Rett Syndrome Pipeline 2025: MOA, ROA, and Clinical Trial Insights Explored by DelveInsight | Neuren Pharma, Anavex Life Sciences, Edison Pharma, Novartis, Ultragenyx Pharma, GW Pharma, Q-State

(Las Vegas, Nevada, United States) As per DelveInsight's assessment, globally, Rett Syndrome pipeline constitutes 20+ key companies continuously working towards developing 20+ Rett Syndrome treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

The Rett 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.

"Rett Syndrome Pipeline Insight, 2025 [https://www.delveinsight.com/sample-request/rett-syndrome-pipeline-insight?utm_source=naver&utm_medium=pressrelease&utm_campaign=gpr]" report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Rett Syndrome Market.

Some of the key takeaways from the Rett Syndrome Pipeline Report:

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Companies across the globe are diligently working toward developing novel Rett Syndrome treatment therapies with a considerable amount of success over the years.

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Rett Syndrome companies working in the treatment market are Prilenia Therapeutics, GEXVal, Neurogene Inc, Taysha Gene Therapies, Vyant Bio, Inc, Neuren Pharmaceuticals, Anavex Life Sciences, PTC Therapeutics, Novartis, Ultragenyx Pharmaceutical, GW Pharmaceuticals, StrideBio, AMO Pharma, Prilenia Therapeutics, Neurolixis, Biohaven Pharmaceuticals, and others, are developing therapies for the Rett Syndrome treatment

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Emerging Rett Syndrome therapies in the different phases of clinical trials are- Pridopidine, GXV-001, NGN-401, TSHA-102, VYNT-0126, Trofinetide, ANAVEX2-73 (blarcamesine), Vatiquinone, Fingolimod, Triheptanoin, Cannabidivarin, STRX 230, AMO 04, Pridopidine, NLX 101, BHV 5000, and others are expected to have a significant impact on the Rett Syndrome market in the coming years.

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In May 2025, Taysha obtained FDA agreement on the pivotal Part B trial design for TSHA-102, enabling the submission of an Investigational New Drug (IND) amendment without holding a formal end-of-phase meeting. An update on the program-covering the pivotal design, safety and efficacy data, and Rett syndrome natural history analysis-is planned for the second quarter of 2025 at the International Rett Syndrome Foundation (IRSF) Meeting. The company also noted that TSHA-102 may qualify for a Priority Review Voucher, potentially expediting its regulatory process.

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In January 2025, Acadia Pharmaceuticals filed its first Marketing Authorization Application (MAA) in Europe with the European Medicines Agency (EMA) for trofinetide, intended for the treatment of Rett syndrome in patients aged two years and older. If approved, trofinetide would be the first authorized therapy for Rett syndrome in the EU.

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In November 2024, Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company dedicated to developing genetic medicines for rare neurological disorders, has reported positive interim results from its ongoing Phase 1/2 open-label trial. The trial, which evaluates NGN-401 gene therapy in female pediatric patients with Rett syndrome, showed promising data in the first four participants of the low-dose cohort, demonstrating a favorable safety profile.

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In October 2024, Unravel Biosciences, Inc. ("Unravel"), an AI-driven therapeutics company focused on developing treatments for complex diseases, has partnered with the clinical research unit of PECET at the University of Antioquia in Medellin, Colombia. Certified in Good Clinical Practices by the Colombian Health Regulatory Authority (INVIMA), PECET has been designated as the study site for Unravel's RVL-001 proof-of-concept clinical trials targeting Rett syndrome (RTT) and Pitt Hopkins syndrome (PTHS).

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In January 2024, Anavex Life Sciences Corp. announced topline results from the Phase II/III EXCELLENCE clinical trial, a randomized, double-blind, placebo-controlled study that assessed the clinical efficacy, safety, and tolerability of 30 mg ANAVEX Registered 2-73 in 92 pediatric patients with Rett syndrome (RTT) aged 5 to 17 years.

Rett Syndrome Overview

Rett syndrome is a rare genetic neurological and developmental disorder that primarily affects girls. It leads to severe cognitive and physical impairments, often becoming apparent after a period of seemingly normal development. The disorder is caused by mutations in the MECP2 gene, which is essential for brain development.

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Emerging Rett Syndrome Drugs Under Different Phases of Clinical Development Include:

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Pridopidine: Prilenia Therapeutics

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GXV-001: GEXVal

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NGN-401: Neurogene Inc

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TSHA-102: Taysha Gene Therapies

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VYNT-0126: Vyant Bio, Inc

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Trofinetide: Neuren Pharmaceuticals

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ANAVEX2-73 (blarcamesine): Anavex Life Sciences

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Vatiquinone: PTC Therapeutics

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Fingolimod: Novartis

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Triheptanoin: Ultragenyx Pharmaceutical

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Cannabidivarin: GW Pharmaceuticals

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STRX 230: StrideBio

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AMO 04: AMO Pharma

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Pridopidine: Prilenia Therapeutics

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NLX 101: Neurolixis

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BHV 5000: Biohaven Pharmaceuticals

