Rett Syndrome Pipeline Analysis Report 2023: Comprehensive Insights for 15+ Companies and 15+ Pipeline Drugs

DUBLIN, July 14, 2023 /PRNewswire/ — The “Rett Syndrome – Pipeline Insight, 2023” clinical trials has been added to’s offering.


This report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in Rett Syndrome pipeline landscape.

This report covers the profiles of pipeline drugs, including both clinical and nonclinical stage products. It also provides a thorough assessment of therapeutics based on product type, stage, route of administration, and molecule type. Additionally, the report highlights inactive pipeline products in this space, providing a comprehensive overview of the Rett Syndrome pipeline landscape.

In this report, we provide detailed insights into the current scenario and growth prospects across the Rett Syndrome indication. A detailed picture of the Rett Syndrome pipeline landscape is presented, encompassing an overview of the disease and Rett Syndrome treatment guidelines. The assessment section of the report delves into the commercial and clinical aspects of Rett Syndrome, conducting an in-depth evaluation of the pipeline products under development.

Detailed descriptions of each drug are included in the report, providing valuable information on the mechanism of action, clinical studies, NDA approvals (if any), and product development activities. This encompasses technology, Rett Syndrome collaborations, licensing, mergers and acquisitions, funding, designations, and other relevant details related to product development.

As companies and academics continue to work diligently in the field of Rett Syndrome, assessing challenges and seeking opportunities, this report provides a comprehensive overview of the advancements in R&D. The therapies under development focus on novel approaches to treat and improve Rett Syndrome, offering promising prospects for patients in need.

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

ANAVEX2-73 (blarcamesine): Anavex Life Sciences

ANAVEX2-73 (blarcamesine) activates the Sigma-1 receptor (S1R) protein, which serves as a molecular chaperone and functional modulator involved in restoring cellular homeostasis. ANAVEX2-73 is an orally available, small-molecule activator of the sigma-1 receptor (SIGMAR1) which, data suggest, is pivotal to restoring neural cell homeostasis and promoting neuroplasticity.

ANAVEX2-73 (blarcamesine) had previously received Fast Track designation, Rare Pediatric Disease designation and Orphan Drug designation from the FDA for the treatment of Rett syndrome. ANAVEX2-73 also exhibited anticonvulsant, anti-amnesic, neuroprotective, and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. Currently the drug is in Phase III stage of Clinical trial evaluation for the treatment of Rett syndrome.

TSHA-102: Taysha Gene Therapies

TSHA-102 comprises a shorter but functional version of MECP2 that is activated only in nerve cells. It also has a self-regulatory suppressor called a miRNA-responsive auto-regulatory element, or miRARE, which prevents the toxic overexpression (excess production) of the MECP2 protein.

The pharmacological activity of TSHA-102 was assessed across three dose levels and three age groups in a Rett mouse model that lacked the mouse equivalent of the MECP2 gene. A one-time intrathecal injection significantly increased survival at all dose levels, with the mid-to-high dose levels improving survival in all age groups compared with controls. Currently the drug is in Phase I/II stage of Clinical trial evaluation for the treatment of 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. 15+ 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.

Rett Syndrome: Pipeline Development 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.

Pipeline 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.


  • 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

Products have been categorized under various ROAs such as

  • Oral
  • Parenteral
  • intravenous
  • Subcutaneous
  • Topical.
  • Molecule Type

Products have been categorized under various Molecule types such as

  • Monoclonal Antibody
  • Peptides
  • Polymer
  • Small molecule
  • Gene therapy
  • Product Type

Rett Syndrome Report Insights

  • Rett Syndrome Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs

Rett Syndrome Report Assessment

  • Pipeline Product Profiles
  • Therapeutic Assessment
  • Pipeline Assessment
  • Inactive drugs assessment
  • Unmet Needs

Key Players

  • Anavex Life Sciences
  • Novartis
  • Ultragenyx Pharmaceutical
  • Q-State Biosciences
  • AMO Pharma
  • Neurolixis
  • Vyant Bio
  • Prilenia Therapeutics
  • StrideBio
  • Taysha Gene Therapies

Key Products

  • Blarcamesine
  • Fingolimod
  • Triheptanoin
  • STRX 230

Research programme: Rett syndrome therapeutics

  • AMO 04
  • Pridopidine
  • NLX 101
  • TSHA 102

For more information about this clinical trials report visit

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Rett Syndrome Pipeline Analysis Report 2023: Comprehensive Insights for 15+ Companies and 15+ Pipeline Drugs WeeklyReviewer

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