Angelman Syndrome Pipeline Assessment, 2023 Updates | In-depth Insights into the Clinical Trials, Emerging Drugs, Latest FDA, EMA, and PMDA Approvals, Treatment Outlook, and Key Companies

Angelman Syndrome Pipeline Assessment, 2023 Updates | In-depth Insights into the Clinical Trials, Emerging Drugs, Latest FDA, EMA, and PMDA Approvals, Treatment Outlook, and Key Companies
Delveinsight Business Research LLP
As per DelveInsight’s assessment, globally, about 15+ key pharma and biotech companies are working on 15+ pipeline drugs in the Angelman Syndrome therapeutics landscape based on different Routes of Administration (ROA), Mechanism of Action (MOA), and molecule types. Several of the therapies are in the advanced stages of clinical development and are expected to launch in the coming years.

Angelman Syndrome therapeutics market is anticipated to evolve immensely in the coming years owing to improved disease awareness, increasing prevalence, improved diagnosis, and the launch of emerging pipeline therapies. Some of the drugs in the pipeline include GTX-102 (GeneTx Biotherapeutics/Ultragenyx Pharmaceutical), ION582 (Ionis Pharmaceuticals/Biogen), among others.

Angelman Syndrome Pipeline Insight, 2023” report by DelveInsight outlines a comprehensive assessment of the present clinical/non-clinical development activities 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.

Angelman Syndrome Pipeline Analysis

Angelman Syndrome Pipeline Analysis

The report provides insights into: 

  • The report provides detailed insights into the emerging therapies for the treatment of Angelman Syndrome and the aggregate therapies developed by major pharma companies.

  • It accesses the different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of clinical development.

  • It outlines the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

  • The report evaluates the drugs that are 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. 

  • It navigates the major collaborations (company-company collaborations and company-academia collaborations), licensing agreements, financing details, data presentation by the pharma giants, and regulatory approval in 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.  

Analysis of Emerging Therapies by Phases

The report covers the emerging products under different phases of clinical development like –

  • Late stage products (Phase III)

  • Mid-stage products (Phase II)

  • Early-stage products (Phase I) 

  • Pre-clinical and Discovery stage candidates

  • Discontinued & Inactive candidates


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 

  • Intrathecal 


Molecule Type

Products have been categorized under various Molecule types, such as

  • Small molecules

  • Gene therapies


Mechanism of Action of the Emerging Pipeline Therapies

  • Neuropeptide receptor modulator

  • RNA interference

  • Undefined mechanism

  • UBE3A protein expression stimulant

  • Gene transference

Learn How the Ongoing Clinical & Commercial Activities will Affect the Angelman Syndrome Therapeutic Segment @


Angelman Syndrome Therapeutics Landscape

Several major pharma and biotech companies are developing therapies for Angelman Syndrome. Currently, GeneTX Biotherapeutics is leading the therapeutics market with its Angelman Syndrome drug candidates in the mid to advanced stage of clinical development.

The Leading Companies in the Angelman Syndrome Therapeutics Market Include:

  • GeneTX Biotherapeutics, LLC

  • Ultragenyx Pharmaceutical Inc

  • Hoffmann-La Roche

  • Biogen

  • Ionis Pharmaceuticals

  • Neuren Pharmaceuticals

  • Anavex Life Science

  • Ovid Therapeutics

  • PTC Therapeutics

  • StrideBio

  • Aingeal

And Many Others

Angelman Syndrome Drugs Covered in the Report Include:

  • GT-AS: PTC Therapeutics

  • GTX-102: GeneTx Biotherapeutics / Ultragenyx Pharmaceutical Inc.

  • GXV 001: GEXVal

  • ION582: Ionis Pharmaceuticals

  • NNZ-2591: Neuren Pharmaceuticals

  • RG 6091: Roche

And Many More

Request the Sample PDF to Get a Better Understanding of the Emerging Drugs and Key Companies @


Table of Content (TOC)

1. Report Introduction

2. Executive Summary

3. Angelman Syndrome Current Treatment Patterns

4. Angelman Syndrome – DelveInsight’s Analytical Perspective

5. Therapeutic Assessment

6. Angelman Syndrome Late Stage Products (Phase-III)

7. Angelman Syndrome Mid-Stage Products (Phase-II)

8. Angelman Syndrome Early Stage Products (Phase-I)

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Angelman Syndrome Discontinued Products

13. Angelman Syndrome Product Profiles

14. Key Companies in the Angelman Syndrome Market

15. Key Products in the Angelman Syndrome Therapeutics Segment

16. Dormant and Discontinued Products

17. Angelman Syndrome Unmet Needs

18. Angelman Syndrome Future Perspectives

19. Angelman Syndrome Analyst Review  

20. Appendix

21. Report Methodology

*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.

Download Sample PDF to Explore the Key Offerings of the Report @


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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.

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