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Report Overview

Huntington’s disease is a rare, inherited neurodegenerative disorder that causes the progressive breakdown of nerve cells in the brain, leading to movement, cognitive, and psychiatric impairments. According to Anitha Ajitkumar et al., 2025, the global prevalence of Huntington’s disease is 2.7 per 100,000 people. The therapeutic pipeline includes gene-silencing therapies, antisense oligonucleotides, and small molecule drugs targeting disease-modifying pathways. According to the Huntington’s disease pipeline analysis by Expert Market Research, increasing research funding, advancements in gene therapy, and growing awareness of neurodegenerative disorders are expected to drive pipeline growth in the coming years.

  • Major companies involved in the Huntington’s disease pipeline analysis include Skyhawk Therapeutics, Inc., Spark Therapeutics, Inc., and others.

  • Leading drugs currently in the pipeline include SKY-0515, PTC518, SPK-10001, and others.

  • The pipeline growth is driven by advancements in gene therapy, increasing clinical trials for RNA-based therapeutics, and rising investments in targeted neurodegenerative treatment development over the coming years.

Report Coverage

The Huntington’s Disease Pipeline Analysis Report by Expert Market Research gives comprehensive insights into Huntington’s disease therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for Huntington’s disease. The Huntington’s disease report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The Huntington’s disease pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with Huntington’s disease treatment guidelines to ensure optimal care practices.

The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to Huntington’s disease.

Huntington’s Disease Pipeline Analysis By Drug Class

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Huntington’s Disease Pipeline Outlook

Huntington’s disease is a hereditary and progressive neurodegenerative disorder caused by a mutation in the HTT gene located on chromosome 4. This defect leads to the gradual degeneration of neurons in specific brain regions, resulting in impaired motor control, cognitive decline, and psychiatric disturbances. The symptoms typically appear between the ages of 30 and 50 and progressively worsen over time.

Huntington’s disease treatment focuses on managing symptoms through medications that help control movement, psychiatric issues, and behavioral changes, improving patients’ quality of life and daily functioning. In August 2023, Neurocrine Biosciences received U.S. Food and Drug Administration approval for INGREZZA (valbenazine) capsules for treating chorea associated with Huntington’s disease. The once-daily selective VMAT2 inhibitor demonstrated significant improvement in chorea severity in Phase 3 KINECT-HD trials.

Huntington’s Disease Epidemiology

According to Anitha Ajitkumar et al., 2025, Huntington’s disease shows a global prevalence of approximately 2.7 per 100,000 individuals. The incidence is significantly higher among populations of Caucasian descent, ranging from 10.6 to 13.7 per 100,000, while much lower rates are observed in Asian and African populations. The geographical variation exceeds tenfold, primarily due to ethnic differences in CAG repeat lengths. The average CAG repeats length ranges from 18.4 to 18.7 in Caucasians and 17.5 to 17.7 in East Asians. Improved genetic testing and longer lifespans have also contributed to the increased prevalence. These epidemiological insights highlight the growing need for continued research and therapeutic development in the Huntington’s disease drug pipeline.

Huntington’s Disease – Pipeline Therapeutic Assessment

This section of the report covers the analysis of Huntington’s disease drug candidates based on several segmentations, including:

By Phase

The pipeline assessment report covers 50+ drug analyses based on phase:

  • Late-Stage Products (Phase 3 and Phase 4)
  • Mid-Stage Products (Phase 2)
  • Early-Stage Products (Phase I)
  • Preclinical and Discovery Stage Products

By Drug Class

The Huntington’s disease pipeline analysis report covers 50+ drug analyses based on drug classes:

  • Small Molecules
  • Monoclonal Antibodies
  • Gene Therapies
  • Peptides
  • Cell Therapies

By Route of Administration

The pipeline assessment report covers 50+ drug analyses based on the route of administration.

  • Oral
  • Parenteral
  • Others

Huntington’s Disease Pipeline Assessment Segmentation, By Phases

The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase II covers a major share of the total Huntington’s disease clinical trials at 52%. It is followed by phase I with 24% and phase III with 20%. The dominance of advanced clinical stages indicates strong research progress, reflecting growing therapeutic innovation and development potential that can significantly enhance treatment options for Huntington’s disease.

Huntington’s Disease Pipeline Assessment Segmentation, By Drug Classes

The drug molecule categories covered under the Huntington’s disease pipeline analysis include small molecules, monoclonal antibodies, gene therapies, peptides, and cell therapies. The Huntington’s disease report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for Huntington’s disease. Gene-silencing drug therapies are emerging as a promising class in the Huntington’s disease drug pipeline. For instance, in December 2024, Alnylam Pharmaceuticals initiated a first-in-human trial for ALN-HTT02, an investigational RNA interference (RNAi) therapeutic co-developed with Regeneron Pharmaceuticals. The drug targets and silences the faulty HTT gene responsible for producing harmful proteins in the brain, aiming to slow disease progression and potentially alter the course of Huntington’s disease.

