Amyotrophic Lateral Sclerosis Clinical Trials, Companies, Therapeutic Assessment, Therapies, Treatment Algorithm, and Pipeline Analysis | Ionis Pharmaceuticals, 1ST Biotherapeutics, Scholar Rock
Amyotrophic Lateral Sclerosis Pipeline constitutes 75+ key companies continuously working towards developing 80+ Amyotrophic Lateral Sclerosis treatment therapies, analyzes DelveInsight.
Amyotrophic Lateral Sclerosis Overview:
Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, is a progressive neurodegenerative condition that primarily targets motor neurons. The disease can arise from both genetic (familial) and non-genetic (sporadic) factors, though a single definitive cause has not been identified. ALS typically impacts both upper and lower motor neurons, beginning with limb weakness and gradually progressing to paralysis and, ultimately, death.
The underlying cause of sporadic ALS remains largely unknown, though several mechanisms have been proposed, including disrupted RNA processing, oxidative stress from SOD1 mutations, inflammation, and glutamate toxicity. Familial ALS, which is less common, is linked to inherited genetic mutations such as expansions in the C9ORF72 gene and SOD1 gene mutations, typically passed down in an autosomal dominant manner.
Breathing difficulties are a major concern in ALS due to respiratory muscle weakening. Noninvasive ventilation is commonly used to manage symptoms like shortness of breath or reduced forced vital capacity (FVC), while invasive ventilation may be required in more severe cases. Difficulty swallowing (dysphagia) is also frequent and may be managed through dietary modifications or, if necessary, feeding via a gastrostomy (G-tube) to prevent weight loss.
ALS can lead to various complications, including respiratory failure, speech and mobility issues, malnutrition, fatigue, muscle spasms, and problems with saliva and mucus control. Additionally, medications used to manage ALS symptoms may cause side effects such as gastrointestinal discomfort, liver complications, or headaches.
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“Amyotrophic Lateral Sclerosis Pipeline Insight 2025” report by DelveInsight provides a comprehensive analysis of the ongoing clinical development activities and growth prospects across the Amyotrophic Lateral Sclerosis Therapeutics Market.
Key Takeaways from the Amyotrophic Lateral Sclerosis Pipeline Report
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DelveInsight’s Amyotrophic Lateral Sclerosis pipeline report depicts a robust space with 75+ active players working to develop 80+ pipeline therapies for Amyotrophic Lateral Sclerosis treatment.
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In November 2025, Ractigen Therapeutics announced that its siRNA therapy RAG-21, which targets ALS associated with FUS gene mutations, received Orphan Drug Designation from the FDA.
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Key Amyotrophic Lateral Sclerosis companies such as Ionis Pharmaceuticals, 1ST Biotherapeutics, Scholar Rock, Revalesio, QurAlis Corporation, Sanofi, MediciNova, Helixmith, Verge Genomics, UCB, and others are evaluating new drugs for Amyotrophic Lateral Sclerosis to improve the treatment landscape.
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Promising Amyotrophic Lateral Sclerosis pipeline therapies in various stages of development include MN-166, RNS60, VM202, QRL-201, and others.
Amyotrophic Lateral Sclerosis Pipeline Analysis
The report provides insights into:
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The report provides detailed insights into the key companies that are developing therapies in the Amyotrophic Lateral Sclerosis Market.
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The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Amyotrophic Lateral Sclerosis treatment.
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It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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It navigates the emerging 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.
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Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement, and financing details for future advancement of the Amyotrophic Lateral Sclerosis market.
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Amyotrophic Lateral Sclerosis Emerging Drugs
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MN-166: MediciNova
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RNS60: Revalesio
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VM202: Helixmith
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QRL-201: QurAlis Corporation
Amyotrophic Lateral Sclerosis Companies
More than 75 major companies are actively involved in developing treatments for Amyotrophic Lateral Sclerosis (ALS). Among them, MediciNova has drug candidates in the most advanced stages of development, currently in Phase II/III clinical trials.
DelveInsight’s report covers around 80+ products under different phases of clinical development like
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Late stage products (Phase III)
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Mid-stage products (Phase II)
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Early-stage product (Phase I) along with the details of
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Pre-clinical and Discovery stage candidates
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Discontinued & Inactive candidates
Amyotrophic Lateral Sclerosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
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Intravenous
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Subcutaneous
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Oral
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Intramuscular
Amyotrophic Lateral Sclerosis Products have been categorized under various Molecule types such as
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Monoclonal antibody
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Small molecule
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Peptide
Download Sample Pages to Get an in-depth Assessment of the Emerging Amyotrophic Lateral Sclerosis Therapies and Key Companies: Amyotrophic Lateral Sclerosis Clinical Trials and advancements
Amyotrophic Lateral Sclerosis Pipeline Therapeutic Assessment
• Amyotrophic Lateral Sclerosis Assessment by Product Type
• Amyotrophic Lateral Sclerosis By Stage
• Amyotrophic Lateral Sclerosis Assessment by Route of Administration
• Amyotrophic Lateral Sclerosis Assessment by Molecule Type
Download Amyotrophic Lateral Sclerosis Sample report to know in detail about the Amyotrophic Lateral Sclerosis treatment market @ Amyotrophic Lateral Sclerosis Therapeutic Assessment
Table of Content
1. Report Introduction
2. Executive Summary
3. Amyotrophic Lateral Sclerosis Current Treatment Patterns
4. Amyotrophic Lateral Sclerosis – DelveInsight’s Analytical Perspective
5. Therapeutic Assessment
6. Amyotrophic Lateral Sclerosis Late-Stage Products (Phase-III)
7. Amyotrophic Lateral Sclerosis Mid-Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Amyotrophic Lateral Sclerosis Discontinued Products
13. Amyotrophic Lateral Sclerosis Product Profiles
14. Amyotrophic Lateral Sclerosis Key Companies
15. Amyotrophic Lateral Sclerosis Key Products
16. Dormant and Discontinued Products
17. Amyotrophic Lateral Sclerosis Unmet Needs
18. Amyotrophic Lateral Sclerosis Future Perspectives
19. Amyotrophic Lateral Sclerosis Analyst Review
20. Appendix
21. Report Methodology
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