Lou Gehrig's Disease Pipeline: 75+ Innovators Pioneering Breakthrough Therapies | DelveInsight

The Lou Gehrig's Disease market is rapidly advancing and is fueled by groundbreaking research and innovative therapies from companies such as Denali Therapeutics, AbbVie, Calico Life Sciences, and Clene Nanomedicine Biosciences. These industry pioneers are transforming treatment strategies and redefining the future of Lou Gehrig's Disease, bringing new hope to patients worldwide.

DelveInsight's "Lou Gehrig's Disease Pipeline Insight, 2025" comprehensively analyzes the current clinical landscape and growth prospects in the Lou Gehrig's Disease market. The report covers disease insights, treatment guidelines, and a detailed pipeline assessment from preclinical to marketed stages. It includes drug mechanisms, clinical studies, regulatory progress, and key developments such as collaborations, mergers, funding, and designations.

For emerging Lou Gehrig's Disease drugs, the Lou Gehrig's Disease pipeline analysis report provides a 360° view of the therapeutics landscape by development point, product type, route of administration, molecule type, and MOA. The pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.

Key Takeaways from the Lou Gehrig's Disease Pipeline Report
• DelveInsight's Lou Gehrig's Disease Pipeline analysis depicts a robust space with 75+ active players working to develop 80+ pipeline drugs for Lou Gehrig's Disease treatment.
• The leading Lou Gehrig's Disease companies include Ionis Pharmaceuticals, 1st Biotherapeutics, Scholar Rock, Revalesio, QurAlis Corporation, Sanofi, MediciNova, Helixmith, Verge Genomics, UCB, AB Science, Alector, GSK, Brainstorm Cell Therapeutics, Denali Therapeutics, AbbVie, Calico Life Sciences, Clene Nanomedicine Biosciences, Seelos Therapeutics, Prilenia Therapeutics, Rapa Therapeutics, Neurosense Therapeutics, Transposon Therapeutics, Annexon Biosciences, Corcept Therapeutics, Al-S Pharma, Orphai Therapeutics, and others are evaluating their lead assets to improve the Lou Gehrig's Disease treatment landscape.
• Key Lou Gehrig's Disease pipeline therapies in various stages of development include Masitinib, Latozinemab (AL001/GSK4527223), Nurown (MSC-NTF Cells), Ulefnersen (ION363), Ibudilast, DNL343, ABBV-CLS-7262, CNM-AU8, SLS-005 (Trehalose), Pridopidine, Rapa-501, Primec, Engensis (VM202), TPN-101, RNS60, ANX005, Dazucorilant (CORT113176), AP-101, SAR443820/DNL788, AIT-101 (LAM-002A), Ulefnersen, MN-166, RNS60, QRL-201, and others.
• In March 2025, DiagnaMed Holdings Corp. announced a significant milestone in rare disease research, receiving Orphan Drug Designation (ODD) from the U.S. FDA for molecular hydrogen in the treatment of amyotrophic lateral sclerosis (ALS).
• In March 2025, Trethera Corporation announced clinical results from treating an ALS patient with TRE-515 under the FDA Expanded Access program.
• In March 2025, SineuGene Therapeutics revealed that the FDA cleared its IND application for SNUG01, a first-in-class TRIM72-targeted gene therapy for ALS.
• In January 2025, the FDA granted approval for Zydus Lifesciences to proceed with a randomized Phase IIb trial of its oral NLRP3 inflammasome inhibitor, Usnoflast, for the treatment of amyotrophic lateral sclerosis (ALS).
• In December 2024, NeuroSense Therapeutics Ltd. announced the conclusion of a Type C meeting with the FDA regarding PrimeC for the treatment of amyotrophic lateral sclerosis (ALS). The meeting focused on the design of a proposed Phase 3 clinical study and the plan for a future 505(b)(2) marketing application submission.
• In November 2024, Ractigen Therapeutics announced that the FDA granted Orphan Drug Designation (ODD) to its novel siRNA therapy, RAG-21, targeting the FUS gene for amyotrophic lateral sclerosis (ALS).

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Lou Gehrig's Disease Overview
Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's Disease, is a progressive neurodegenerative disorder that affects motor neurons in the brain and spinal cord, leading to muscle weakness and loss of voluntary movement. As the disease advances, individuals experience increasing difficulty with walking, speaking, swallowing, and breathing. Although relatively rare, ALS is one of the most severe forms of motor neuron disease.
ALS occurs in two primary forms: sporadic and familial. Sporadic ALS is the most common, occurring without a known genetic link, while familial ALS is hereditary and associated with genetic mutations. Symptoms typically begin with muscle weakness in the hands, legs, or throat and progressively lead to widespread paralysis, though cognitive and sensory functions generally remain unaffected.
The exact causes of ALS remain unclear, but a combination of genetic, molecular, and environmental factors is believed to contribute. Research suggests that disruptions in protein folding, oxidative stress, and inflammation may play a role in motor neuron degeneration. Environmental triggers such as toxin exposure and head trauma have also been explored, though no single cause has been definitively identified.
Currently, there is no cure for ALS, and treatment focuses on managing symptoms and slowing disease progression. Multidisciplinary care, including physical, occupational, and speech therapy, can help maintain patients' quality of life. Assistive devices, such as ventilators and communication aids, become essential as the disease advances. Ongoing research aims to uncover the biological mechanisms behind ALS to develop potential disease-modifying therapies.

