Huntington'S Disease - Pipeline Insight, 2025

The “Huntington’s Disease - Pipeline Insight, 2025” report provides comprehensive insights about 20+ companies and 20+ pipeline drugs in Huntington’s Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Huntington’s Disease: Understanding

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. It is characterized by rapid uncontrollable muscle movements such as tics or muscle jerks (choreiform movements or chorea). This disorder causes a loss of coordination and personality changes. As the disease progresses, the ability to speak may be impaired, memory may fade, and the involuntary jerky muscle movements (chorea) become more severe.

Huntington’s disease is inherited as an autosomal dominant trait. It is caused by a single defective gene on chromosome 4 - one of the 23 human chromosomes that carry a person’s entire genetic code. The defective gene codes the blueprint for a protein called huntingtin. This protein's normal function isn't yet known, but it's called "huntingtin" because scientists identified its defective form as the cause of Huntington's disease. Defective huntingtin protein leads to brain changes that cause abnormal involuntary movements, a severe decline in thinking and reasoning skills, and irritability, depression and other mood changes. The huntingtin gene defect involves extra repeats of one specific chemical code in one small section of chromosome 4. The normal huntingtin gene includes 17 to 20 repetitions of this code among its total of more than 3,100 codes. The defect that causes Huntington's disease includes 40 or more repeats.

The diagnosis of Huntington's disease may be confirmed by a thorough clinical evaluation, detailed patient history, and a variety of specialized tests. Tests include: Blood test, Genetic testing, and Imaging tests. Genetic testing determines if an individual have a mutation on the HTT gene. Specialized x-ray studies such as computerized tomography (CT) scanning, magnetic resonance imaging (MRI), or electroencephalography (EEG) may help confirm the diagnosis of Huntington's Disease. During CT scanning, a computer and x-rays are used to create a file showing cross-sectional images of the brain. During MRI, a magnetic field and radio waves are used to create cross-sectional images of the brain. During an EEG, an instrument records electrical activity of the brain.

Treatments focus on managing symptoms. Austedo and Xenazine are the only USFDA approved symptomatic therapies, but their approved indication is the treatment of chorea associated with Huntington's disease. Other treatment for Huntington's disease is symptomatic and supportive. There are some treatments that may alleviate various symptoms temporarily. To help with emotional symptoms Antidepressants, Anti-psychotic, and mood-stabilizing drugs are preferred.

“Huntington’s Disease - Pipeline Insight, 2025" report outlays comprehensive insights of present scenario and growth prospects across the mechanism of action. A detailed picture of the Huntington’s Disease pipeline landscape is provided which includes the disease overview and Huntington’s Disease treatment guidelines. The assessment part of the report embraces, in depth Huntington’s Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Huntington’s Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Huntington’s Disease R&D. The therapies under development are focused on novel approaches to treat/improve Huntington’s Disease.

Huntington’s Disease Emerging Drugs Chapters

This segment of the Huntington’s Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, Phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Huntington’s Disease Emerging Drugs

• 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

Huntington’s Disease: Therapeutic Assessment

This segment of the report provides insights about the different Huntington’s Disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Huntington’s Disease

There are approx. 20+ key companies which are developing the therapies for Huntington’s disease. The companies which have their Huntington’s Disease drug candidates in the most advanced stage, i.e. Phase III include Hoffmann-La Roche.

Phases

The report covers around 20+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

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

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Huntington’s Disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Huntington’s Disease therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Huntington’s Disease drugs.

Huntington’s Disease Report Insights

• Huntington’s Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Huntington’s Disease Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Huntington’s Disease drugs?
• How many Huntington’s Disease drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Huntington’s Disease?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Huntington’s Disease therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Huntington’s Disease and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Hoffmann-La Roche
• PTC Therapeutics
• Annexon
• Alnylam Pharmaceuticals
• Neuvivo
• BPG Bio

Key Products

• RG6042
• PTC518
• ANX-005
• ALN-HTT02
• NP-001
• Research programme

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