Dublin, May 08, 2025 (GLOBE NEWSWIRE) -- The "Rett Syndrome - Pipeline Insight, 2025" clinical trials has been added to ResearchAndMarkets.com's offering.
This report provides comprehensive insights about 20+ companies and 20+ pipeline drugs in Rett Syndrome pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Report Highlights
The companies and academics are working to assess challenges and seek opportunities that could influence Rett Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Rett Syndrome.
Rett Syndrome Emerging Drugs Chapters
This segment of the Rett Syndrome report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Rett Syndrome Emerging Drugs
Blarcamesine: Anavex Life Sciences Corp
Blarcamesine is a small molecule, orally administered drug that restores cellular homeostasis by targeting SIMGAR1 and muscarinic receptors that is pivotal to restoring cellular homeostasis and promoting neuroplasticity. The drug candidate has received Fast Track designation, Rare Pediatric Disease designation and Orphan Drug designation from the FDA. Currently, the drug is in Phase III stage of its development for the treatment of Rett syndrome.
TSHA-102: Taysha GTx
TSHA-102 is a self-complementary intrathecal delivered AAV9 gene transfer therapy in clinical evaluation. TSHA-102 is constructed from a neuronal specific promoter, MECP2, a gene essential for neuronal and synaptic function in the brain. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform designed to regulate cellular MECP2 expression. TSHA-102 has received Orphan Drug and Rare Pediatric Disease designations from the United States (U.S.) Food and Drug Administration (FDA) and has been granted Orphan Drug designation from the European Commission. Currently, the drug is in Phase I/II stage of its development for the treatment of Rett syndrome.
NGN-401: Neurogene
NGN-401 is an investigational AAV9 gene therapy being developed as a one line treatment for Rett syndrome. It is the first clinical candidate to deliver the full-length human MECP2 gene under the control of Neurogene's EXACT technology. The drug is currently being evaluated under Phase I/II clinical trial for the treatment of patients with Rett Syndrome.
Rett Syndrome: Therapeutic Assessment
This segment of the report provides insights about the different Rett Syndrome drugs segregated based on following parameters that define the scope of the report, such as:
Major Players in Rett Syndrome
There are approx. 12+ key companies which are developing the therapies for Rett Syndrome. The companies which have their Rett Syndrome drug candidates in the most advanced stage, i.e. phase III include, Anavex Life Sciences Corp.
Phases
The report covers around 15+ products under different phases of clinical development like:
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
Route of Administration
Rett 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:
- Oral
- Intravenous
- Subcutaneous
- Parenteral
- Topical
Molecule Type
Products have been categorized under various Molecule types such as:
- Recombinant fusion proteins
- Small molecule
- Monoclonal antibody
- Peptide
- Polymer
- Gene therapy
Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Pipeline Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Rett Syndrome therapeutic drugs key players involved in developing key drugs.
Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Rett Syndrome drugs.
Key Questions
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Rett Syndrome drugs?
- How many Rett Syndrome drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Rett Syndrome?
- What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Rett Syndrome therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Rett Syndrome and their status?
- What are the key designations that have been granted to the emerging drugs?
Key Topics Covered
Introduction
Executive Summary
Rett Syndrome: Overview
- Causes
- Pathophysiology
- Signs and Symptoms
- Diagnosis
- Disease Management
Pipeline Therapeutics
- Comparative Analysis
Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
Rett Syndrome - The Publisher's Analytical Perspective
Late Stage Products (Phase III)
- Comparative Analysis
Blarcamesine: Anavex Life Sciences Corp
- Product Description
- Research and Development
- Product Development Activities
Mid Stage Products (Phase II)
- Comparative Analysis
Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
Early Stage Products (Phase I/II)
- Comparative Analysis
TSHA-102: Taysha GTx
- Product Description
- Research and Development
- Product Development Activities
Preclinical and Discovery Stage Products
- Comparative Analysis
Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
Inactive Products
- Comparative Analysis
Rett Syndrome Key Companies
Rett Syndrome Key Products
Rett Syndrome - Unmet Needs
Rett Syndrome - Market Drivers and Barriers
Rett Syndrome - Future Perspectives and Conclusion
Rett Syndrome Analyst Views
Rett Syndrome Key Companies
For more information about this clinical trials report visit https://www.researchandmarkets.com/r/b3ou7n
About ResearchAndMarkets.com
ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.