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Lou Gehrig's Disease Treatment Analysis: Drug Profile
Ulefnersen: Ionis Pharmaceuticals
Ulefnersen (formerly ION363) is an investigational antisense therapy designed to lower the production of the fused in sarcoma (FUS) protein, targeting ALS caused by FUS gene mutations. Named Jacifusen in honor of the first patient treated under expanded access, the therapy showed potential in preclinical models by preventing motor neuron loss. Ulefnersen is currently in Phase III clinical trials for ALS treatment.

MN-166: MediciNova
MN-166 is an oral small-molecule glial attenuator that modulates neuroinflammation by suppressing pro-inflammatory cytokines (IL-1ß, TNF-a, IL-6) and potentially enhancing anti-inflammatory responses. It also functions as a toll-like receptor 4 (TLR4) antagonist, which may further reduce neuroinflammation. MN-166 is currently in Phase II/III clinical trials for ALS.

Key Lou Gehrig's Disease Therapies and Companies
• MASITINIB: AB SCIENCE
• LATOZINEMAB (AL001/GSK4527223): ALECTOR/GSK
• NUROWN (MSC-NTF CELLS): BRAINSTORM CELL THERAPEUTICS
• ULEFNERSEN (ION363): IONIS PHARMACEUTICALS
• IBUDILAST: MEDICINOVA
• DNL343: DENALI THERAPEUTICS
• ABBV-CLS-7262: ABBVIE/CALICO LIFE SCIENCES
• CNM-AU8: CLENE NANOMEDICINE BIOSCIENCES
• SLS-005 (TREHALOSE): SEELOS THERAPEUTICS
• PRIDOPIDINE: PRILENIA THERAPEUTICS
• RAPA-501: RAPA THERAPEUTICS
• PRIMEC: NEUROSENSE THERAPEUTICS
• ENGENSIS (VM202): HELIXMITH
• TPN-101: TRANSPOSON THERAPEUTICS
• RNS60: REVALESIO CORPORATION
• ANX005: ANNEXON BIOSCIENCES
• DAZUCORILANT (CORT113176): CORCEPT THERAPEUTICS
• AP-101: AL-S PHARMA
• SAR443820/DNL788: SANOFI/DENALI THERAPEUTICS
• AIT-101 (LAM-002A): ORPHAI THERAPEUTICS
• ULEFNERSEN: IONIS PHARMACEUTICALS
• MN-166: MEDICINOVA
• RNS60: REVALESIO
• QRL-201: QURALIS CORPORATION

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Lou Gehrig's Disease Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.

By Stage
• 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

By Route of Administration
• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

By Molecule Type
• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Scope of the Lou Gehrig's Disease Pipeline Report
• Coverage: Global
• Key Lou Gehrig's Disease Companies: Ionis Pharmaceuticals, 1st Biotherapeutics, Scholar Rock, Revalesio, QurAlis Corporation, Sanofi, MediciNova, Helixmith, Verge Genomics, UCB, AB Science, Alector, GSK, Brainstorm Cell Therapeutics, Denali Therapeutics, AbbVie, Calico Life Sciences, Clene Nanomedicine Biosciences, Seelos Therapeutics, Prilenia Therapeutics, Rapa Therapeutics, Neurosense Therapeutics, Transposon Therapeutics, Annexon Biosciences, Corcept Therapeutics, Al-S Pharma, Orphai Therapeutics, and others.
• Key Lou Gehrig's Disease Pipeline Therapies: Masitinib, Latozinemab (AL001/GSK4527223), Nurown (MSC-NTF Cells), Ulefnersen (ION363), Ibudilast, DNL343, ABBV-CLS-7262, CNM-AU8, SLS-005 (Trehalose), Pridopidine, Rapa-501, Primec, Engensis (VM202), TPN-101, RNS60, ANX005, Dazucorilant (CORT113176), AP-101, SAR443820/DNL788, AIT-101 (LAM-002A), Ulefnersen, MN-166, RNS60, QRL-201, and others.

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Table of Contents
1. Introduction
2. Executive Summary
3. Lou Gehrig's Disease Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Lou Gehrig's Disease Pipeline Therapeutics
6. Lou Gehrig's Disease Pipeline: Late-Stage Products (Phase III)
7. Lou Gehrig's Disease Pipeline: Late-Stage Products (Phase III)
8. Lou Gehrig's Disease Pipeline: Mid-Stage Products (Phase II)
9. Lou Gehrig's Disease Pipeline: Early Stage Products (Phase I)
10. Therapeutic Assessment
11. Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Key Companies
14. Key Products
15. Unmet Needs
16. Market Drivers and Barriers
17. Future Perspectives and Conclusion
18. Analyst Views
19. Appendix

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This release was published on openPR.