Angelman Syndrome Pipeline Drugs Insights Report 2025: Clinical Trials, EMA, PDMA, FDA Approvals, MOA, ROA, NDA, IND, and Companies

DelveInsight's "Angelman Syndrome Pipeline Insight 2025" report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in the Angelman Syndrome pipeline landscape. It covers the Angelman Syndrome Pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Angelman Syndrome Pipeline Therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Angelman Syndrome Pipeline Report

* On 20 October 2025, Ionis Pharmaceuticals Inc. announced a study in people with Angelman syndrome. The study will consist of 4 periods: a screening period of up to 28 days, an approximate 60-week double blind, placebo-controlled treatment period, followed by an approximate 25-month Long-Term Extension (LTE) treatment period, and an approximate 8-month Post-LTE follow-up period.
* DelveInsight's Angelman Syndrome Pipeline report depicts a robust space with 10+ active players working to develop 10+ pipeline therapies for Angelman Syndrome treatment.
* The leading Angelman Syndrome Companies such as Neuren Pharmaceuticals, Ultragenyx Pharmaceutical, Ionis Pharmaceuticals, Roche, GEXVal, PTC Therapeutics, Taysha Gene Therapies, Ovid Therapeutics and others.
* Promising Angelman Syndrome Therapies such as ION582, GTX-102, MVX-220, NNZ-2591, Alogabat, Gaboxadol, Circadin 2/5/10 mg and others.

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The Angelman Syndrome Pipeline Report provides disease overview, pipeline scenario and therapeutic assessment of the key pipeline therapies in this domain. The Angelman Syndrome Pipeline Report also highlights the unmet needs with respect to the Angelman Syndrome.

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 Emerging Drugs Profile

* NNZ-2591: Neuren Pharmaceuticals

NNZ-2591 is an analog of cyclic glycine proline, a peptide (small protein) that exists naturally in the brain and is important for the development of neurons (nerve cells). NNZ-2591 was tested in the ube3a knockout mouse model, which resembles features of Angelman syndrome in humans and includes motor deficits, learning problems and alterations in synaptic connectivity and plasticity. The study compared normal mice and "knockout mice" with a disrupted gene. In the knockout mice, treatment with NNZ-2591 for 6 weeks normalized the deficits in all the tests of anxiety, daily living, sociability, motor performance and cognition as well as eliminating seizures.

* GTX-102: Ultragenyx Pharmaceutical

GTX-102 is an investigational antisense oligonucleotide (ASO) therapy designed to inhibit expression of UBE3A-AS in order to prevent silencing of the paternally inherited allele of the UBE3A gene and reactivate expression of the deficient protein. GTX-102 is delivered as an intrathecal infusion. A Phase 1/2 study evaluating the tolerability and safety of GTX-102 and its effect on all major domains of the AS in pediatric patients is currently ongoing in the U.S., U.K. and Canada.

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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 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.

Angelman Syndrome Companies

Neuren Pharmaceuticals, Ultragenyx Pharmaceutical, Ionis Pharmaceuticals, Roche, GEXVal, PTC Therapeutics, Taysha Gene Therapies, Ovid Therapeutics and others.

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 Products have been categorized under various Molecule types such as,

* Oligonucleotide
* Peptide
* Small molecule

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Scope of the Angelman Syndrome Pipeline Report

* Coverage- Global
* Angelman Syndrome Companies- Neuren Pharmaceuticals, Ultragenyx Pharmaceutical, Ionis Pharmaceuticals, Roche, GEXVal, PTC Therapeutics, Taysha Gene Therapies, Ovid Therapeutics and others.
* Angelman Syndrome Therapies - ION582, GTX-102, MVX-220, NNZ-2591, Alogabat, Gaboxadol, Circadin 2/5/10 mg and others.
* Angelman Syndrome Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
* Angelman Syndrome Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Contents

* Introduction
* Executive Summary
* Angelman Syndrome: Overview
* Pipeline Therapeutics
* Therapeutic Assessment
* Angelman Syndrome- DelveInsight's Analytical Perspective
* Mid Stage Products (Phase II)
* NNZ-2591: Neuren Pharmaceuticals
* Drug profiles in the detailed report.....
* Mid Stage Products (Phase I/II)
* GTX-102: Ultragenyx Pharmaceutical
* Drug profiles in the detailed report.....
* Early Stage Products (Phase I)
* RG 6091: Roche
* Drug profiles in the detailed report.....
* Preclinical and Discovery Stage Products
* GT-AS: PTC Therapeutics
* Drug profiles in the detailed report.....
* Inactive Products
* Angelman Syndrome Key Companies
* Angelman Syndrome Key Products
* Angelman Syndrome- Unmet Needs
* Angelman Syndrome- Market Drivers and Barriers
* Angelman Syndrome- Future Perspectives and Conclusion
* Angelman Syndrome Analyst Views
* Angelman Syndrome Key Companies
* Appendix

About Us

DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.

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