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The Huntington’s Disease Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.

“Huntington’s Disease Pipeline Insight, 2026“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Huntington’s Disease Market.

Some of the key takeaways from the Huntington’s Disease Pipeline Report:

• Companies across the globe are diligently working toward developing novel Huntington’s Disease treatment therapies with a considerable amount of success over the years.

• Huntington’s Disease companies working in the treatment market are Teva Pharmaceutical, Annexon Biosciences, Prilenia Therapeutics, Neurocrine Biosciences, Som Biotech, Annexon Biosciences, Vaccinex, Sage Therapeutics, UniQure Biopharma, Roche, Ionis Pharmaceuticals, Neurocrine Biosciences, Vaccinex, Azevan Pharmaceuticals, WaVe life Sciences, Luye Pharma Group, SOLA Biosciences, and others, are developing therapies for the Huntington’s Disease treatment

• Emerging Huntington’s Disease therapies in the different phases of clinical trials are- AUSTEDO (deutetrabenazine/SD-809), ANX005, Pridopidine(ACR-16; Huntexil), Ingrezza (valbenazine), SOM3355(bevantolol), ANX005, VX15/2503 (pepinemab), SAGE-718, AMT-130/ intrastriatal rAAV5-miHTT, RO7234292, Tominersen, Valbenazine, Pepinemab, SRX246, WVE-003, LPM3770164, SOL-175, and others are expected to have a significant impact on the Huntington’s Disease market in the coming years.

• In November 2026, Skyhawk Therapeutics announced that the first patients in the United States had received doses in the pivotal FALCON-HD study evaluating SKY-0515. This milestone expanded the clinical development programme as the company continued investigating the therapy’s safety and potential disease-modifying effects.

• In November 2026, uniQure announced that the FDA had accepted its BLA for ifezuntirgene inilparvovec (AMT-130) and granted Priority Review under the Accelerated Approval pathway. The FDA established a PDUFA target action date of May 1, 2027. If approved, the therapy could become the first disease-modifying treatment for Huntington’s Disease.

• In October 2026, Alnylam Pharmaceuticals announced positive initial Phase Ib results for brezosiran (ALN-HTT02), an investigational RNA interference (RNAi) therapy for Huntington’s Disease. The treatment demonstrated a mean reduction of approximately 60–64% in cerebrospinal fluid mutant huntingtin levels at three months, with individual reductions reaching up to 83%. The company reported encouraging safety and tolerability findings and planned a placebo-controlled pivotal study in 2027, subject to regulatory alignment.

• In October 2026, Prilenia and Ferrer announced authorization to initiate the European component of the confirmatory Phase III PRECISE-HD study. Recruitment was expected to begin across more than 31 sites in nine European countries, complementing ongoing enrolment in the United States and United Kingdom.

• In September 2026, UniQure has submitted a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) seeking approval for AMT-130, its investigational gene therapy for Huntington’s disease. The company has also filed for regulatory approval in the United Kingdom. The submissions follow months of uncertainty surrounding the therapy’s regulatory pathway, including shifts in the FDA’s position. If the FDA grants priority review, a decision on AMT-130 could potentially be reached by the second quarter of 2027.

• In April 2026, uniQure N.V., a gene therapy company focused on developing transformative treatments for patients with serious medical conditions, announced that it recently conducted a Pre-Submission Meeting with the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA). The company also revealed plans to file a Marketing Authorization Application (MAA) for AMT-130 as a treatment for Huntington’s disease in the third quarter of 2026.

• In March 2026, uniQure announced that it has received the official meeting minutes from the U.S. Food and Drug Administration (FDA) following a Type A meeting held on January 30, 2026, to discuss AMT-130, its investigational gene therapy for Huntington’s disease. The company stated that the FDA indicated the current data are not sufficient to support approval of AMT-130, which comes as disappointing news for the Huntington’s disease community.

• In January 2026, Skyhawk Therapeutics, Inc., a clinical-stage biotech company focused on small-molecule therapies that target RNA, announced encouraging results from a nine-month interim analysis of its investigational Huntington’s disease treatment SKY-0515. The therapy achieved dose-dependent reductions in mutant huntingtin (mHTT) protein, reaching 62% at the 9 mg dose, along with a 26% dose-dependent decrease in PMS1 mRNA—a key contributor to somatic CAG repeat expansion and Huntington’s disease progression. SKY-0515 also showed strong central nervous system exposure and was generally safe and well tolerated in the study.

