Spinal Muscular Atrophy Pipeline Therapeutics Assessment Report 2024 (Updated)

DelveInsight's, "Spinal Muscular Atrophy Pipeline Insight 2024" report provides comprehensive insights about 18+ companies and 20+ pipeline drugs in the Spinal muscular atrophy 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.

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Key Takeaways from the Spinal Muscular Atrophy Pipeline Report
• In October 2024:- Hoffmann-La Roche- This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children < 2 years of age genetically diagnosed with SMA.
• In October 2024:- Lantu Biopharma- Study duration per participant is approximately 25 months including an approximately 30-day screening/baseline period, an approximately 24-month study observation period including 1 treatment day, and an approximately 24-month follow-up period. Patients will be tested at baseline and return for follow-up visits twice a week through the first month post dose, and followed by visits at months 2, 3, 6 12, 18 and 24 post infusion. Unscheduled visits may occur if the investigator determines that they are necessary.
• In October 2024:- Genentech Inc.- A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S. Package Insert (USPI) in adult and pediatric participants with spinal muscular atrophy (SMA). In this study, participants will be followed for up to 5 years from enrollment or until withdrawal of consent, loss to follow-up, or death. Participants who discontinue risdiplam may still remain in the study, if they agree to continue participating in the follow-up assessments.
• 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 Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Therapeutics, 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 Therapies such as Risdiplam, Nusinersen, Vesemnogene Iantuparvovec, OAV101, NMD670, GC101, Apitegromab, 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.

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Spinal muscular atrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration
• 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- Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Therapeutics, 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 Therapies- Risdiplam, Nusinersen, Vesemnogene Iantuparvovec, OAV101, NMD670, GC101, Apitegromab, 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 Drugs and Companies- https://www.delveinsight.com/sample-request/spinal-muscular-atrophy-sma-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

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

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