Huntington’s disease is a progressive brain disorder caused by a single defective gene on chromosome 4. This gene produces a protein called huntingtin, which is essential for normal brain development. However, when the gene is defective, it produces an abnormal form of huntingtin that is toxic to brain cells. The damage caused by the abnormal protein gradually destroys certain nerve cells in the brain, which leads to the characteristic symptoms of Huntington’s disease.

The exact cause of the genetic defect that leads to Huntington’s disease is unknown. However, it is thought to be due to a combination of environmental and genetic factors. Huntington’s disease is usually diagnosed in adults between the ages of 30 and 50, but it can also occur in children and adolescents. The disease is usually fatal within 15 to 20 years of onset.

Huntington’s disease patients may develop symptoms like uncontrolled movements (chorea), mood swings, depression, irritability, slowed movements and speech, loss of hand coordination and difficulty swallowing.

Healthcare provider will perform a physical exam and look for twitches and jerking as well as problems with balance, reflexes and coordination. Neurologist will also examine the family history. Blood test, genetic testing and imaging tests such as magnetic resonance imaging (MRI) and computed tomography (CT) scan can also be recommended.

Huntington’s disease can be treated with medications like drugs to control movement, antipsychotic drugs, antidepressants, mood stabilizing drugs and other medications. Psychotherapy, speech therapy, physical therapy and occupational therapy can be used for treatment.

There are many clinical trials underway for Huntington’s disease drug development. Some of these trials are testing new drugs, while others are testing new combinations of existing drugs. Many of these trials are small, and are focused on safety and tolerability rather than efficacy.

Major pharmaceutical companies are involved in the development of potential drug candidates to improve the treatment of Huntington’s Disease treatment such as Deutetrabenazine, Pridopidine, LPM3770164, and others. Key players involved in the development of therapies to treat Huntington’s Disease are Luye Pharma, Prilenia Therapeutics, Asklepios, Biopharmaceuticals, and others. Six drugs are underpahse III stage, 10+ drugs are in Phase II clinical trials and five drugs are in Phase I clinical trials and some other drugs are under Phase I, preclinical and discovery stages of development. In August 2022, Asklepios biopharmaceutical received clearance to conduct a Phase I/II trial of BV-101 for Huntington’s disease.

Report Highlights

Global Insight Service’s, Huntington’s Disease – Drug Pipeline Landscape, 2023 report provides an overview of the Huntington disease pipeline drugs. This report covers detailed insights on Huntington’s Disease drugs under development, assessment by target, mechanism of action, route of administration and molecule type. Product pipeline by companies, stage of development and key regulatory designations, deals and milestones have been presented to provide insights and thus help industry participants in their decision making. Huntington’s Disease pipeline report helps gain insights on drugs which are under development stage of drug development process across globally.


The research process includes extensive secondary research on public domain and other authentic sources to add or update the pipeline products information. The secondary research sources include, but are not limited to company websites, annual reports, financial reports, company pipeline chart, investor presentations and SEC filings, journals and conferences, and clinical trials registries.


The pipeline landscape report provides analysis of pipeline products based on several stages of development ranging from Discovery to Pre-Registration. The report provides a review of pipeline therapeutics by companies based on information derived from company and industry-specific sources. The pipeline report covers assessment of therapeutics by mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. Comprehensive profiles of the pipeline products with details such as company overview, development stage; molecule type, target, mechanism of action, route of administration, dosage form, regulatory designations, key deals, clinical trials, and key upcoming milestones are included.

Reasons to Buy
    Helps to find and recognize significant therapeutics under development. Thorough understanding of pipeline structure and helps in developing corrective measures for pipeline projects.
    Effective R&D strategies can be developed with deep knowledge of competitor information, analysis, and insights.
    Plan collaborations with various industry partners that have role in some or the other stage of drug development such as contract manufacturing, co-development, contract research organization and commercialization etc.
    Helps to create in-licensing and out-licensing opportunities by identifying prospective partners with attractive projects to expand business potential and scope.
Table of Contents

List of Tables
List of Figures

1. Introduction
1.1 Huntington’s Disease – Pipeline Drugs, 2023-Coverage

2. Disease Overview – Huntington’s Disease
2.1 Causes
2.2 Signs and Symptoms
2.3 Diagnosis
2.4 Disease Management

3. Huntington’s Disease – Pipeline Drugs Development – Overview
3.1 Comparative Analysis by Stage of Development
3.2 Number of Products under Development by Companies, 2023
3.3 Products under Development by Companies, 2023

