DelveInsight’s “Huntington’s Disease Pipeline Insight 2025” report offers in-depth analysis of over 20 companies and more than 20 pipeline drugs in the Huntington’s Disease treatment landscape. It features detailed profiles of Huntington’s Disease Pipeline drugs, covering both clinical and nonclinical development phases. Moreover, it assesses Huntington’s Disease pipeline therapeutics by product type, development stage, administration route, and molecular type. It also highlights discontinued pipeline products in this area.
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Key Takeaways from the Huntington’s Disease Pipeline Report
On September 19, 2025, Azidus Brasil conducted a Phase II dose-response study where participants with HD will receive three intravenous injections of the investigational product or placebo (one every month for three months), totaling three cycles. The subjects will be randomized in a 2:2:1 ratio for the groups G1: lower dose (1×10^6 cells/weight range), G2: higher dose (2×10^6 cells/weight range), or G3: placebo.
On September 18, 2025, Brainvectis, a subsidiary of Asklepios BioPharmaceutical, Inc. (AskBio) conducted a trial with 2 parts: Dose-Finding Part and Expansion Part; each part has 3 phases: Screening Phase (8 weeks, extendable to 12 weeks for scheduling), Treatment and Initial Follow-Up Phase (52 weeks), and Long-Term Follow-Up Phase (4 years). In the Dose-Finding Part, 2 dose titers will be tested in 3-6 subjects per cohort. Once a dose is selected based on Dose-Limiting Toxicities, an additional 6 subjects will be enrolled into the Dose Expansion Part.
On September 15, 2025, Supernus Pharmaceuticals Inc. organized a study to evaluate the size of baseline differences between participants with early Huntington’s Disease (HD) and healthy participants (HP) regarding cognitive performance metrics. DelveInsight’s Huntington’s Disease pipeline report showcases a thriving field with over 20 active participants developing more than 20 pipeline therapies for Huntington’s Disease management.
The leading Huntington’s Disease Companies include Hoffmann-La Roche, PTC Therapeutics, Annexon, Alnylam Pharmaceuticals, Neuvivo, BPG Bio, and others.
Promising Huntington’s Disease Pipeline Therapies include 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 emphasizes the unmet needs related to Huntington’s Disease.
Huntington’s Disease Overview
Huntington’s disease is a hereditary, progressive, neurodegenerative condition characterized by the gradual onset of involuntary muscle movements affecting the hands, feet, face, and trunk, along with progressive decline in cognitive processes and memory (dementia). This condition causes alterations in the central brain region, impacting movement, mood, and thinking abilities. Symptoms of Huntington’s disease typically appear between ages 30 and 50, but can start 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 Huntington’s disease treatment. Tominersen is engineered to decrease the production of all forms of the huntingtin (HTT) protein, where its mutated variant (mHTT) is responsible for Huntington’s disease. The drug is currently in Phase III clinical trials for Huntington’s disease.
PTC518: PTC Therapeutics PTC518, an orally administered small molecule, lowers the production of the mutated Huntingtin protein that causes neuronal injury and death, resulting in disease progression. The orally bioavailable small molecule crosses the blood-brain barrier, is selective, adjustable, and not effluxed. The drug is in Phase II clinical trials for Huntington’s disease.
ALN-HTT02: Alnylam Pharmaceuticals ALN-HTT02 is an innovative therapeutic strategy for Huntington’s disease from Alnylam Pharmaceuticals. This drug is a small interfering RNA (siRNA) aimed at reducing huntingtin protein production, which is linked to the disease’s pathology. It functions by targeting a specific HTT gene messenger RNA (mRNA) region, facilitating its breakdown and thus blocking the synthesis of both normal and mutant huntingtin proteins. Delivered via intrathecal injection. The drug is in Phase I clinical trials for Huntington’s disease.
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The Huntington’s Disease Pipeline Report Provides Insights into
The report offers detailed information on companies developing therapies for Huntington’s Disease, including the total therapies each company is advancing. It reviews various therapeutic candidates divided into early-stage, mid-stage, and late-stage development for Huntington’s Disease treatment. Huntington’s Disease Companies are focused on targeted therapeutics with active and inactive (dormant or discontinued) projects. Huntington’s Disease Drugs in development, categorized by development stage, administration route, target receptor, monotherapy or combination therapy, distinct mechanism of action, and molecular type. In-depth analysis of collaborations (company-to-company and company-academia partnerships), licensing agreements, and financing details to support future progress in 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 evaluates the therapeutic potential of pipeline drugs by Route of Administration. Products are classified 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 Transform your understanding of the Huntington’s Disease Pipeline! See the latest progress in drug development and clinical research @ Huntington’s Disease Market Drivers and Barriers, and Future Perspectives
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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