Rett Syndrome 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

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Oral

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Parenteral

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Intravenous

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Subcutaneous

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Topical

Rett Syndrome Molecule Type

Rett Syndrome Products have been categorized under various Molecule types, such as

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Monoclonal Antibody

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Peptides

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Polymer

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Small molecule

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Gene therapy

Rett Syndrome Pipeline Therapeutics Assessment

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Rett Syndrome Assessment by Product Type

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Rett Syndrome By Stage and Product Type

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Rett Syndrome Assessment by Route of Administration

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Rett Syndrome By Stage and Route of Administration

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Rett Syndrome Assessment by Molecule Type

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Rett Syndrome by Stage and Molecule Type

DelveInsight's Rett Syndrome Report covers around 20+ products under different phases of clinical development like

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Late-stage products (Phase III)

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Mid-stage products (Phase II)

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Early-stage product (Phase I)

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Pre-clinical and Discovery stage candidates

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Discontinued & Inactive candidates

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Route of Administration

Further Rett Syndrome product details are provided in the report. Download the Rett Syndrome pipeline report to learn more about the emerging Rett Syndrome therapies [https://www.delveinsight.com/sample-request/rett-syndrome-pipeline-insight?utm_source=naver&utm_medium=pressrelease&utm_campaign=gpr]

Some of the key companies in the Rett Syndrome Therapeutics Market include:

Key companies developing therapies for Rett Syndrome are - Neuren Pharmaceuticals, Anavex Life Sciences, Edison Pharmaceuticals, Novartis, Ultragenyx Pharmaceutical, GW Pharmaceuticals, Q-State Biosciences, AveXis, AMO Pharma, Neurolixis, Biohaven Pharmaceuticals, Echo Pharmaceuticals, and others.

Rett Syndrome Pipeline Analysis:

The Rett Syndrome pipeline report provides insights into

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The report provides detailed insights about companies that are developing therapies for the treatment of Rett Syndrome with aggregate therapies developed by each company for the same.

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It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Rett Syndrome Treatment.

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Rett Syndrome key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

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Rett 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.

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Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Rett 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.

Download Sample PDF Report to know more about Rett Syndrome drugs and therapies [https://www.delveinsight.com/sample-request/rett-syndrome-pipeline-insight?utm_source=naver&utm_medium=pressrelease&utm_campaign=gpr]

Rett Syndrome Pipeline Market Drivers

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Increasing Prevalence in Rett Syndrome, increasing investment for healthcare infrastructure are some of the important factors that are fueling the Rett Syndrome Market.

Rett Syndrome Pipeline Market Barriers

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However, side-effects associated with the treatment of Rett Syndrome, complications involved with Rett syndrome and other factors are creating obstacles in the Rett Syndrome Market growth.

Scope of Rett Syndrome Pipeline Drug Insight

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Coverage: Global

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Key Rett Syndrome Companies: Prilenia Therapeutics, GEXVal, Neurogene Inc, Taysha Gene Therapies, Vyant Bio, Inc, Neuren Pharmaceuticals, Anavex Life Sciences, PTC Therapeutics, Novartis, Ultragenyx Pharmaceutical, GW Pharmaceuticals, StrideBio, AMO Pharma, Prilenia Therapeutics, Neurolixis, Biohaven Pharmaceuticals, and others

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Key Rett Syndrome Therapies: Pridopidine, GXV-001, NGN-401, TSHA-102, VYNT-0126, Trofinetide, ANAVEX2-73 (blarcamesine), Vatiquinone, Fingolimod, Triheptanoin, Cannabidivarin, STRX 230, AMO 04, Pridopidine, NLX 101, BHV 5000, and others

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Rett Syndrome Therapeutic Assessment: Rett Syndrome current marketed and Rett Syndrome emerging therapies

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Rett Syndrome Market Dynamics: Rett Syndrome market drivers and Rett Syndrome market barriers

Request for Sample PDF Report for Rett Syndrome Pipeline Assessment and clinical trials [https://www.delveinsight.com/sample-request/rett-syndrome-pipeline-insight?utm_source=naver&utm_medium=pressrelease&utm_campaign=gpr]

Table of Contents

1. Rett Syndrome Report Introduction

2. Rett Syndrome Executive Summary

3. Rett Syndrome Overview

4. Rett Syndrome- Analytical Perspective In-depth Commercial Assessment

5. Rett Syndrome Pipeline Therapeutics

6. Rett Syndrome Late Stage Products (Phase II/III)

7. Rett Syndrome Mid Stage Products (Phase II)

8. Rett Syndrome Early Stage Products (Phase I)

9. Rett Syndrome Preclinical Stage Products

10. Rett Syndrome Therapeutics Assessment

11. Rett Syndrome Inactive Products

12. Company-University Collaborations (Licensing/Partnering) Analysis

13. Rett Syndrome Key Companies

14. Rett Syndrome Key Products

15. Rett Syndrome Unmet Needs

16 . Rett Syndrome Market Drivers and Barriers

17. Rett Syndrome Future Perspectives and Conclusion

18. Rett Syndrome Analyst Views

19. Appendix

20. About DelveInsight

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

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