal polyglutamine stretches in the HTT protein, causing brain cell degeneration. HD typically manifests in adults between the ages of 30 and 50, with symptoms including involuntary movements (chorea), cognitive decline, and behavioral changes. Juvenile HD, which occurs before age 20, presents with learning difficulties and behavioral issues. As the disease progresses, patients become increasingly dependent on caregivers, with common complications including pneumonia and suicide. Genetic testing can confirm the diagnosis, and pre-manifest testing is available for those at risk. While there is no cure, symptomatic treatments aim to improve quality of life and manage motor, cognitive, and psychiatric symptoms. Research into potential therapies continues, but effective disease-modifying treatments remain elusive.
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The HTT gene encodes the HTT protein, which is involved in synaptic function and neuroprotection. The mutation leads to toxic aggregates of the mutant HTT protein, resulting in brain atrophy, particularly in the striatum, and neuronal loss. The length of CAG repeats in the HTT gene correlates with the age of onset, with longer repeats leading to earlier symptom development, especially in cases of juvenile-onset HD. This condition exhibits anticipation, where the disease manifests at a younger age in successive generations, especially when inherited from the father due to repeat instability during spermatogenesis. While alleles with 27-35 repeats do not cause symptoms, they are prone to instability and potential expansion in future generations.
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HD is characterized by the degeneration of neurons in the putamen, caudate, and cerebral cortex, leading to motor and cognitive symptoms. The preferential loss of enkephalin-containing neurons in the basal ganglia's indirect pathway results in chorea, while the loss of substance-P-containing neurons in the direct pathway contributes to dystonia and akinesia. The specific regions affected in the cortex and basal ganglia explain the variability in symptoms. Several mechanisms contribute to HD's pathogenesis, including neuronal aggregates formed by mutant HTT proteins, impairing the proteolytic pathway, transcriptional dysregulation, excitotoxicity due to increased glutamate, mitochondrial dysfunction, and disturbances in axonal transport and synaptic function. These processes likely occur simultaneously, exacerbating neurodegeneration.
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There is no cure for Huntington's disease (HD), but various therapeutic options aim to manage symptoms and improve quality of life. Treatment primarily involves pharmacological approaches, including medications for chorea, psychiatric disturbances, and parkinsonism, along with supportive care. Surgical options are limited, but deep brain stimulation has been explored for managing motor symptoms. For chorea, tetrabenazine (TBZ), amantadine, and riluzole are commonly used, with TBZ being the most recommended. Medications such as atypical neuroleptics (e.g., olanzapine, quetiapine) may help with motor and psychiatric symptoms, although side effects like akathisia must be monitored. For parkinsonism, levodopa and dopamine agonists are considered. Behavioral and psychiatric symptoms, including depression and aggression, are managed with antidepressants and other therapies. Non-medical interventions like supportive care, dietary management, and emotional support are crucial for patient well-being. Gene therapy, aiming to silence the mutant gene, offers promising potential for future treatment, though further research is needed.
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"Huntington&rsquo;s Disease- Pipeline Insight, 2025" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Huntington&rsquo;s Disease pipeline landscape is provided which includes the disease overview and Huntington&rsquo;s Disease treatment guidelines. The assessment part of the report embraces, in depth Huntington&rsquo;s Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Huntington&rsquo;s Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
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<h3>Report Highlights</h3>
<ul>
<li>The companies and academics are working to assess challenges and seek opportunities that could influence Huntington&rsquo;s Disease R&amp;D. The therapies under development are focused on novel approaches to treat/improve Huntington&rsquo;s Disease.</li>
</ul>
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<h2>Huntington&rsquo;s Disease Emerging Drugs Chapters</h2>
This segment of the Huntington&rsquo;s Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, Preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
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Huntington&rsquo;s Disease Emerging Drugs
<ul>
<li>Pridopidine: Prilenia Therapeutics</li>
</ul>
Pridopidine (45 mg twice daily) is an oral, highly selective and potent investigational S1R agonist that has exhibited a safety and tolerability profile similar to placebo in clinical studies to date. The S1R protein is highly expressed in the brain and spinal cord, regulating several key processes that are commonly impaired in HD and other neurodegenerative diseases. Activation of the S1R by pridopidine stimulates multiple cellular protective pathways, including enhancing autophagy, axonal transport, mitochondrial energy production and respiration, and restores calcium homeostasis. These effects are essential to neuronal function and survival, and lead to neuroprotective effects. Prilenia holds Orphan Drug designation for pridopidine in HD in the US and EU. In addition, pridopidine has received Fast Track designation by the US Food and Drug Administration (FDA) for the treatment of HD. Currently, the drug is Registration stage of its development for the treatment of Huntington&rsquo;s Disease.
