Angelman Syndrome Pipeline Assessment, 2023 Updates: FDA, EMA, and PMDA Approvals, Emerging Drugs, Clinical Trials, Therapeutic Analysis, and Growth Prospects | Ovid Therapeutics, Taysha Gene Therapies, PTC Therapeutics, GEXVal, Roche, Ionis Pharma, Ultra

(Las Vegas, Nevada, United States) As per DelveInsight's assessment, globally, 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, 2023" 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 companies working in the treatment market are Ovid Therapeutics, Taysha Gene Therapies, PTC Therapeutics, GEXVal, Roche, Ionis Pharmaceuticals, Ultragenyx Pharmaceutical, Neuren Pharmaceuticals, and others, are developing therapies for the Angelman Syndrome treatment
• Emerging Angelman Syndrome therapies in the different phases of clinical trials are- OV 882, 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 August 2022, Ultragenyx Pharmaceutical has acquired GeneTx Biotherapeutics, obtaining control of the ongoing GTX-102 clinical program intended for Angelman syndrome. This acquisition follows their collaboration initiated in 2019, where they jointly commenced a Phase I/II clinical trial (NCT04259281) of GTX-102, an investigative gene therapy for Angelman syndrome across various locations in the U.K., Canada, and the U.S. Ultragenyx exercised an exclusive right to purchase GeneTx's shares, securing complete authority over the study and the GTX-102 clinical program.
• In July 2022, Ultragenyx Pharmaceutical Inc. and GeneTx Biotherapeutics LLC shared a progress report on GTX102, a therapy designed for Angelman syndrome (AS). They presented promising interim findings from the ongoing Phase 1/2 trial, focusing on pediatric patients diagnosed with complete maternal UBE3A gene deletion, showing positive results.
• In July 2022, Neuren Pharmaceuticals Limited has commenced an open-label research investigation to assess the safety, tolerability, and pharmacokinetics of oral NNZ-2591 in Angelman Syndrome. This study aims to evaluate these aspects along with efficacy measurements in children and adolescents diagnosed with Angelman syndrome.
• In March 2022, Regulatory approval has been granted to Neuren Pharmaceuticals in both the U.S. and Australia, allowing the initiation of its Phase 2 trial involving NNZ-2591, an experimental treatment intended for Angelman syndrome.

Angelman Syndrome Overview
A rare neuro-genetic condition known as Angelman syndrome (AS) affects one in every 15,000 live births, or 500,000 individuals globally. It is brought on by the UBE3A gene on the mother's 15th chromosome no longer functioning properly.

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Emerging Angelman Syndrome Drugs Under Different Phases of Clinical Development Include:
• OV 882: Ovid Therapeutics
• 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 Route of Administration
Angelman Syndrome pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as
• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Angelman Syndrome Molecule Type
Angelman Syndrome Products have been categorized under various Molecule types, such as
• Oligonucleotide
• Peptide
• Small molecule

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 5+ 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 at:
https://www.delveinsight.com/sample-request/angelman-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Some of the key companies in the Angelman Syndrome Therapeutics Market include:
Key companies developing therapies for Angelman Syndrome are -

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 treatment of Angelman Syndrome 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.

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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 are some of the important factors that are fueling the Angelman Syndrome Market.

Angelman Syndrome Pipeline Market Barriers
• However, lack of awareness regarding the Angelman Syndrome, misdiagnosis of the angelman syndrome as cerebral palsy or autism and other factors are creating obstacles in the Angelman Syndrome Market growth.

Scope of Angelman Syndrome Pipeline Drug Insight
• Coverage: Global
• Key Angelman Syndrome Companies: Ovid Therapeutics, Taysha Gene Therapies, PTC Therapeutics, GEXVal, Roche, Ionis Pharmaceuticals, Ultragenyx Pharmaceutical, Neuren Pharmaceuticals, and others
• Key Angelman Syndrome Therapies: OV 882, 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

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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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About DelveInsight

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