Huntington's Disease | Clinical Landscape, Key Companies, Therapeutic Assessment, Treatment Algorithms, and Pipeline Insights - featuring leading players such as Hoffmann-La Roche, PTC Therapeutics, A

DelveInsight's, "Huntington's Disease - Pipeline Insight, 2025" report provides comprehensive insights about 20+ companies and 20+ 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.
According to DelveInsight's analysis, the Huntington's Disease pipeline features more than 20 leading companies actively engaged in developing over 20 therapeutic candidates aimed at treating Huntington's Disease.

Huntington's Disease Overview:

Huntington's disease is an inherited, progressive neurodegenerative disorder characterized by the gradual development of involuntary movements in the hands, feet, face, and trunk, along with a continuous decline in cognitive and memory functions that eventually leads to dementia. The condition stems from damage to specific brain regions responsible for movement, mood regulation, and cognition. Symptoms usually appear between the ages of 30 and 50 but can manifest as early as 2 or as late as 80. A key feature of the disease is chorea-sudden, uncontrollable muscle movements such as jerks or twitches. As the disease progresses, patients may experience worsening coordination, personality and mood changes, speech difficulties, memory impairment, and intensified involuntary movements.

Huntington's disease follows an autosomal dominant inheritance pattern, meaning that a single copy of the defective gene can cause the condition. It is associated with a mutation in the huntingtin gene located on chromosome 4. Although the precise role of the normal huntingtin protein is not fully understood, the mutated version is toxic to brain cells. This mutation involves an abnormal expansion of a DNA sequence (CAG repeat). While healthy individuals have 17-20 repeats, those with Huntington's disease typically possess 40 or more, leading to the formation of a harmful huntingtin protein that disrupts neuronal function and contributes to movement abnormalities, cognitive decline, and emotional disturbances such as depression and irritability.

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"Huntington's Disease Pipeline Insight 2025" report by DelveInsight provides a comprehensive analysis of the ongoing clinical development activities and growth prospects across the Huntington's Disease Therapeutics Market.

Key Takeaways from the Huntington's Disease Pipeline Report

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DelveInsight's Huntington's Disease pipeline report highlights a dynamic landscape, with over 20 active companies developing more than 20 investigational therapies for the treatment of Huntington's Disease.

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In April 2025, the FDA granted Breakthrough Therapy designation based on interim Phase I/II results demonstrating a substantial reduction in disease progression-approximately 80% at higher doses-on the composite Unified Huntington's Disease Rating Scale (cUHDRS). These findings were further supported by favorable biomarker data, including decreased neurofilament light chain levels in treated patients compared to external natural-history controls.

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Leading companies such as Hoffmann-La Roche, PTC Therapeutics, Annexon, Alnylam Pharmaceuticals, Neuvivo, and BPG Bio are actively advancing novel therapeutic approaches aimed at transforming the Huntington's Disease treatment landscape. Notable pipeline candidates currently in various stages of development include RG6042, PTC518, ALN-HTT02, and others.

Huntington's Disease Pipeline Analysis

The report provides insights into:

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The report provides detailed insights into the key companies that are developing therapies in the Huntington's Disease Market.

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The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Huntington's Disease treatment.

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It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

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It navigates the emerging drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.

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Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement, and financing details for future advancement of the Huntington's Disease market.

Download our free sample page report on Huntington's Disease pipeline insights [https://www.delveinsight.com/sample-request/huntingtons-disease-pipeline-insight?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr]

Huntington's Disease Emerging Drugs

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RG6042: Hoffmann-La Roche

Tominersen (also known as ASO-HTT or RG6042) is an antisense oligonucleotide therapy in development for the treatment of Huntington's disease. It is designed to reduce the production of the huntingtin (HTT) protein-both the normal and the mutant forms (mHTT), the latter being responsible for disease onset and progression. The therapy is currently in Phase III clinical trials to assess its efficacy and safety in patients with Huntington's disease.

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PTC518: PTC Therapeutics

PTC518 is an orally available small molecule developed to reduce the production of the mutant huntingtin protein, which drives neuronal degeneration and disease progression in Huntington's disease. The drug is capable of crossing the blood-brain barrier, exhibits selective activity, offers dose flexibility (titratable), and is not subject to efflux from the brain. PTC518 is currently being evaluated in Phase II clinical trials for the treatment of Huntington's disease.

Huntington's Disease Companies

Around 20 or more major companies are actively engaged in developing therapies for Huntington's disease. Among them, Hoffmann-La Roche has a drug candidate currently in the most advanced phase of development-Phase III.

DelveInsight's report covers around 75+ products under different phases of clinical development like

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Late stage products (Phase III)

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Mid-stage products (Phase II)

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Early-stage product (Phase I) along with the details of

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Pre-clinical and Discovery stage candidates

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Discontinued & Inactive candidates

Huntington'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

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Intravenous

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Subcutaneous

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Oral

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Intramuscular

Huntington's Disease Products have been categorized under various Molecule types such as

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Monoclonal antibody

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Small molecule

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Peptide

Download Sample Pages to Get an in-depth Assessment of the Emerging Huntington's Disease Therapies and Key Companies: Huntington's Disease Clinical Trials and advancements [https://www.delveinsight.com/report-store/huntingtons-disease-pipeline-insight?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr]

Huntington's Disease Pipeline Therapeutic Assessment

- Huntington's Disease Assessment by Product Type

- Huntington's Disease By Stage

- Huntington's Disease Assessment by Route of Administration

- Huntington's Disease Assessment by Molecule Type

Download Huntington's Disease Sample report to know in detail about the Huntington's Disease treatment market @ Huntington's Disease Therapeutic Assessment [https://www.delveinsight.com/sample-request/huntingtons-disease-pipeline-insight?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr]

Table of Content

1. Report Introduction

2. Executive Summary

3. Huntington's Disease Current Treatment Patterns

4. Huntington's Disease - DelveInsight's Analytical Perspective

5. Therapeutic Assessment

6. Huntington's Disease Late-Stage Products (Phase-III)

7. Huntington's Disease Mid-Stage Products (Phase-II)

8. Early Stage Products (Phase-I)

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Huntington's Disease Discontinued Products

13. Huntington's Disease Product Profiles

14. Huntington's Disease Key Companies

15. Huntington's Disease Key Products

16. Dormant and Discontinued Products

17. Huntington's Disease Unmet Needs

18. Huntington's Disease Future Perspectives

19. Huntington's Disease Analyst Review

20. Appendix

21. Report Methodology

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About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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