Huntington’s Disease Clinical Trials – Key Players

The EMR report for the Huntington’s disease pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed Huntington’s disease therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in Huntington’s disease clinical trials:

  • Skyhawk Therapeutics, Inc.
  • Spark Therapeutics, Inc.
  • PTC Therapeutics
  • Brainvectis
  • Alnylam Pharmaceuticals
  • UniQure Biopharma B.V.
  • Hoffmann-La Roche
  • Vico Therapeutics B. V.
  • Teva Branded Pharmaceutical Products R&D, Inc.

Huntington’s Disease – Emerging Drugs Profile

This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for Huntington’s disease. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of Huntington’s disease drug candidates.

Drug: SKY-0515

SKY-0515 is an orally administered small-molecule mRNA splicing modulator developed by Skyhawk Therapeutics, Inc. The ongoing Phase 2/3 FALCON-HD study is evaluating its safety, efficacy, and pharmacodynamics in participants with Huntington’s disease (HD). The trial is aiming to determine whether SKY-0515 can reduce mutant huntingtin (mHTT) and PMS1 protein levels, both key drivers of HD progression, through targeted RNA splicing modulation. This study is enrolling 120 participants across Australia and New Zealand.

Drug: PTC518

PTC518, developed by PTC Therapeutics, is an orally bioavailable small molecule designed to reduce the production of the mutant Huntingtin protein responsible for neuronal damage in Huntington’s disease. The ongoing Phase 2b extension study is evaluating the long-term safety, efficacy, and pharmacodynamic effects of PTC518 in participants who completed the prior Phase 2a trial. This study is assessing continued treatment over 54 months to determine sustained therapeutic benefits and tolerability.

Genetic: SPK-10001

SPK-10001, developed and sponsored by Spark Therapeutics, Inc., is an investigational gene therapy currently being evaluated in a Phase 1/2 clinical study for Huntington’s disease. The study is examining the safety, tolerability, and early efficacy of a single, bilateral intraparenchymal infusion of SPK-10001 into the caudate and putamen. Delivered via an adeno-associated virus (AAV), SPK-10001 introduces genetic instructions for microRNA designed to lower both normal and mutant huntingtin protein levels, aiming to slow disease progression.

*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*

Key Questions Answered in the Huntington’s Disease Pipeline Insight Report

  • Which companies/institutions are leading the Huntington’s disease drug development?
  • Which company is leading the Huntington’s disease pipeline development activities?
  • What is the current Huntington’s disease commercial assessment?
  • What are the opportunities and challenges present in the Huntington’s disease pipeline landscape?
  • What is the efficacy and safety profile of Huntington’s disease pipeline drugs?
  • Which company is conducting major trials for Huntington’s disease drugs?
  • Which companies/institutions are involved in Huntington’s disease collaborations aimed at providing enhanced therapeutic alternatives for patients?
  • What are the geographies covered for clinical trials in Huntington’s disease?

Reasons To Buy This Report

The Huntington’s Disease Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for Huntington’s disease. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into Huntington’s disease collaborations, regulatory environments, and potential growth opportunities.

Related Reports

Global Clinical Trials Market

Huntington’s Disease Treatment Market

Huntington’s Disease Epidemiology Forecast

*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*

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Report Summary

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Key Highlights of the Report

Please note that the figures mentioned in the description serve as estimates and may vary from the actual figures presented in the final report.

Scope of the Report

Details

Drug Pipeline by Clinical Trial Phase

  • Late-Stage Products (Phase III and Phase IV)
  • Mid-Stage Products (Phase II)
  • Early-Stage Products (Phase I)
  • Preclinical and Discovery Stage Products

Route of Administration

  • Oral
  • Parenteral
  • Others

Drug Classes

  • Small Molecules
  • Monoclonal Antibodies
  • Gene Therapies
  • Peptides
  • Cell Therapies

Leading Sponsors Covered

  • Skyhawk Therapeutics, Inc.
  • Spark Therapeutics, Inc.
  • PTC Therapeutics
  • Brainvectis
  • Alnylam Pharmaceuticals
  • UniQure Biopharma B.V.
  • Hoffmann-La Roche
  • Vico Therapeutics B. V.
  • Teva Branded Pharmaceutical Products R&D, Inc.

Geographies Covered

  • North America
  • Europe
  • Asia Pacific
  • Others

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