Huntington Disease Pipeline Advancements Report 2025: Novel Mechanisms and High-Impact Therapies Transforming Global Healthcare

DelveInsight's "Huntington's Disease Pipeline Insight 2025" 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 21 November 2025, Novartis Pharmaceuticals announced a Phase 2a Study PTC518-CNS-002-HD (NCT05358717), fulfilled the enrollment criteria, and chose to enroll in this extension study will undergo baseline evaluations and be assessed for 54 additional months.
* On 18 November 2025, Hoffmann-La Roche initiated a phase II study will evaluate the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntington's Disease (HD).
* On 17 November 2025, Skyhawk Therapeutics Inc . conducted a clinical trial aims to evaluate the safety, pharmacodynamics, and efficacy of SKY-0515, a novel, orally-administered small molecule mRNA-splicing modulator for treating Huntington's Disease (HD). HD is a rare, inherited neurodegenerative condition caused by a mutation in the huntingtin (HTT) gene, resulting in excessive production of mutant huntingtin (mHTT) protein, which damages nerve cells and leads to progressive motor, cognitive, and psychiatric symptoms.
* 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

About Us

DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.

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