• In November 2025, The approval prospects for uniQure’s gene therapy candidate for Huntington’s disease are uncertain after the US FDA questioned the sufficiency of the company’s Phase I/II trial data, causing the stock to drop nearly 66% at market open on November 3. The single-use gene therapy, AMT-130, had been positioned to become the first approved treatment for Huntington’s disease following positive mid-stage trial results, which had previously driven uniQure’s stock up 247% in late September. Recent discussions with the FDA indicated that the Phase I/II data would not be adequate for a biologics license application (BLA), a “drastic change” from prior guidance provided in multiple Type B meetings, according to uniQure.

• In September 2035, UniQure reported positive top-line results from its Phase I/II study of AMT-130, a one-time gene therapy for Huntington’s disease (HD). Top-line data provides a rapid summary of key study results at a predetermined timepoint. The update indicates that AMT-130 significantly slows symptom progression and met the trial’s primary endpoint, marking the first time a therapy has demonstrated the ability to alter the course of HD in a clinical setting.

• In June 2025, Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company focused on developing small molecule therapies targeting RNA, announced the dosing of the first patient in its Phase 2/3 FALCON-HD trial. This study is evaluating SKY-0515, an investigational oral RNA splicing modulator for Huntington’s disease. SKY-0515 is designed to lower the production of HTT and PMS1 proteins, both of which play key roles in Huntington’s disease pathology. In a Phase 1 study involving healthy volunteers, SKY-0515 showed a dose-dependent reduction in HTT mRNA, achieving an average 72% decrease at the highest dose, and was generally well tolerated. Additionally, the Phase 1 trial in Huntington’s disease patients, which began in January 2025, completed enrollment ahead of schedule by March 2025.

Huntington’s Disease Overview

Huntington’s disease is a hereditary neurological disorder characterized by progressive degeneration of brain cells, particularly in areas associated with movement, cognition, and behavior. It is caused by a mutation in the HTT gene, leading to the production of abnormal huntingtin protein aggregates in the brain. Symptoms typically manifest in adulthood and worsen over time, including involuntary movements (chorea), cognitive decline, psychiatric symptoms, and impaired motor function.

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Emerging Huntington’s Disease Drugs Under Different Phases of Clinical Development Include:

• AUSTEDO (deutetrabenazine/SD-809): Teva Pharmaceutical

• ANX005: Annexon Biosciences

• Pridopidine(ACR-16; Huntexil): Prilenia Therapeutics

• Ingrezza (valbenazine): Neurocrine Biosciences

• SOM3355(bevantolol): Som Biotech

• ANX005: Annexon Biosciences

• VX15/2503 (pepinemab): Vaccinex

• SAGE-718: Sage Therapeutics

• AMT-130/ intrastriatal rAAV5-miHTT: UniQure Biopharma

• RO7234292: Roche

• Tominersen: Ionis Pharmaceuticals

• Valbenazine: Neurocrine Biosciences

• Pepinemab: Vaccinex

• SRX246: Azevan Pharmaceuticals

• WVE-003: WaVe life Sciences

• LPM3770164: Luye Pharma Group

• SOL-175: SOLA Biosciences

Huntington’s Disease Route of Administration

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

• Oral

• Parenteral

• Intravenous

• Subcutaneous

• Topical

Huntington’s Disease Molecule Type

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

• Monoclonal Antibody

• Peptides

• Polymer

• Small molecule

• Gene therapy

Huntington’s Disease Pipeline Therapeutics Assessment

• Huntington’s Disease Assessment by Product Type

• Huntington’s Disease By Stage and Product Type

• Huntington’s Disease Assessment by Route of Administration

• Huntington’s Disease By Stage and Route of Administration

• Huntington’s Disease Assessment by Molecule Type

• Huntington’s Disease by Stage and Molecule Type

DelveInsight’s Huntington’s Disease Report covers around 20+ products under different phases of clinical development like

• Late-stage products (Phase III)

• Mid-stage products (Phase II)

• Early-stage product (Phase I)

• Pre-clinical and Discovery stage candidates

• Discontinued & Inactive candidates

• Route of Administration

Further Huntington’s Disease product details are provided in the report. Download the Huntington’s Disease pipeline report to learn more about the emerging Huntington’s Disease therapies

Some of the key companies in the Huntington’s Disease Therapeutics Market include:

