Huntington's Disease Clinical, Companies, Therapeutic Assessment, Therapies, Treatment Algorithm, Pipeline Analysis | Hoffmann-La Roche, PTC Therapeutics, Annexon, Alnylam Pharmaceuticals, Neuvivo, BPG Bio

Huntington's Disease Pipeline constitutes 20+ key companies continuously working towards developing 20+ Huntington's Disease treatment therapies, analyzes DelveInsight.

Huntington's Disease Overview:

Huntington's disease is a hereditary, progressive neurological disorder marked by the gradual onset of involuntary movements in the hands, feet, face, and torso, along with a steady decline in cognitive abilities and memory, ultimately leading to dementia. It results from damage to specific areas in the brain that control movement, mood, and cognitive functions. Symptoms typically emerge between the ages of 30 and 50 but can start as early as 2 or as late as 80. A hallmark of the disease is rapid, involuntary muscle movements such as jerks or tics, known as chorea. Over time, individuals may experience difficulty with coordination, personality shifts, speech impairments, memory loss, and worsening chorea.

Huntington's disease follows an autosomal dominant inheritance pattern, meaning only one copy of the defective gene is needed to cause the disorder. It is linked to a mutation in a gene on chromosome 4, which encodes a protein known as huntingtin. While the exact function of the normal huntingtin protein remains unclear, its mutated form is known to cause the disease. This mutation involves an abnormal repetition of a specific DNA sequence. Normally, the huntingtin gene contains 17 to 20 repeats of this sequence, but in people with Huntington's disease, the gene has 40 or more. This excessive repetition leads to the production of a toxic form of the protein, resulting in brain cell damage, uncontrolled movements, cognitive decline, and mood 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

DelveInsight's Huntington's Disease pipeline report depicts a robust space with 20+ active players working to develop 20+ pipeline therapies for Huntington's Disease treatment.
Breakthrough Therapy designation was granted by the FDA in April 2025 based on interim Phase I/II data showing significant slowing of disease progression (~80% reduction at high dose on the composite Unified Huntington's Disease Rating Scale, cUHDRS), along with supportive biomarker changes (lower neurofilament light chain levels) from treated patients versus external natural-history controls
Key Huntington's Disease companies such as Hoffmann-La Roche, PTC Therapeutics, Annexon, Alnylam Pharmaceuticals, Neuvivo, BPG Bio, and others are evaluating new drugs for Huntington's Disease to improve the treatment landscape.
Promising Huntington's Disease pipeline therapies in various stages of development include RG6042, PTC518, ALN-HTT02, and others.

Huntington's Disease Pipeline Analysis
The report provides insights into:
The report provides detailed insights into the key companies that are developing therapies in the Huntington's Disease Market.
The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Huntington's Disease treatment.
It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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.
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.

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Huntington's Disease Emerging Drugs

RG6042: Hoffmann-La Roche

Tominersen (also known as ASO-HTT or RG6042) is an antisense therapy being developed as a potential treatment for Huntington's disease. Its primary goal is to lower the levels of the huntingtin (HTT) protein, including both the normal and mutated forms (mHTT), the latter of which is the cause of the disease. The drug is currently undergoing Phase III clinical trials to evaluate its effectiveness and safety in treating Huntington's disease.

PTC518: PTC Therapeutics

PTC518 is an orally administered small molecule designed to decrease the production of the mutated huntingtin protein, which contributes to neuronal damage and disease progression in Huntington's disease. This compound is able to cross the blood-brain barrier, is selective in its action, allows for dose adjustment (titratable), and is not expelled from the brain by efflux mechanisms. It is currently in Phase II clinical trials for the treatment of Huntington's disease.

Huntington's Disease Companies

There are approx. 20+ key companies which are developing the therapies for Huntington's disease. The companies which have their Huntington's Disease drug candidates in the most advanced stage, i.e. Phase III include Hoffmann-La Roche.

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

Late stage products (Phase III)
Mid-stage products (Phase II)
Early-stage product (Phase I) along with the details of
Pre-clinical and Discovery stage candidates
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

Intravenous
Subcutaneous
Oral
Intramuscular

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

Monoclonal antibody
Small molecule
Peptide

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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

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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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Contact Info:
Kritika Rehani
krehani@delveinsight.com
info@delveinsight.com
+14699457679

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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