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<ul>
<li>ANX005: Annexon, Inc.</li>
</ul>
ANX005, is a first-of-its kind selective, targeted and rapid-acting Monoclonal antibody designed to reduce inflammation and nerve damage by stopping C1q activity in the peripheral and central nervous systems. ANX005 is administered intravenously and has been observed to act almost immediately in blocking C1q function. ANX005 selectively suppresses C1q, thereby preventing classical complement activation. In doing so, it is thought to protect against synaptic loss and slow neurodegeneration in Huntington&rsquo;s and other neurodegenerative conditions. ANX005 has also received Orphan Drug Designation in the US for Huntington&rsquo;s. Currently, the drug is in Phase II stage of its development for the treatment of Huntington&rsquo;s Disease.
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<ul>
<li>AMT-130: UniQure Biopharma B.V.</li>
</ul>
AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging UniQure&rsquo;s proprietary miQURE&trade; silencing technology. The therapeutic goal is to inhibit the production of the mutant protein (mHTT). Using AAV vectors to deliver micro-RNAs directly to the brain for non-selective knockdown of the huntingtin gene represents a highly innovative and promising approach to treating Huntington&rsquo;s disease. AMT-130 targets the deep brain structures known for the disease pathology onset. It also silences mutant huntingtin protein at levels not demonstrated in other studies. AMT-130 targets the accumulation of the exon 1 HTT fragment, the most toxic source of abnormal protein aggregation in Huntington&rsquo;s disease. AMT-130 has also received the Regenerative Medicine Advanced Therapy designation by the US FDA. Currently, the drug is in Phase I/II stage of its development for the treatment of Huntington&rsquo;s Disease.
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<ul>
<li>ALN-HTT02: Alnylam Pharmaceuticals</li>
</ul>
ALN-HTT02 works through a RNA interference. RNAi is unique because it takes advantage of molecular machinery that already exists inside cells for processing. ALN-HTT02 itself is a piece of synthetic genetic material that contains a code to recognize HTT. Once it&rsquo;s introduced into a cell, the cell&rsquo;s own molecular machines are used to process the synthetic genetic material. This creates a fragment of the synthetic genetic material that binds to the HTT message to lower it. ALN-HTT02 targets the first bit of the HTT gene called &ldquo;exon 1&rdquo;. This is the part of the genetic message that contains the spelling error that causes HD. Currently, the drug is in Phase I stage of its development for the treatment of Huntington&rsquo;s Disease.
Further product details are provided in the report&hellip;&hellip;..
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Huntington&rsquo;s Disease: Therapeutic Assessment
This segment of the report provides insights about the different Huntington&rsquo;s Disease drugs segregated based on following parameters that define the scope of the report, such as:
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Major&nbsp; Players in Huntington&rsquo;s Disease
There are approx. 22+ key companies which are developing the therapies for Huntington&rsquo;s Disease. The companies which have their Huntington&rsquo;s Disease drug candidates in the most advanced stage, i.e. Registration include, Prilenia Therapeutics.
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Phases
DelveInsight&rsquo;s report covers around 25+ products under different phases of clinical development like
<ul>
<li>Late stage products (Phase III)</li>
<li>Mid-stage products (Phase II)</li>
<li>Early-stage product (Phase I) along with the details of&nbsp;</li>
<li>Pre-clinical and Discovery stage candidates</li>
<li>Discontinued &amp; Inactive candidates</li>
</ul>
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Route of Administration
Huntington&rsquo;s Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as&nbsp;
<ul>
<li>Oral</li>
<li>Intravenous</li>
<li>Subcutaneous</li>
<li>Parenteral&nbsp;</li>
<li>Topical</li>
</ul>
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Molecule Type
Products have been categorized under various Molecule types such as
<ul>
<li>Recombinant fusion proteins</li>
<li>Small molecule</li>
<li>Monoclonal antibody</li>
<li>Peptide</li>
<li>Polymer&nbsp;</li>
<li>Gene therapy</li>
</ul>
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Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
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<h2>Huntington&rsquo;s Disease: Pipeline Development Activities&nbsp;</h2>
The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Huntington&rsquo;s Disease therapeutic drugs key players involved in developing key drugs.&nbsp;
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Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Huntington&rsquo;s Disease drugs.
