DelveInsight’s, “Spinal Muscular Atrophy Pipeline Insight, 2025,” report provides comprehensive insights about 18+ companies and 20+ pipeline drugs in the Spinal muscular atrophy pipeline landscape. It covers the Spinal Muscular Atrophy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Spinal Muscular Atrophy pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Spinal Muscular Atrophy Pipeline Report

• In July 2025, GeneCradle Inc. announced a study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients. GC101 will be administrated intrathecally. Short-term safety will be evaluated in 52 weeks and enter long-term follow-up study of 5 years at will. Patients will be tested at baseline and followed up at various time points.
• In July 2025, Novartis Pharmaceuticals conducted a Phase III multi-center, single dose (1.2 x 1014 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never ambulatory SMA patients 2 to
• DelveInsight’s Spinal Muscular Atrophy pipeline report depicts a robust space with 18+ active players working to develop 20+ pipeline therapies for Spinal Muscular Atrophy treatment.
• The leading Spinal Muscular Atrophy Companies such as Ionis Pharmaceuticals, Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Theraputics, Voyager Therapeutics, Hoffmann-La Roche, Catalyst Pharmaceuticals, NMD Pharma, Biohaven Pharmaceuticals, CANbridge Pharmaceuticals Inc., Aurimed Pharma, Exicure, Amylon Therapeutics, Amniotics and others.
• Promising Spinal Muscular Atrophy Pipeline Therapies such as OAV101, Risdiplam, Nusinersen, Amifampridine Phosphate, vesemnogene lantuparvovec, Olesoxime, NMD670 and others.

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Spinal Muscular Atrophy Emerging Drugs Profile

• Apitegromab: Scholar Rock

Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans. Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA. The US Food and Drug Administration (FDA) has granted Fast Track (FTD), Orphan Drug (ODD) and Rare Pediatric Disease (RPD) designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations, to apitegromab for the treatment of SMA. Currently, it is in phase III stage of development.

• ACTX-401: Alcyone Therapeutics

ACTX-401 is an AAV9-delivered gene replacement therapy for the treatment of a rare form of Spinal Muscular Atrophy (SMA) called SMA with Respiratory Distress type 1 (SMARD1), and for Charcot-Marie-Tooth type 2S (CMT2S). SMARD1 and CMT2S are autosomal recessive genetic disorders caused by mutations in the gene that produces immunoglobulin mu-binding protein 2 (IGHMBP2) and are part of a larger family of disorders called IGHMBP2-related disorders (IRDs). ACTX-401 delivers a functional copy of the IGHMBP2 gene to patients, restoring expression of functional IGHMBP2. ACTX-401 clinical success in SMARD1 and CMT2S could lead to applicability to other IRDs in the future. Currently, it is being investigated in Phase I/II stage of development for the treatment of spinal muscular atrophy.

The Spinal Muscular Atrophy Pipeline Report provides insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Spinal Muscular Atrophy with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Spinal Muscular Atrophy Treatment.
• Spinal Muscular Atrophy Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Spinal Muscular Atrophy 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.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Spinal Muscular Atrophy market.

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Spinal Muscular Atrophy Companies

Ionis Pharmaceuticals, Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Theraputics, Voyager Therapeutics, Hoffmann-La Roche, Catalyst Pharmaceuticals, NMD Pharma, Biohaven Pharmaceuticals, CANbridge Pharmaceuticals Inc., Aurimed Pharma, Exicure, Amylon Therapeutics, Amniotics and others.

Spinal muscular atrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical.

Spinal Muscular Atrophy Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

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Scope of the Spinal Muscular Atrophy Pipeline Report

• Coverage- Global
• Spinal Muscular Atrophy Companies- Ionis Pharmaceuticals, Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Theraputics, Voyager Therapeutics, Hoffmann-La Roche, Catalyst Pharmaceuticals, NMD Pharma, Biohaven Pharmaceuticals, CANbridge Pharmaceuticals Inc., Aurimed Pharma, Exicure, Amylon Therapeutics, Amniotics and others.
• Spinal Muscular Atrophy Pipeline Therapies- OAV101, Risdiplam, Nusinersen, Amifampridine Phosphate, vesemnogene lantuparvovec, Olesoxime, NMD670 and others.
• Spinal Muscular Atrophy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Spinal Muscular Atrophy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

For a detailed overview of our latest research findings and future plans, read the full details of Spinal Muscular Atrophy Pipeline on our website @ Spinal Muscular Atrophy Emerging Drugs and Companies

Table of Contents

1. Introduction
2. Executive Summary
3. Spinal muscular atrophy: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Spinal muscular atrophy – DelveInsight’s Analytical Perspective
7. Late Stage Products (Phase III)
8. Apitegromab: Scholar Rock
9. Drug profiles in the detailed report…..
10. Mid Stage Products (Phase II)
11. Branaplam: Novartis
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I/II)
14. ACTX 401: Alcyone Therapeutics
15. Drug profiles in the detailed report….
16. Inactive Products
17. Spinal muscular atrophy- Unmet Needs
18. Spinal muscular atrophy- Market Drivers and Barriers
19. 19. Appendix

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