Rett Syndrome Clinical Trial Pipeline Appears Robust With 20+ Key Pharma Companies Actively Working in the Domain | DelveInsight

DelveInsight's, "Rett Syndrome Pipeline Insight 2026" 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.

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Key Takeaways from the Rett Syndrome Pipeline Report

* In January 2026, Neurogene Inc . announced a phase 1/2 study and is an open-label, baseline-controlled, multicenter, single-arm study designed to assess the efficacy, safety, and tolerability of administration of NGN401, an adeno-associated viral vector serotype 9 (AAV9) using Neurogene's proprietary transgene regulation technology. NGN-401 contains a full-length human MECP2 gene and is designed to express therapeutic levels of the MeCP2 protein while avoiding overexpression.
* 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.

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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.

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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

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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

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Table of Contents

* Introduction
* Executive Summary
* Rett Syndrome: Overview
* Pipeline Therapeutics
* Therapeutics Assessment
* Rett Syndrome - DelveInsight's Analytical Perspective
* Late Stage Products (Phase III)
* Blarcamesine: Anavex Life Sciences Corp
* Mid Stage Products (Phase II)
* Early Stage Products (Phase I/II)
* TSHA-102: Taysha GTx
* Preclinical and Discovery Stage Products
* Inactive Products
* 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
* Appendix

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