엔젤만 증후군(AS) : 신규 요법, 미충족 수요 및 TPP 인사이트 리포트(2026년)

Angelman Syndrome (AS) Emerging Therapy and TPP Insights

Thelansis's "Angelman Syndrome (AS) Emerging Therapy, with Unmet Needs and TPP Insights Report - 2026" provides a comprehensive analysis of the emerging competitive landscape, unmet needs, target product profiles (TPPs), trial designs, and KOL insights on key emerging therapies and key drug development opportunities in the indication.

Angelman Syndrome (AS) Overview

Angelman Syndrome is a neurogenetic disorder caused by loss of maternal UBE3A expression, impairing ubiquitin-mediated protein degradation and synaptic function. It presents with severe intellectual disability, absent speech, ataxic gait, seizures, and a characteristically happy demeanor. Diagnosis combines methylation analysis, UBE3A sequencing, and chromosomal microarray; seizure burden and functional impairment reflect severity. Seizure management incorporates valproate, levetiracetam, and cannabidiol, the latter offering efficacy in refractory myoclonic episodes with limited cognitive sedation. ASO therapies targeting UBE3A antisense transcripts to restore paternal gene expression are in advanced clinical trials. Regular neurodevelopmental monitoring, augmentative communication, and family-centred support underpin lifelong quality-of-life management.

Key Highlights

• In Germany, total prevalent Angelman syndrome cases are projected to increase from approximately 6,772 in 2025 to 6,896 by 2035.
• Angelman syndrome remains a rare genetic neurodevelopmental disorder associated with severe cognitive and motor impairment.
• Patients experience developmental delays, speech impairment, seizures, and behavioral abnormalities.
• Significant unmet need persists due to limited disease-modifying treatment options.
• Emerging gene therapies and targeted neurological treatments are transforming the future treatment landscape.

Market Overview

• The Italy Angelman syndrome market is projected to grow from approximately $28 MN to $195 MN by 2035.
• Market growth is supported by:
• Increasing development of gene-targeted therapies
• Improved diagnosis and patient identification
• Market expansion is expected to be driven by high-value rare disease treatments.
• Future growth will depend on successful commercialization of disease-modifying and gene replacement therapies.

Insights driven by surveys with physician / key opinion leaders:

• Survey findings are corroborated and enriched by insights from interviews with leading KOLs
• Survey is customized based on client requirements

Deliverables format:

• PowerPoint presentation
• MS Excel

Key business questions answered:

• Detailed emerging competitive landscape
• Pipeline analysis
• Target patients for emerging therapies
• Key companies
• Key mechanism of actions
• Launch date estimates, etc.
• Clinical trial landscape analysis
• Target patient segments
• Trial endpoints
• Trial design
• Recruitment criteria, etc.
• Unmet Needs and Opportunities
• Performance of key current therapies
• Top areas of unmet needs
• Opportunity sizing for key unmet needs
• Target Product Profiles
• Attributes and levels
• Physician likelihood of prescribing
• Expected patient shares
• KOL insights on key emerging therapies
• Level of awareness
• Expected use / line of therapy
• Extent to fulfil key unmet needs
• KOL quotes

Countries Covered

• G8
  • United States
  • EU5
    • France
    • Germany
    • Italy
    • Spain
    • U.K.
  • Japan
  • China

Apart from the G8 Market, adding any additional country data to the dashboard will cost USD 1,750 per country

Companies Mentioned

• Ionis Pharmaceuticals, Inc.
• Ultragenyx Pharmaceutical Inc.
• MavriX Bio, LLC
• Neuren Pharmaceuticals Limited
• Hoffmann-La Roche
• Healx AI

Table of Contents

1. Key Findings and Analyst Commentary

• Key trends: market snapshots, SWOT analysis, commercial benefits and risk, etc.

2. Competitive Landscape

• Current therapies
• Key takeaways
• Dx and Tx journey/algorithm
• Key current therapies - profiles and KOL insights
• Emerging therapies
• Key takeaways
• Dx and Tx journey/algorithm
• Key emerging therapies - profiles and KOL insights

3. Product Attribute Analysis

• Key takeaways
• Scientific attributes
• Commercial attributes
• Product positioning

4. Primary Market Research

• Current treatment landscape
• Key therapies vs. focused patient segment
• Key attributes and benefits
• Futures treatment landscape
• Current challenges
• Unmet needs
• Emerging therapies
• Key therapies vs. focused patient segment
• Key attributes and benefits
• Futures treatment landscape
• Unmet needs and KOL expectations

5. Unmet Need and TPP Analysis

• Top unmet needs and future attainment by emerging therapies
• TPP analysis and KOL expectations

6. Regulatory and Reimbursement Environments (by country and payer insights)

7. Appendix (e.g., bibliography, methodology)