DelveInsight’s, “Rett Syndrome Pipeline Insight 2025” report provides comprehensive insights about 20+ companies and 20+ pipeline drugs in Rett Syndrome pipeline landscape. It covers the Rett Syndrome pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Rett Syndrome pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Curious about the latest updates in the Rett Syndrome Pipeline? Click here to explore the therapies and trials making headlines @ Rett Syndrome Pipeline Outlook Report

Key Takeaways from the Rett Syndrome Pipeline Report

• On December 12, 2025 — Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) announced that the U.S. Food and Drug Administration (FDA) has approved Daybue Stix (trofinetide) for oral solution, a dye- and preservative-free powder formulation of trofinetide, for the treatment of Rett syndrome in adult and pediatric patients aged two years and older. The newly approved formulation is designed to provide the same established efficacy and safety profile as the Daybue oral solution, while offering greater flexibility in dosing volume and improved taste options, giving both children and adults living with Rett syndrome more choice and convenience in their treatment experience.
• On December 05, 2025- Ionis Pharmaceuticals Inc. announced a phase 1-2 randomized, double-blind, sham-controlled, multiple-ascending dose (MAD) study to evaluate ION440 in pediatric and adult participants with MECP2 Duplication Syndrome (MDS) and will be conducted in two parts. During Part 1 (MAD) (36 weeks), participants will be randomized in a 3:1 ratio to receive ION440 or sham. Individuals who complete Part 1 may enter Part 2, an open label long-term extension study (LTE), where they will receive ION440 for up to approximately 156 weeks. Multiple dose cohorts (Dose A, Dose B, and Dose C) will be evaluated in the study.
• DelveInsight’s Rett Syndrome Pipeline report depicts a robust space with 20+ active players working to develop 20+ pipeline therapies for Rett Syndrome treatment.
• The leading Rett Syndrome Companies such as Xuanwu Hospital, Beijing, Aegle Therapeutics, ZEO ScientifiX Inc., Direct Biologics LLC, Rion Inc. and others.
• Promising Rett Syndrome Therapies such as Fingolimod (FTY720), Bionetide, TSHA-102, NNZ-2566, NTI164, ANAVEX2-73 and others.

Want to know which companies are leading innovation in Rett Syndrome? Dive into the full pipeline insights @ Rett Syndrome Clinical Trials Assessment

The Rett Syndrome Pipeline Report provides disease overview, pipeline scenario and therapeutic assessment of the key pipeline therapies in this domain. The Rett Syndrome Pipeline Report also highlights the unmet needs with respect to the Rett Syndrome.

Rett Syndrome Overview

Rett syndrome is a rare, severe neurodevelopmental disorder that primarily affects girls and leads to progressive impairments in motor skills, communication, cognition, and autonomic function. It is most commonly caused by mutations in the MECP2 gene on the X chromosome, a gene essential for normal brain development and synaptic function.

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.

The Rett Syndrome Pipeline report provides insights into

• 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.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Rett Syndrome Treatment.
• Rett Syndrome Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• 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.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Rett Syndrome market.

If you’re tracking ongoing Rett Syndrome Clinical trials, this press release is a must-read. Tap to see the breakthroughs @ Rett Syndrome Treatment Drugs

Rett Syndrome Companies

Xuanwu Hospital, Beijing, Aegle Therapeutics, ZEO ScientifiX Inc., Direct Biologics LLC, Rion Inc. and others.

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

• Intravenous
• Subcutaneous
• Oral
• Intramuscular

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

• Monoclonal antibody
• Small molecule
• Peptide

From emerging drug candidates to competitive intelligence, the Rett Syndrome Pipeline Report covers it all – check it out now @ Rett Syndrome Market Drivers and Barriers, and Future Perspectives

Scope of the Rett Syndrome Pipeline Report

• Coverage- Global
• Rett Syndrome Companies- Xuanwu Hospital, Beijing, Aegle Therapeutics, ZEO ScientifiX Inc., Direct Biologics LLC, Rion Inc. and others.
• Rett Syndrome Therapies- Fingolimod (FTY720), Bionetide, TSHA-102, NNZ-2566, NTI164, ANAVEX2-73 and others.
• Rett Syndrome Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Rett Syndrome Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

Stay ahead in oncology research – discover what’s next for the Rett Syndrome treatment landscape in this detailed analysis @ Rett Syndrome Emerging Drugs and Major Players

Table of Contents

1. Introduction
2. Executive Summary
3. Rett Syndrome: Overview
4. Pipeline Therapeutics
5. Therapeutics Assessment
6. Rett Syndrome – DelveInsight’s Analytical Perspective
7. Late Stage Products (Phase III)
8. Blarcamesine: Anavex Life Sciences Corp
9. Mid Stage Products (Phase II)
10. Early Stage Products (Phase I/II)
11. TSHA-102: Taysha GTx
12. Preclinical and Discovery Stage Products
13. Inactive Products
14. Rett Syndrome Key Companies
15. Rett Syndrome Key Products
16. Rett Syndrome- Unmet Needs
17. Rett Syndrome- Market Drivers and Barriers
18. Rett Syndrome- Future Perspectives and Conclusion
19. Rett Syndrome Analyst Views
20. Rett Syndrome Key Companies
21. Appendix

About Us

DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Yash Bhardwaj
Email: Send Email
Phone: 09650213330
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: NV
Country: United States
Website: https://www.delveinsight.com/report-store/rett-syndrome-pipeline-insight