Angelman Syndrome Overview

Angelman syndrome (AS) is a rare neuro-genetic disorder that occurs in one in 15,000 live births or 500,000 people worldwide. It is caused by a loss of function of the UBE3A gene in the 15th chromosome derived from the mother. Angelman syndrome (AS) is characterized by severe developmental delay or intellectual disability, severe speech impairment, gait ataxia and/or tremulousness of the limbs, and unique behavior with an apparent happy demeanor that includes frequent laughing, smiling, and excitability.

“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:

• 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 Pharmaceuticals, 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.   
• GeneTX Biotherapeutics, LLC in collaboration with Ultragenyx Pharmaceutical Inc Initiated a Phase I/II open-label, multiple-dose, dose-escalating clinical trial of the safety and tolerability of GTX-102in pediatric patients with Angelman Syndrome
• In March 2022, Neuren Pharmaceuticals received regulatory approval in the U.S. and Australia to start its Phase 2 trial of NNZ-2591, an investigational therapy for 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 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 15+ 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

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

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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.

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

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 Pharmaceuticals, 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

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