4. Assessment of Therapeutics
4.1 Assessment by Target
4.2 Assessment by Mechanism of Action
4.3 Assessment by Molecule Type
4.3 Assessment by Route of Administration

5. Drug Profiles
5.1 Clinical Stage Drugs-Phase III
5.1.1 Cellavita-HD
5.1.2 Deutetrabenazine
5.1.3 mAB C6-17
5.1.4 Pridopidine
5.1.5 RO7234292 (RG6042)
5.1.6 Valbenazine
5.2 Clinical Stage Drugs-Phase II
5.2.1 ANX005
5.2.2 LMI070/Branaplam
5.2.3 MP101
5.2.4 PTC518
5.2.5 rAAV5-miHTT
5.2.6 SAGE-718
5.2.7 Selisistat
5.2.8 SOM3355
5.2.9 SRX246
5.2.10 VX15/2503
5.2.11 WVE-003
5.3 Clinical Stage Drugs-Phase I
5.3.1 AJ201
5.3.2 CKD-504
5.3.3 NanoLithium
5.3.4 Nilotinib
5.3.5 T3D-959
5.4 Early Stage Drugs- IND/CTA Filed
5.4.1 ALS205
5.4.2 MMJ-002
5.4.3 BV-101
5.5 Early Stage Drugs- Preclinical
5.5.1 ASRE therapeutic
5.5.2 CTX0E03
5.5.3 DFMO
5.5.4 Drug for Huntington’s disease
5.5.5 Drug for Huntington’s disease
5.5.6 EHP-102
5.5.7 Engineered Exosomes
5.5.8 Gold-memantine (AuM)
5.5.9 HTT
5.5.10 HV-3
5.5.11 INT41
5.5.12 JNK/Mitochondria
5.5.13 LPM3770164
5.5.14 mHTT
5.5.15 MR-101
5.5.16 MSC-NTF Cells
5.5.17 MTK-458
5.5.18 NanosiRNA
5.5.19 NT-0100
5.5.20 NXL-001
5.5.21 OCCT-HTT siRNA
5.5.22 OXD4
5.5.23 RNA-targeted gene therapy
5.5.24 SC379
5.5.25 Small molecules for Huntington disease
5.5.26 SOL-176
5.5.27 TTP-34
5.5.28 TTX-3360
5.5.29 UBE2K Modulators
5.5.30 VO659
5.6 Early Stage Drugs – Discovery
5.6.1 Antibody therapy for Huntington’s disease
5.6.2 Asdera LLC
5.6.3 Cathepsin Inhibitor
5.6.4 Drug for Huntington’s disease
5.6.5 Drug for Huntington’s disease
5.6.6 Drug for Huntington’s disease
5.6.7 Drug for Huntington’s disease
5.6.8 Drug for Huntington’s disease
5.6.9 HTT
5.6.10 Huntington program
5.6.11 Huntington’s Program
5.6.12 NI302
5.6.13 ORI-113
5.6.14 ORI-503
5.6.15 Other stabilized PUFAs
5.6.16 PRI-100
5.7 Unknown Stage Drugs
5.7.1 PDE10A inhibitor

