Angelman Syndrome Pipeline 2025: Mechanism of Action, Route of Administration, and Clinical Trial Insights Explored by DelveInsight | Ovid Therapeutics, GeneTx Biotherapeutics, PTC Therapeutics

Angelman Syndrome pipeline constitutes 10+ key companies continuously working towards developing 10+ Angelman Syndrome treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

"Angelman Syndrome Pipeline Insight, 2025" report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Angelman Syndrome Market.

The Angelman Syndrome 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.

Some of the key takeaways from the Angelman Syndrome Pipeline Report: https://www.delveinsight.com/sample-request/angelman-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr
• Companies across the globe are diligently working toward developing novel Angelman Syndrome treatment therapies with a considerable amount of success over the years. Angelman Syndrome Key players such as - Taysha Gene Therapies, PTC Therapeutics, GEXVal, Roche, Ionis Pharmaceuticals, Ultragenyx Pharmaceutical, Neuren Pharmaceuticals , and others, are developing therapies for the Angelman Syndrome treatment
• Angelman Syndrome Emerging therapies such as - AAV-mediated UBE3A Gene Replacement therapy, GT-AS, GXV 001, RG 6091, ION582, GTX-102, NNZ-2591, and others are expected to have a significant impact on the Angelman Syndrome market in the coming years.
• In February 2025, Encoded Therapeutics Inc., a clinical-stage biotech company focused on genetic medicines for serious central nervous system (CNS) disorders, announced ongoing progress in its lead clinical candidate, ETX101. The company also highlighted key milestones that have strengthened its research pipeline and expanded its infrastructure to support the full integration of its gene therapy capabilities.
• In January 2025, Dr. Roger Hollis, a project scientist at the University of California, Los Angeles, working in Dr. Donald Kohn's renowned gene therapy lab, has received a $5.8 million translational research grant from the California Institute for Regenerative Medicine (CIRM). The funding will advance the development of a hematopoietic stem cell gene therapy (HSC-GT) aimed at treating Angelman syndrome (AS).
• In January 2025, The first patient has been treated in a Phase 3 clinical trial assessing the safety and effectiveness of GTX-102, an investigational gene therapy by Ultragenyx Pharmaceutical for Angelman syndrome. The global Aspire study (NCT06617429) aims to enroll approximately 120 children and adolescents, ages 4 to 17, with Angelman syndrome due to the complete loss of the maternal UBE3A gene. Recruitment is underway at sites in the U.S., with additional locations planned in Canada, Australia, Japan, and several European countries.
• In November 2024, Encoded Therapeutics Inc., a clinical-stage biotech company focused on genetic medicines for severe central nervous system (CNS) disorders, announced plans to present new preclinical findings on ETX201. This AAV9-based vectorized miRNA therapy is designed to suppress UBE3A-ATS expression and activate paternal UBE3A, offering a potential treatment for Angelman syndrome.
• In November 2024, Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced the Phase 3 study design for ION582 after reaching an agreement with the U.S. Food and Drug Administration (FDA). ION582 is an investigational treatment for Angelman syndrome (AS), a condition that usually manifests in infancy and is marked by significant intellectual disability, speech impairments, and severe motor dysfunction.

Angelman Syndrome Overview
Angelman Syndrome is a rare genetic disorder affecting the nervous system, primarily caused by mutations or deletions in the UBE3A gene on chromosome 15. It is characterized by severe developmental delays, intellectual disability, speech impairment, movement and balance issues, seizures, and a happy demeanor with frequent laughter. Individuals with Angelman Syndrome often have difficulty with coordination and may exhibit hyperactivity and sleep disturbances. While there is no cure, treatment focuses on managing symptoms through therapies, medications, and supportive care.

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Angelman Syndrome Pipeline Therapeutics Assessment
• Angelman Syndrome Assessment by Product Type
• Angelman Syndrome By Stage and Product Type
• Angelman Syndrome Assessment by Route of Administration
• Angelman Syndrome By Stage and Route of Administration
• Angelman Syndrome Assessment by Molecule Type
• Angelman Syndrome by Stage and Molecule Type

DelveInsight's Angelman Syndrome Report covers around 10+ 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 Angelman Syndrome product details are provided in the report. Download the Angelman Syndrome pipeline report to learn more about the emerging Angelman Syndrome therapies
https://www.delveinsight.com/sample-request/angelman-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Emerging Angelman Syndrome Drugs Under Different Phases of Clinical Development Include:
• AAV-mediated UBE3A Gene Replacement therapy: Taysha Gene Therapies
• GT-AS: PTC Therapeutics
• GXV 001: GEXVal
• RG 6091: Roche
• ION582: Ionis Pharmaceuticals
• GTX-102: Ultragenyx Pharmaceutical
• NNZ-2591: Neuren Pharmaceuticals

Angelman Syndrome Pipeline Analysis:
The Angelman Syndrome pipeline report provides insights into
• The report provides detailed insights about companies that are developing therapies for the Angelman Syndrome treatment 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 Angelman Syndrome Treatment.
• Angelman Syndrome key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Angelman Syndrome 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 Angelman Syndrome 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.

Get a Free Sample PDF Report to know more about Angelman Syndrome Pipeline Assessment
https://www.delveinsight.com/sample-request/angelman-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Angelman Syndrome Pipeline Market Drivers
• Improving Healthcare Infrastructure
• Rising investments aimed at research and development activities to extract enhanced and more effective solutions for the treatment of Angelman syndrome

Angelman Syndrome Pipeline Market Barriers
• Lack of awareness regarding the Angelman Syndrome
• Misdiagnosis of the angelman syndrome as cerebral palsy or autism

Scope of Angelman Syndrome Pipeline Drug Insight
• Coverage: Global
• Key Angelman Syndrome Companies: Taysha Gene Therapies, PTC Therapeutics, GEXVal, Roche, Ionis Pharmaceuticals, Ultragenyx Pharmaceutical, Neuren Pharmaceuticals , and others
• Key Angelman Syndrome Therapies: AAV-mediated UBE3A Gene Replacement therapy, GT-AS, GXV 001, RG 6091, ION582, GTX-102, NNZ-2591, and others
• Angelman Syndrome Therapeutic Assessment: Angelman Syndrome current marketed and Angelman Syndrome emerging therapies
• Angelman Syndrome Market Dynamics: Angelman Syndrome market drivers and Angelman Syndrome market barriers

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

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Gaurav Bora
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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

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