Key companies developing therapies for Huntington’s Disease are – Prilenia Therapeutics, Ionis Pharmaceuticals, Annexon, Vaccinex, Neurocrine Biosciences, SAGE Therapeutics, PTC Therapeutics, WaVe life Sciences, Novartis, Neurimmune Therapeutics, SOLA Biosciences, UniQure Biopharma, Roche, Annexon, Inc., Azevan Pharmaceuticals, SOM Innovation Biotech SA, Asklepios BioPharmaceutical, Luye Pharma Group Ltd., BrainStorm Cell Therapeutics, Voyager Therapeutics, Passage Bio, Locana Bio, Ceptur Therapeutics, Enzerna Biosciences, AFFiRiS AG, Arvinas, Atalanta Therapeutics, Anima Biotech, and others.

Huntington’s Disease Pipeline Analysis:

The Huntington’s Disease pipeline report provides insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Huntington’s Disease with aggregate therapies developed by each company for the same.

• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Huntington’s Disease Treatment.

• Huntington’s Disease key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

• Huntington’s Disease 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.

The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.

Download Sample PDF Report to know more about Huntington’s Disease drugs and therapies

Huntington’s Disease Pipeline Market Drivers

• Strong unmet medical need due to the lack of disease-modifying therapies for Huntington’s disease.

• Advancements in genetic research and RNA-based technologies (antisense oligonucleotides, gene silencing) are accelerating pipeline development.

• Increasing investment from biotech companies and support from rare disease funding initiatives.

• Growing awareness, improved genetic testing, and earlier diagnosis are expanding the eligible patient pool for clinical trials.

Huntington’s Disease Pipeline Market Barriers

• High clinical trial failure rates due to disease complexity and challenges in demonstrating clinical efficacy.

• Stringent regulatory requirements and long development timelines for neurodegenerative disorders.

• Limited patient population, making trial recruitment and large-scale studies difficult.

• Safety concerns, particularly with gene and RNA-based therapies, may slow regulatory approval and adoption.

Scope of Huntington’s Disease Pipeline Drug Insight

• Coverage: Global

• Key Huntington’s Disease Companies: Teva Pharmaceutical, Annexon Biosciences, Prilenia Therapeutics, Neurocrine Biosciences, Som Biotech, Annexon Biosciences, Vaccinex, Sage Therapeutics, UniQure Biopharma, Roche, Ionis Pharmaceuticals, Neurocrine Biosciences, Vaccinex, Azevan Pharmaceuticals, WaVe life Sciences, Luye Pharma Group, SOLA Biosciences, and others

• Key Huntington’s Disease Therapies: AUSTEDO (deutetrabenazine/SD-809), ANX005, Pridopidine(ACR-16; Huntexil), Ingrezza (valbenazine), SOM3355(bevantolol), ANX005, VX15/2503 (pepinemab), SAGE-718, AMT-130/ intrastriatal rAAV5-miHTT, RO7234292, Tominersen, Valbenazine, Pepinemab, SRX246, WVE-003, LPM3770164, SOL-175, and others

• Huntington’s Disease Therapeutic Assessment: Huntington’s Disease current marketed and Huntington’s Disease emerging therapies

• Huntington’s Disease Market Dynamics: Huntington’s Disease market drivers and Huntington’s Disease market barriers

Request for Sample PDF Report for Huntington’s Disease Pipeline Assessment and clinical trials

Table of Contents

1. Huntington’s Disease Report Introduction

2. Huntington’s Disease Executive Summary

3. Huntington’s Disease Overview

4. Huntington’s Disease- Analytical Perspective In-depth Commercial Assessment

5. Huntington’s Disease Pipeline Therapeutics

6. Huntington’s Disease Late Stage Products (Phase II/III)

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

8. Huntington’s Disease Early Stage Products (Phase I)

9. Huntington’s Disease Preclinical Stage Products

10. Huntington’s Disease Therapeutics Assessment

11. Huntington’s Disease Inactive Products

12. Company-University Collaborations (Licensing/Partnering) Analysis

13. Huntington’s Disease Key Companies

14. Huntington’s Disease Key Products

15. Huntington’s Disease Unmet Needs

16 . Huntington’s Disease Market Drivers and Barriers

17. Huntington’s Disease Future Perspectives and Conclusion

18. Huntington’s Disease Analyst Views

19. Appendix

20. About DelveInsight

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. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

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