Angelman Syndrome Market Outlook 2025-2035:
The 7 major angelman syndrome markets reached USD 109.5 Million in 2024. As the market continues to grow, looking forward, the 7MM are expected to touch USD 172.8 Million by 2035 at a CAGR of 4.23% over the forecast period 2025-2035. The angelman syndrome (AS) market is expected to expand due to growing awareness and diagnosis rates and progress in genetic research. Recent advances in gene therapy and targeted treatment will give AS sufferers more reasons for optimism regarding its potential control. The increase in healthcare facilities and specialized centers for genetic disorders is anticipated to drive market expansion. Moreover, increasing backing from governmental programs and non-profit entities aimed at AS awareness and research propels the market forward. The launch of novel therapeutic products designed for Angelman Syndrome patients is expected to enhance treatment alternatives, driving market growth.
Advancements in Genetic and Molecular Research: Driving the Angelman Syndrome Market.
Angelman syndrome is an extremely rare genetic condition resulting from a malfunction of the UBE3A gene in chromosome 15. This rare disorder has faced immense management issues until very recently. Nevertheless, the rapidly emerging knowledge base of gene therapy, coupled with further insight into the molecular pathophysiology driving this condition, promises to present novel therapeutic prospects. The most exciting innovation is the use of gene-editing tools like CRISPR-Cas9, which holds promise to directly address the genetic mutations that cause Angelman syndrome. Continued studies on the UBE3A gene have led to the exploration of treatments designed to restore its function in those affected by the condition. The treatments that focus on RNA, such as antisense oligonucleotides, are also being investigated for their potential to elevate the expression of the UBE3A gene within the brain. First clinical trials of these treatments have shown very promising results in animal studies, and human trials have just started to be organized. In addition to these efforts, advances in molecular diagnostics are allowing earlier, more accurate diagnosis of Angelman syndrome, and interventions become more timely and effective, thus improving the outcome of the patient. Moreover, new information regarding the molecular pathways modified by mutations in UBE3A could bring to light potential biomarkers, assuming that these should play a crucial role in a step forward, bringing about more tailored and individualized treatments. These findings, collectively, lead to a more efficient, tailored treatment approach that holds out the promise for better management of this challenging genetic disorder.
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Development of Novel Therapies and Pharmacological Treatments: Contributing to Market Expansion
The advancement of new therapies and drug treatments for Angelman Syndrome is a crucial element driving market growth in this treatment sector. Angelman Syndrome, an uncommon neurodevelopmental condition, has experienced notable advancements in recent years owing to improvements in genetic studies and medication development. Current clinical trials focused on the genetic underlying factors, like treatments designed to reactivate the silenced UBE3A gene, are crucial for broadening treatment alternatives. These groundbreaking therapies are anticipated to target the primary neurological issues, such as developmental delays, motor challenges, and seizures. Moreover, the arrival of gene therapies, RNA-driven treatments, and small molecules aimed at fixing genetic abnormalities is speeding up market expansion. The growing attention of biopharmaceutical firms on uncommon genetic diseases is boosting investments, while partnerships between research organizations and pharmaceutical companies are accelerating the speed of drug development. Furthermore, developments in precision medicine, customized to personal genetic profiles, are expected to improve treatment effectiveness. Consequently, these advancements are set to establish a strong market featuring various treatment choices, enhancing the quality of life for individuals with Angelman Syndrome and broadening market prospects in the healthcare industry.
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Emerging Therapies in Angelman Syndrome Market
GTX 102: Ultragenyx Pharmaceutical
GTX-102 is an investigational treatment that uses antisense oligonucleotides (ASOs) delivered through an intrathecal injection. Its purpose is to target and block the UBE3A antisense transcript (UBE3A-AS), which stops the silencing of the paternal UBE3A gene, effectively reactivating the production of the missing protein. This therapy has received several accolades, such as Orphan Drug and Rare Pediatric Disease designations from the FDA, Fast Track status from the FDA, and Orphan Designation and PRIME status from the EMA.
NNZ-2591: Neuren Pharmaceuticals
NNZ-2591 is a clinical trial treatment in development for Angelman syndrome, a rare genetic condition that leads to developmental delays, speech challenges, and motor skill issues. It aims to address the root cause of the disorder by adjusting the UBE3A gene, which is usually inactive in individuals with Angelman syndrome. NNZ-2591 aims to reactivate the expression of this gene and enhance cognitive and motor abilities in those impacted. Neuren has received Orphan Drug designation from the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for NNZ-2591 aimed at treating Angelman syndrome.
ION 582: Ionis Pharmaceuticals
ION582 is an antisense therapy, blocking the production of the UBE3A antisense RNA, UBE3A-ATS, with the intention of increasing UBE3A protein production. It is one such drug used in the potential treatment of Angelman syndrome, abbreviated as AS. In 2022, ION582 received the Orphan Drug and Rare Pediatric designations by the U.S. Food and Drug Administration (FDA).