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Huntington&rsquo;s Disease Report Insights
<ul>
<li>Huntington&rsquo;s Disease Pipeline Analysis</li>
<li>Therapeutic Assessment</li>
<li>Unmet Needs</li>
<li>Impact of Drugs</li>
</ul>
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Huntington&rsquo;s Disease Report Assessment
<ul>
<li>Pipeline Product Profiles</li>
<li>Therapeutic Assessment</li>
<li>Pipeline Assessment</li>
<li>Inactive drugs assessment</li>
<li>Unmet Needs</li>
</ul>
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<h2>Key Questions</h2>
Current Treatment Scenario and Emerging Therapies:
<ul>
<li>How many companies are developing Huntington&rsquo;s Disease drugs?</li>
<li>How many Huntington&rsquo;s Disease drugs are developed by each company?</li>
<li>How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Huntington&rsquo;s Disease?</li>
<li>What are the key collaborations (Industry&ndash;Industry, Industry&ndash;Academia), Mergers and acquisitions, licensing activities related to the Huntington&rsquo;s Disease therapeutics?&nbsp;</li>
<li>What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?&nbsp;</li>
<li>What are the clinical studies going on for Huntington&rsquo;s Disease and their status?</li>
<li>What are the key designations that have been granted to the emerging drugs?</li>
</ul>

Introduction
Executive Summary
Huntington&rsquo;s Disease: Overview
<ul>
<li>Introduction</li>
<li>Causes</li>
<li>Pathophysiology&nbsp;</li>
<li>Signs and Symptoms</li>
<li>Diagnosis</li>
<li>Treatment</li>
</ul>
Pipeline Therapeutics
<ul>
<li>Comparative Analysis</li>
</ul>
Therapeutic Assessment
<ul>
<li>Assessment by Product Type</li>
<li>Assessment by Stage and Product Type</li>
<li>Assessment by Route of Administration</li>
<li>Assessment by Stage and Route of Administration</li>
<li>Assessment by Molecule Type</li>
<li>Assessment by Stage and Molecule Type&nbsp;</li>
</ul>
Huntington&rsquo;s Disease&ndash; DelveInsight&rsquo;s Analytical Perspective
Late Stage Products (Registration)
<ul>
<li>Comparative Analysis</li>
</ul>
Pridopidine: Prilenia Therapeutics
<ul>
<li>Product Description</li>
<li>Research and Development&nbsp;</li>
<li>Product Development Activities&nbsp;</li>
</ul>
Drug profiles in the detailed report&hellip;..
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Mid Stage Products (Phase II)
<ul>
<li>Comparative Analysis</li>
</ul>
ANX005: Annexon, Inc.
<ul>
<li>Product Description</li>
<li>Research and Development&nbsp;</li>
<li>Product Development Activities&nbsp;</li>
</ul>
Drug profiles in the detailed report&hellip;..
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Early Stage Products (Phase I)&nbsp;
<ul>
<li>Comparative Analysis</li>
</ul>
&nbsp;ALN-HTT02: Alnylam Pharmaceuticals
<ul>
<li>Product Description</li>
<li>Research and Development&nbsp;</li>
<li>Product Development Activities&nbsp;</li>
</ul>
Drug profiles in the detailed report&hellip;..
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Preclinical and Discovery Stage Products&nbsp;
<ul>
<li>Comparative Analysis</li>
</ul>
Drug Name: Company Name
<ul>
<li>Product Description</li>
<li>Research and Development&nbsp;</li>
<li>Product Development Activities&nbsp;</li>
</ul>
Drug profiles in the detailed report&hellip;..
&nbsp;
Inactive Products
<ul>
<li>Comparative Analysis</li>
</ul>
Huntington&rsquo;s Disease Key Companies
Huntington&rsquo;s Disease Key Products
Huntington&rsquo;s Disease- Unmet Needs
Huntington&rsquo;s Disease- Market Drivers and Barriers
Huntington&rsquo;s Disease- Future Perspectives and Conclusion
Huntington&rsquo;s Disease Analyst Views
Huntington&rsquo;s Disease Key Companies
Appendix

DelveInsight’s, “Huntington’s Disease - Pipeline Insight, 2025,” report provides comprehensive insights about 20+ companies and 25+ pipeline drugs in Huntington’s Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Huntington’s Disease Pipeline

Huntington’s Disease - Pipeline Insight, 2025

Huntington's Disease Pipeline Insights, 2025 report by DelveInsight outlays comprehensive insights of present clinical development scenario and growth prospects across the  market.