Huntington's Disease Pipeline 2025: Innovative Clinical Developments By Over 20 Global Leaders Delveinsight Highlighting Hoffmann-La Roche, PTC Therapeutics, Annexon, Alnylam Pharmaceuticals, Neuv

(MENAFN- GetNews)


"Huntington's Disease Pipeline 2025"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.

With Huntington's Disease reaching epidemic levels worldwide and contributing to comorbidities such as diabetes, cardiovascular disease, and certain cancers, the need for safer and more effective treatments is increasing. According to DelveInsight, over 20 pharmaceutical and biotech companies are actively developing more than 20 therapeutic candidates for Huntington's Disease. These therapies are at various stages of clinical and preclinical development, reflecting significant innovation and a strong commitment to tackling this major public health challenge.

DelveInsight's“Huntington's Disease Pipeline Insight 2025” offers a comprehensive strategic assessment of the current R&D landscape. The report examines clinical trial progress, emerging therapies, mechanisms of action, competitive positioning, and key company initiatives. It serves as an essential resource for stakeholders-including researchers, healthcare investors, and decision-makers-seeking insights into the evolving Huntington's Disease therapeutics market and the breakthroughs shaping its future.

Explore the Cutting-Edge Landscape of Huntington's Disease Drug Development

Key Takeaways from the Huntington's Disease Pipeline Report

• DelveInsight's Huntington's Disease pipeline report highlights a vibrant landscape, with over 20 active companies developing more than 20 therapeutic candidates for Huntington's Disease.

• PTC Therapeutics is advancing PTC518, an oral small molecule aimed at lowering production of the mutant huntingtin protein. In September 2024, the FDA granted Fast Track designation to PTC518, recognizing its potential to address unmet needs in HD treatment. In a major move, PTC Therapeutics signed a licensing agreement with Novartis worth up to $2.9 billion for the continued development and commercialization of PTC518, emphasizing the therapy's promise and the industry's commitment to HD innovation.

• In August 2023, the FDA expanded the indication for valbenazine (Ingrezza) to include treatment of chorea associated with Huntington's Disease. This decision was supported by data from the Phase 3 KINECT-HD study and its ongoing open-label extension, KINECT-HD2. Valbenazine is now the only selective vesicular monoamine transporter 2 (VMAT2) inhibitor approved for this condition, providing a new option for managing HD-related movement disorders.

• Key companies in the Huntington's Disease space-such as Hoffmann-La Roche, PTC Therapeutics, Annexon, Alnylam Pharmaceuticals, Neuvivo, and BPG Bio-are actively developing new therapies to enhance the treatment landscape. Promising pipeline candidates include RG6042, PTC518, ALN-HTT02, and several others at various stages of development.

Huntington's Disease Overview:

Huntington's disease is an inherited, progressive neurodegenerative disorder characterized by the gradual emergence of involuntary movements-affecting the limbs, face, and trunk-and a steady decline in cognitive abilities and memory. The disease primarily affects central brain regions responsible for movement, mood, and cognition. Symptoms usually appear between ages 30 and 50, though onset can occur as early as age 2 or as late as 80. Key manifestations include rapid, uncontrollable muscle jerks called chorea, loss of coordination, and changes in personality. As the disease advances, individuals may experience speech difficulties, memory impairments, and increasingly severe involuntary movements.

Huntington's disease is inherited in an autosomal dominant pattern and arises from a mutation in a single gene on chromosome 4. This gene encodes the huntingtin protein, whose exact normal function is not fully understood. The mutated form of huntingtin causes brain changes that result in motor impairments, cognitive decline, and emotional disturbances such as depression and irritability. The mutation involves an abnormal expansion of a DNA sequence within the gene: while a normal gene contains 17–20 repeats, the mutated version associated with Huntington's disease has 40 or more, leading to the development of the condition.

Download the Huntington's Disease sample report to know in detail about the Huntington's Disease treatment market

Huntington's Disease Pipeline Analysis

The Huntington's Disease pipeline insights report 2025, provides insights into:

• Provides comprehensive insights into key companies developing therapies in the Huntington's Disease Market.

• Categorizes Huntington's Disease therapeutic companies by development stage: early, mid, and late-stage.

• Highlights major companies involved in targeted therapy development, including both active and inactive (paused/discontinued) projects.

• Reviews emerging Huntington's Disease drugs under development based on:

  • Stage of development

  • Huntington's Disease Route of administration

  • Target receptor

  • Monotherapy vs. combination therapy

  • Huntington's Disease Mechanism of action

  • Molecular type

• Offers detailed analysis of:

  • Company-to-company and company-academia collaborations

  • Huntington's Disease Licensing agreements

  • Funding and investment activities supporting future Huntington's Disease market advancement.

Unlock key insights into emerging Huntington's Disease therapies and market strategies here:

Huntington's Disease Emerging Drugs

• RG6042: Hoffmann-La Roche

Tominersen (also referred to as ASO-HTT or RG6042) is an antisense oligonucleotide therapy under development for Huntington's disease. It functions by reducing the production of all forms of the huntingtin (HTT) protein, including the mutant variant (mHTT) responsible for the disorder. The therapy is currently in Phase III clinical trials to assess its efficacy in treating Huntington's disease.

• PTC518: PTC Therapeutics

PTC518 is an oral small-molecule therapy aimed at reducing levels of the mutant huntingtin protein, a key driver of neuronal damage and disease progression in Huntington's disease. The compound can cross the blood-brain barrier, acts selectively, allows for dose titration, and is not actively expelled from the brain. It is currently being studied in Phase II clinical trials for the treatment of Huntington's disease.

• ALN-HTT02: Alnylam Pharmaceuticals

ALN-HTT02 is an innovative therapy for Huntington's disease under development by Alnylam Pharmaceuticals. It employs small interfering RNA (siRNA) technology to reduce the production of the huntingtin protein, a key factor in the disease. The therapy specifically targets a portion of the HTT gene's messenger RNA (mRNA), promoting its degradation and preventing the formation of both normal and mutant protein forms. Administered via intrathecal injection, ALN-HTT02 is currently in Phase I clinical trials for Huntington's disease.

Huntington's Disease Pipeline Therapeutic Assessment

Huntington's Disease Assessment by Product Type

. Mono

. Combination

. Mono/Combination

Huntington's Disease By Stage

. 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 Assessment by Route of Administration

. Oral

. Parenteral

. Intravenous

. Subcutaneous

. Topical

Huntington's Disease Assessment by Molecule Type

. Recombinant fusion proteins

. Small molecule

. Monoclonal antibody

. Peptide

. Polymer

. Gene therapy

Download sample pages to get an in-depth assessment of the emerging Huntington's Disease therapies and key Huntington's Disease companies

Table of Contents

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

Request the sample PDF to get detailed insights about the Huntington's Disease pipeline reports offerings

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.

MENAFN24092025003238003268ID1110107677