Drug Name | Company Name | MOA | ROA |
GTX 102 | Ultragenyx Pharmaceutical | Gene silencing; UBE3A protein expression stimulants | Intrathecal |
NNZ-2591 | Neuren Pharmaceuticals | Neuropeptide receptor modulators; Synaptic transmission modulators | Oral |
ION 582 | Ionis Pharmaceuticals | UBE3A protein expression stimulants | Intrathecal |
Detailed list of emerging therapies in Angelman Syndrome is provided in the final report…
Leading Companies in the Angelman Syndrome Market:
The market research report by IMARC encompasses a comprehensive analysis of the competitive landscape in the market. Across the global Angelman Syndrome market, several leading companies are at the forefront of developing integrated platforms to enhance the management of Angelman Syndrome. The major players include Neuren Pharmaceuticals, Ultragenyx Pharmaceutical, and others. These companies are driving innovation in the Angelman Syndrome market through continuous research, diagnostic tools, and expanding their product offerings to meet the growing demand for Angelman Syndrome.
In August 2024, Neuren released the key findings from its Phase 2 trial of NNZ-2591 in children diagnosed with Angelman syndrome (AS). The trial demonstrated significant progress, with notable improvements observed in both clinician and caregiver global efficacy measures tailored for AS. These improvements were evident in clinically significant areas of the condition.
Key Players in Angelman Syndrome Market:
The key players in the Angelman Syndrome market who are in different phases of developing different therapies are Ultragenyx Pharmaceutical, Neuren Pharmaceuticals, Ionis Pharmaceuticals, and Others.
Regional Analysis:
The major markets for Angelman Syndrome include the United States, Germany, France, the United Kingdom, Italy, Spain, and Japan. According to projections by IMARC, the United States has the largest patient pool for Angelman Syndrome while also representing the biggest market for its treatment. Exciting new therapies for Angelman Syndrome are being developed, showcasing notable advancements in gene therapy, RNA modulation, and enzyme replacement approaches. Furthermore, studies are exploring the application of small molecules and CRISPR-based methods to rectify the genetic malfunction and reinstate typical gene activity. These groundbreaking treatments have the capability to enhance symptom management and elevate the quality of life for those with Angelman Syndrome. At present, these treatments are in different phases of clinical trials, offering hope for future medical progress.
Recent advancements in Angelman syndrome research have focused on gene therapy, especially attempts to restore the UBE3A gene that is either missing or not functioning in those affected. Clinical research is exploring the application of antisense oligonucleotides (ASOs) to activate the dormant gene, showing encouraging indications of enhancements in cognitive and motor abilities. Moreover, scientists are investigating novel drug therapies that focus on particular molecular pathways related to brain development, with some initial trials showing promising outcomes. These advancements bring hope for improved treatments down the line.
Recent Developments in Angelman Syndrome Market:
· In December 2024, Ultragenyx Pharmaceutical reported an important milestone that the first patient has begun treatment in their Phase 3 Aspire study (NCT06617429). This is a pivotal trial intended to assess how safe and effective GTX-102, the company’s investigational antisense therapy, may be for people with Angelman syndrome.
· In November 2024, During the Foundation for Angelman Syndrome Therapeutics (FAST) Global Science Summit, Ultragenyx presented positive Phase 1/2 trial results, where improvements were recorded in various areas such as cognitive functions, receptive communication, behavioral skills, gross motor functions, and quality of sleep.
· In July 2024, Ionis Pharmaceuticals reported positive results on the multiple ascending dose (MAD) phase which was successfully finished in its phase 1/2 open label trial of ION582 administered to patients who have Angelman syndrome (AS). The results reveal consistent and highly promising clinical benefits across different function areas, with communication, cognitive, and motor skills.
Key information covered in the report.
Base Year: 2024
Historical Period: 2019-2024
Market Forecast: 2025-2035
Countries Covered
- United States
- Germany
- France
- United Kingdom
- Italy
- Spain
- Japan
Analysis Covered Across Each Country
- Historical, current, and future epidemiology scenario
- Historical, current, and future performance of the Angelman Syndrome market
- Historical, current, and future performance of various therapeutic categories in the market
- Sales of various drugs across the Angelman Syndrome market
- Reimbursement scenario in the market
- In-market and pipeline drugs
Competitive Landscape:
This report offers a comprehensive analysis of current Angelman Syndrome marketed drugs and late-stage pipeline drugs.
In-Market Drugs
- Drug Overview
- Mechanism of Action
- Regulatory Status
- Clinical Trial Results
- Drug Uptake and Market Performance
Late-Stage Pipeline Drugs
- Drug Overview
- Mechanism of Action
- Regulatory Status
- Clinical Trial Results
- Drug Uptake and Market Performance
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