Spinal Muscular Atrophy Pipeline Insights 2024: Therapies, Clinical Trials, MOA, ROA by DelveInsight | Genentech Inc, Chugai Pharma, Cytokinetics Inc., Ionis Pharma, Genzyme Corporation, Novartis, Astellas Pharma Inc., Takeda Pharma

(Las Vegas, Nevada, United States) As per DelveInsight's assessment, globally, Spinal Muscular Atrophy pipeline constitutes 18+ key companies continuously working towards developing 20+ Spinal Muscular Atrophy treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

The Spinal Muscular Atrophy Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.

"Spinal Muscular Atrophy Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Spinal Muscular Atrophy Market.

Some of the key takeaways from the Spinal Muscular Atrophy Pipeline Report:
https://www.delveinsight.com/sample-request/spinal-muscular-atrophy-sma-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr
• Companies across the globe are diligently working toward developing novel Spinal Muscular Atrophy treatment therapies with a considerable amount of success over the years.
• Spinal Muscular Atrophy companies working in the treatment market are Skyline Therapeutics, Exegenesis Bio, Biogen, GeneCradle Inc., Hangzhou Jiayin Biotech Ltd, Scholar Rock, Inc., Biohaven Pharmaceuticals, Inc, and others, are developing therapies for the Spinal Muscular Atrophy treatment
• Emerging Spinal Muscular Atrophy therapies in the different phases of clinical trials are- SKG 0201, Spinal muscular atrophy gene therapy, BIIB115, GC101, EXG001-307, Apitegromab, Talditercept alfa, and others are expected to have a significant impact on the Spinal Muscular Atrophy market in the coming years.
• In April 2024, Exegenesis Bio, a fast-growing global genetic medicines company, has announced the presentation of clinical efficacy and safety data from its EXG001-307 Phase 1/2 clinical trial for Spinal Muscular Atrophy (SMA) Type I at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Baltimore.
• In January 2024, Alcyone Therapeutics Inc. (Alcyone), a biotechnology company at the forefront of precision genetic therapies and delivery solutions for intricate neurological conditions, has reported the initiation of the PIERRE pivotal study. This study aims to assess the safety and efficacy of the ThecaFlex DRx System (ThecaFlex), a subcutaneous port and intrathecal catheter system designed for repeat intrathecal access, cerebrospinal fluid (CSF) aspiration, and the administration of SPINRAZA® (nusinersen) in individuals with spinal muscular atrophy (SMA). The inaugural patient in the PIERRE study underwent implantation at Texas Children's Hospital in Houston, Texas, under the supervision of Investigator David Bauer, MD, MPH
• In January 2024, Avenue Therapeutics Inc. (Nasdaq: ATXI), a specialty pharmaceutical company dedicated to advancing therapies for neurologic diseases, has successfully enrolled all participants in the Phase 1b/2a clinical trial of AJ201 targeting the treatment of spinal and bulbar muscular atrophy (SBMA), also recognized as Kennedy's Disease. AJ201 is presently the primary drug candidate under evaluation for SBMA in clinical trials, with the release of key findings expected in the second quarter of 2024
• In February 2023, Biohaven Ltd. announced that it received Fast Track designation from the U.S. Food and DrugAdministration (FDA) for taldefgrobep alfa, a novel anti-myostatin adnectin, for the treatment of spinal muscularatrophy (SMA). Biohaven also previously received orphan drug designation from the FDA for taldefgrobep in thetreatment of SMA.

Spinal Muscular Atrophy Overview
Spinal Muscular Atrophy (SMA) is a genetic neuromuscular disorder characterized by the progressive degeneration of motor neurons in the spinal cord and brainstem. Motor neurons are nerve cells responsible for controlling voluntary muscle movement. When these motor neurons degenerate, muscles weaken and atrophy, leading to difficulties with movement, muscle weakness, and potentially life-threatening respiratory problems.

