Angelman Syndrome – Pipeline Review, 2025

The global Angelman Syndrome pipeline has evolved and grown with rising awareness and diagnosis rates and progress in genetic research and growing healthcare facilities for genetic disorders. Angelman syndrome (AS) is a neuro-genetic disorder, which is a rare disease, which occurs in one in 15,000 childbirths globally. The cause of this disorder is deficiency of the UBE3A gene in the 15th chromosome that comes from the mother. Intellectual disability and acute developmental delay is the sign of AS disorder. These symptoms include tremulousness of the limbs and communication disorder.  Patients may develop unique behavior with a noticeable happy demeanor such as frequent laughing, smiling, and excitability. Cognitive disabilities are visible at about age six months and the unique clinical features of AS are evident after age of one year.

The diagnosis of AS is recognized in a proband who convenes the consensus clinical diagnostic criteria and/or who has findings on molecular genetic testing. Occupational treatment, physical therapy, and speech therapy are some practical treatments. Thus, various companies and academics are performing clinical research for identifying challenges and finding opportunities to influence Angelman Syndrome drug R&D activities for drug development. Various therapies are under development, emphasizing improvement in treatment by novel approach with individualization and flexibility in school settings.

Angelman Syndrome Pipeline Insights 2025: Report Scope

Comprising 10+ companies and 10+ pipeline drugs, Fortune Business Insights has released its report Angelman Syndrome Pipeline Insights 2025”. It presents an entire pipeline insight regarding clinical research and non-clinical stages of drug manufacturing and develops new treatments of Angelman Syndrome. This report also presents pipeline reviews, which includes phase 1, phase 2, and phase 3 trials of drugs development and treatments. The drugs under development are on the basis of mechanism of action of the drugs, commercial assessment and clinical assessment. In addition, major regions of geography such as North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa are also covered in this report.

Reasons to Buy this Report

• Develop effective growth strategies induced by complete overview of the R&D activity and Angelman Syndrome pipeline products for disease therapies.
• Identify new companies in the market based on the pipeline products.
• Identify development plans to counteract the emergency of the latest players in the market.
• Identify the focus of R&D activities of major companies for Angelman Syndrome treatments.
• Identify possibility of collaborations and acquisition and merger based on licensing activities related to the treatment.
• Analyse the causes for inactive and discontinued products to propose changes in R&D activities, if required.

Know Answers to Your Questions

• How many companies are manufacturing Angelman Syndrome drugs?
• What are the numbers of drugs for Angelman Syndrome developed by each company?
• What are the numbers of new drugs in Phase 2 and Phase 3 trials stage of development for the management of Angelman Syndrome?
• What are the main alliances (Industry–Industry, Industry–Academia), mergers and acquisitions, licensing activities connected to the Angelman Syndrome therapeutics?
• What are the latest trends, novel technologies, and drug types developed to beat the limitation of existing therapies?
• What are the clinical studies going on for Angelman Syndrome and their position?
• What are the major designations that have been approved for emerging drugs?

Report Methodology

• These pipeline reports are created by the assessment of several data that is mainly collected from credible desk research sources. In addition, secondary data has also been collected that is supplemented by conducting interviews with expert opinion leaders.

• The desk research sources include press releases and investor presentations of companies and annual reports, white papers, websites, news articles. These sources also comprise databases from global and regional clinical trials, industrial associations published reports, as well as articles/reports published on databases from NCBI, internal databases, and ResearchGate.

Clinical Trial Insights

The rising emphasis on requirement for needed drugs and effective therapies for disease prevention and curing are impelling the demand for development of drug pipelines which is pushing R&D activities. There are numerous healthcare institutes, players connected with the medical sectors, and diverse research organizations are conducting clinical trials for developing new drugs for treatment of Angelman Syndrome. Government bodies have increased emphasis on improving infrastructure for healthcares supporting the worldwide clinical trials scenario in the field of Angelman Syndrome treatment.

Angelman Syndrome Pipeline Overview

Escalating awareness activities and encouraging government policies has encouraged development initiatives for products. Diverse new drug candidates are under preclinical as well as discovery stages along with their Phase 1, Phase 2, and Phase 3 trials. There are additional developments in the market comprising collaborations, merger, and licensing as well as systematic therapeutic assessment for emerging drugs. Key healthcare is also keen to look for drug approvals from regulatory bodies to bring candidates to the market.

A short explanation of Angelman Syndrome emerging drugs in pipeline:

NNZ-2591: Neuren Pharmaceuticals

NNZ-2591 is an analog of cyclic glycine proline. It is a peptide (small protein) that exists naturally in the brain and is vital for the development of neurons (nerve cells). In its phase 3 trials, it is analyzed that treatments with NNZ-2591 for 6 weeks stabilize the deficit in all the tests such as motor performance, anxiety, sociability, daily living, and cognition and reduce seizures.

GTX-102: Ultragenyx Pharmaceutical

GTX-102 is an ASO (investigational antisense oligonucleotide) therapy. It is created to reduce expression of UBE3A-AS to avoid silencing of the UBE3A gene allele, which is paternally inherited, and reactivate expression of the scarce protein. The Phase 1 trial and Phase 2 trial studies have evaluated the tolerability and safety of GTX-102 and its effect on all major domains of the AS in pediatric patients is presently ongoing in the U.K. U.S., and Canada.