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"Angelman Syndrome Pipeline Insights 2024"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, 2024 ” 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
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
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
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
Download Sample PDF to Explore the Key Offerings of the Report @
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