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Emerging Spinal Muscular Atrophy Drugs Under Different Phases of Clinical Development Include:
• SKG 0201: Skyline Therapeutics
• Spinal muscular atrophy gene therapy: Exegenesis Bio
• BIIB115: Biogen
• GC101: GeneCradle Inc.
• EXG001-307: Hangzhou Jiayin Biotech Ltd
• Apitegromab: Scholar Rock, Inc.
• Talditercept alfa: Biohaven Pharmaceuticals, Inc

Spinal Muscular Atrophy Route of Administration
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 Molecule Type
Spinal Muscular Atrophy Products have been categorized under various Molecule types, such as
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Spinal Muscular Atrophy Pipeline Therapeutics Assessment
• Spinal Muscular Atrophy Assessment by Product Type
• Spinal Muscular Atrophy By Stage and Product Type
• Spinal Muscular Atrophy Assessment by Route of Administration
• Spinal Muscular Atrophy By Stage and Route of Administration
• Spinal Muscular Atrophy Assessment by Molecule Type
• Spinal Muscular Atrophy by Stage and Molecule Type

DelveInsight's Spinal Muscular Atrophy 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)
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• Route of Administration

Further Spinal Muscular Atrophy product details are provided in the report. Download the Spinal Muscular Atrophy pipeline report to learn more about the emerging Spinal Muscular Atrophy therapies
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Some of the key companies in the Spinal Muscular Atrophy Therapeutics Market include:
Key companies developing therapies for Spinal Muscular Atrophy are - Genentech Inc, Chugai Pharmaceutical, Cytokinetics Inc., Ionis Pharmaceuticals Inc., Genzyme Corporation, Novartis International AG, Astellas Pharma Inc., Takeda Pharmaceutical Company Limited, PerkinElmer Inc., Pfizer Inc., UW Health, Novo Nordisk A/S, Cure SMA, Boehringer Ingelheim Gmbh, Biogen Inc, F. Hoffmann-La Roche AG, Catalyst Pharmaceutical, Salarius Pharmaceuticals Inc, Regeneron Pharmaceuticals Inc, Isis Pharmaceuticals Inc., PTC Therapeutics, Natera Inc, AstraZeneca PLC, and others.

Spinal Muscular Atrophy Pipeline Analysis:
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 key 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.
The report is built using data and information traced from the researcher's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.

Download Sample PDF Report to know more about Spinal Muscular Atrophy drugs and therapies
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Spinal Muscular Atrophy Pipeline Market Drivers
• Increase in prevalence of Spinal Muscular Atrophy, increasing number of clinical trials and government funding for R&D are some of the important factors that are fueling the Spinal Muscular Atrophy Market.

Spinal Muscular Atrophy Pipeline Market Barriers
• However, lack of public knowledge of this rare medical condition, cost associated with the treatment and other factors are creating obstacles in the Spinal Muscular Atrophy Market growth.

Scope of Spinal Muscular Atrophy Pipeline Drug Insight
• Coverage: Global
• Key Spinal Muscular Atrophy Companies: Skyline Therapeutics, Exegenesis Bio, Biogen, GeneCradle Inc., Hangzhou Jiayin Biotech Ltd, Scholar Rock, Inc., Biohaven Pharmaceuticals, Inc, and others
• Key Spinal Muscular Atrophy Therapies: SKG 0201, Spinal muscular atrophy gene therapy, BIIB115, GC101, EXG001-307, Apitegromab, Talditercept alfa, and others
• Spinal Muscular Atrophy Therapeutic Assessment: Spinal Muscular Atrophy current marketed and Spinal Muscular Atrophy emerging therapies
• Spinal Muscular Atrophy Market Dynamics: Spinal Muscular Atrophy market drivers and Spinal Muscular Atrophy market barriers

Request for Sample PDF Report for Spinal Muscular Atrophy Pipeline Assessment and clinical trials
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Table of Contents
1. Spinal Muscular Atrophy Report Introduction
2. Spinal Muscular Atrophy Executive Summary
3. Spinal Muscular Atrophy Overview
4. Spinal Muscular Atrophy- Analytical Perspective In-depth Commercial Assessment
5. Spinal Muscular Atrophy Pipeline Therapeutics
6. Spinal Muscular Atrophy Late Stage Products (Phase II/III)
7. Spinal Muscular Atrophy Mid Stage Products (Phase II)
8. Spinal Muscular Atrophy Early Stage Products (Phase I)
9. Spinal Muscular Atrophy Preclinical Stage Products
10. Spinal Muscular Atrophy Therapeutics Assessment
11. Spinal Muscular Atrophy Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Spinal Muscular Atrophy Key Companies
14. Spinal Muscular Atrophy Key Products
15. Spinal Muscular Atrophy Unmet Needs
16 . Spinal Muscular Atrophy Market Drivers and Barriers
17. Spinal Muscular Atrophy Future Perspectives and Conclusion
18. Spinal Muscular Atrophy Analyst Views
19. Appendix
20. About DelveInsight

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