6. Key Regulatory Designations

7. Key Deals

8. Key Upcoming Milestones

9. Key Companies Involved
9.1 AFFiRiS AG
9.2 Alchemab Therapeutics Ltd
9.3 Alsonex Pty Ltd
9.4 Anima Biotech Inc
9.5 Annexon, Inc.
9.6 Annji Pharmaceutical Co Ltd
9.7 Aop Orphan Pharmaceuticals AG
9.8 Arvinas Inc
9.9 Asdera LLC
9.10 Asklepios BioPharmaceutical Inc
9.11 Atalanta Therapeutics Inc
9.12 Azevan Pharmaceuticals
9.13 Berg LLC
9.14 Bionaut Labs Inc
9.15 BrainStorm Cell Therapeutics Inc
9.16 Cellavita Pesquisa Científica Ltda
9.17 Celon Pharma
9.18 Ceptur Therapeutics Inc
9.19 Chaperone Therapeutics Inc
9.20 Chong Kun Dang Pharmaceutical Corp
9.21 Collaborative Medicinal Development LLC
9.22 Emerald Health Sciences Inc
9.23 Enzerna Biosciences LLC
9.24 Exicure Inc
9.25 Exopharm Ltd
9.26 Hoffmann-La Roche
9.27 Janusq LLC
9.28 KeifeRx LLC
9.29 Locanabio Inc
9.30 Luye Pharma Group Ltd.
9.31 Medesis Pharma SA
9.32 Mitochon Pharmaceuticals Inc
9.33 Mitokinin LLC
9.34 MMJ International Holdings Corp
9.35 NeuBase Therapeutics Inc
9.36 NeuExcell Therapeutics Inc
9.37 Neurano Bioscience
9.38 Neurimmune Holding AG
9.39 Neurocrine Biosciences
9.40 Neurodon LLC
9.41 Neuropore Therapies Inc
9.42 Novartis Pharmaceuticals
9.43 Ophidion Inc
9.44 Origami Therapeutics Inc
9.45 Oxalys Pharmaceuticals Inc
9.46 PolyCore Therapeutics LLC
9.47 Priavoid GmbH
9.48 Prilenia
9.49 PTC Therapeutics
9.50 reMYND NV
9.51 ReNeuron Group Plc
9.52 Resilio Therapeutics LLC
9.53 ResQ Biotech
9.54 Retrotope Inc
9.55 Sage Therapeutics
9.56 Sana Biotechnology Inc
9.57 Shinkei Therapeutics LLC
9.58 SOLA Biosciences LLC
9.59 SOM Innovation Biotech SA
9.60 Spark Therapeutics Inc
9.61 T3D Therapeutics Inc
9.62 Teitur Trophics ApS
9.63 Teva Branded Pharmaceutical Products R&D, Inc.
9.64 Triplet Therapeutics Inc
9.65 UniQure Biopharma B.V.
9.66 Vaccinex Inc
9.67 Vico Therapeutics BV
9.68 Voyager Therapeutics Inc
9.69 Vybion Inc
9.70 Wave Life Sciences Ltd.

10. Dormant Drugs
10.1 Inactive Drugs
10.2 Discontinued Drugs

11. Appendix

List of Tables

Table 1. 2 Number of Products under Development by Companies
Table 1. 3 Products under Development by Companies
Table 1. 4 Products by Targets
Table 1. 5 Products by Mechanism of Action
Table 1. 6 Products by Molecule Type
Table 1. 7 Products by Route of Administration
Table 2.1 Clinical Trial Details – Cellavita-HD/Cellavita Pesquisa Científica Ltda
Table 2.2 Clinical Trial Details – Deutetrabenazine/Teva Branded Pharmaceutical Products R&D, Inc.
Table 2.3 Clinical Trial Details – mAB C6-17/AFFiRiS AG
Table 2.4 Clinical Trial Details – Pridopidine/Prilenia
Table 2.5 Clinical Trial Details – RO7234292 (RG6042)/Hoffmann-La Roche
Table 2.6 Clinical Trial Details – Valbenazine/Neurocrine Biosciences
Table 2.7 Clinical Trial Details – ANX005/Annexon, Inc.
Table 2.8 Clinical Trial Details – LMI070/Branaplam/Novartis Pharmaceuticals
Table 2.9 Clinical Trial Details – PTC518/PTC Therapeutics
Table 2.10 Clinical Trial Details – rAAV5-miHTT/UniQure Biopharma B.V.
Table 2.11 Clinical Trial Details – SAGE-718/Sage Therapeutics
Table 2.12 Clinical Trial Details – SOM3355/SOM Innovation Biotech SA
Table 2.13 Clinical Trial Details – SRX246/Azevan Pharmaceuticals
Table 2.14 Clinical Trial Details – VX15/2503/Vaccinex Inc
Table 2.15 Clinical Trial Details – WVE-003/Wave Life Sciences Ltd.
Table 2.16 Clinical Trial Details – CKD-504/Chong Kun Dang Pharmaceutical Corp
Table 3. 1 Regulatory Designations
Table 4. 1 Inactive Drugs
Table 4. 2 Discontinued Drugs

List of Figures

Figure 1. 1 Number of Products under Development for Huntington’s Disease, 2023
Figure 1. 2 Products by Top 5 Targets and Stage of Development for Huntington’s Disease, 2023
Figure 1. 3 Products by Top 5 Mechanism of Action and Stage of Development for Huntington’s Disease, 2023
Figure 1. 4 Products by Top 5 Molecule Type and Stage of Development for Huntington’s Disease, 2023
Figure 1. 5 Products by Top 5 Route of Administration and Stage of Development for Huntington’s Disease, 2023

Key Players
Report Segmentation
  • Assessment by Stage of Development
  • Assessment by Companies
  • Assessment by Target
  • Assessment by Mechanism of Action
  • Assessment by Molecule Type
  • Assessment by Route of Administration

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