Angelman Syndrome Pipeline Review, 2024 Updates | Latest FDA, EMA, and PMDA Approvals, Novel and Emerging Therapies, Clinical Trials, and Treatment Outlook | Ovid Therapeutics, GeneTx Biotherapeutics

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.

“Angelman Syndrome Pipeline Insight, 2024“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Angelman Syndrome Market.

Some of the key takeaways from the Angelman Syndrome Pipeline Report:

• 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 November 2023, Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) has reported the successful completion of enrollment and positive initial results from Part 1 of the ongoing HALOS Phase 1/2a trial of ION582 (BIIB121) in Angelman syndrome. The study demonstrated that ION582 was generally well-tolerated, and it exhibited promising trends in electroencephalogram (EEG) activity and early indications of clinical improvement. However, these findings will require confirmation upon the analysis of the complete dataset expected by mid-2024. The results were presented at the FAST (Foundation for Angelman Syndrome Therapeutics) Summit, with the presentation available for reference. Part 1 of the HALOS trial consists of a three-month, open-label, multiple-ascending dose study, while Part 2 involves a long-term extension study evaluating ION582 for an additional 12 months.
• In May 2023, Ultragenyx Pharmaceutical revealed that the US Food and Drug Administration (FDA) has reviewed and approved modifications to the protocol of its Phase I/II trial for GTX-102, aimed at treating children with Angelman syndrome.
• In July 2022, Neuren Pharmaceuticals Limited initiated an open-label study of the safety, tolerability, and pharmacokinetics of oral NNZ-2591 in Angelman Syndrome. It is a study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Angelman syndrome.

Angelman Syndrome Overview

Angelman syndrome is a rare genetic disorder that primarily affects the nervous system, causing developmental disabilities and neurological problems. It is typically characterized by severe intellectual disability, developmental delay, speech impairment, movement and balance issues, seizures, and a characteristic behavioral phenotype that includes frequent laughter and a happy demeanor.

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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 

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.

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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

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 

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Table of Contents

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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