Huntington Disease Pipeline Gains Momentum: 20+ Companies Lead the Charge in Pioneering New Treatments | DelveInsight
DelveInsight’s “Huntington’s Disease Pipeline Insight 2026” report provides comprehensive insights about 20+ companies and 20+ pipeline drugs in the Huntington’s Disease pipeline landscape. It covers the Huntington’s Disease Pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Huntington’s Disease pipeline 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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Key Takeaways from the Huntington’s Disease Pipeline Report
- On July 01, 2026, Skyhawk Therapeutics Inc. initiated a phase 2/3 clinical trial is to test if the drug SKY-0515, an oral medication, can lower harmful proteins linked to Huntington’s Disease (HD) and improve the symptoms of participants with HD. This study includes men and women aged 25 and older who have HD confirmed by genetic testing and meet certain requirements for physical ability and independence.
- On June 23, 2026- Novartis Pharmaceuticals conducted a Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington’s Disease. The purpose is to assess safety and tolerability of votoplam and to determine whether votoplam slows disease progression in patients with early symptomatic Huntington’s disease (HD) compared to the control arm.
- On June 17, 2026- Neurocrine Biosciences highlighted a Phase 3, open-label study to evaluate the long-term safety and tolerability of valbenazine, and to provide participants continued access to valbenazine for the treatment of chorea associated with Huntington disease.
- On June 09, 2026- Teva Branded Pharmaceutical Products R&D LLC announced a study is to evaluate the real-world effectiveness of deutetrabenazine in adult patients with chorea associated with Huntington’s disease in China.
- 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.
- The leading Huntington’s Disease Companies such as Hoffmann-La Roche, PTC Therapeutics, Annexon, Alnylam Pharmaceuticals, Neuvivo, BPG Bio, and others.
- Promising Huntington’s Disease Pipeline Therapies such as Dimebon, Pridopidine, SAGE-718, Tominersen 60 mg, AB-1001, ISIS 443139 10 mg, ER2001 injection, and others.
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The Huntington’s Disease Pipeline Report provides disease overview, pipeline scenario and therapeutic assessment of the key pipeline therapies in this domain. The Huntington’s Disease Pipeline Report also highlights the unmet needs with respect to the Huntington’s Disease.
Huntington’s Disease Overview
Huntington’s disease is a genetic, progressive, neurodegenerative disorder characterized by the gradual development of involuntary muscle movements affecting the hands, feet, face, and trunk and progressive deterioration of cognitive processes and memory (dementia). This disease causes changes in the central area of the brain, which affect movement, mood and thinking skills. Symptoms of Huntington’s disease usually develop between ages 30 and 50, but they can appear as early as age 2 or as late as 80.
Huntington’s Disease Emerging Drugs Profile
- RG6042: Hoffmann-La Roche
Tominersen (ASO-HTT, RG6042) is an antisense drug in development for the treatment of Huntington’s disease. Tominersen is designed to reduce the production of all forms of the huntingtin (HTT) protein, which in its mutated variant (mHTT) is responsible for Huntington’s disease. Currently, the drug is in Phase III stage of its clinical trial for the treatment of Huntington’s disease.
- PTC518: PTC Therapeutics
PTC518, a small molecule that can be taken orally, reduces the production of the mutated Huntingtin protein that leads to injury and death of the neuron, which results in disease progression. The orally bioavailable small molecule penetrates the blood brain barrier, is selective, titratable, and not effluxed. Currently the drug is in Phase II stage of its clinical trial for the treatment of Huntington’s disease.
- ALN-HTT02: Alnylam Pharmaceuticals
ALN-HTT02 is an innovative therapeutic approach for Huntington’s disease developed by Alnylam Pharmaceuticals. This drug is a small interfering RNA (siRNA) designed to reduce the production of the huntingtin protein, which is implicated in the disease’s pathology. It works by targeting a specific region of the HTT gene’s messenger RNA (mRNA), promoting its degradation and thereby preventing the synthesis of both normal and mutant huntingtin proteins. Administered via intrathecal injection. Currently, the drug is in Phase I stage of its clinical trial for the treatment of Huntington’s disease.
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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 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
Huntington’s Disease Companies
Hoffmann-La Roche, PTC Therapeutics, Annexon, Alnylam Pharmaceuticals, Neuvivo, BPG Bio and others.
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
- Intra-articular
- Intraocular
- Intrathecal
- Intravenous
- Oral
- Parenteral
- Subcutaneous
- Topical
- Transdermal
Huntington’s Disease Products have been categorized under various Molecule types such as
- Oligonucleotide
- Peptide
- Small molecule
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Scope of the Huntington’s Disease Pipeline Report
- Coverage- Global
- Huntington’s Disease Companies- Hoffmann-La Roche, PTC Therapeutics, Annexon, Alnylam Pharmaceuticals, Neuvivo, BPG Bio and others.
- Huntington’s Disease Pipeline Therapies- Dimebon, Pridopidine, SAGE-718, Tominersen 60 mg, AB-1001, ISIS 443139 10 mg, ER2001 injection, and others.
- Huntington’s Disease Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
- Huntington’s Disease Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
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Table of Contents
- Introduction
- Executive Summary
- Huntington’s Disease: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Huntington’s Disease – DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- RG6042: Hoffmann-La Roche
- Mid Stage Products (Phase II)
- PTC518: PTC Therapeutics
- Early Stage Products (Phase I)
- ALN-HTT02: Alnylam Pharmaceuticals
- Mid Stage Products (Phase II)
- Preclinical Stage Products
- Drug Name: Company Name
- Inactive Products
- Huntington’s Disease – Collaborations Assessment- Licensing / Partnering / Funding
- Huntington’s Disease – Unmet Needs
- Huntington’s Disease – Market Drivers and Barriers
- Appendix
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