Rett Syndrome - Pipeline Insight, 2025

This “Rett Syndrome - Pipeline Insight, 2025” 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.

Rett Syndrome: Understanding

Rett Syndrome: Overview

Rett syndrome is a rare genetic neurological disorder that occurs almost exclusively in girls and leads to severe impairments, affecting nearly every aspect of the child’s life: their ability to speak, walk, eat, and even breathe easily. The hallmark of Rett syndrome is near constant repetitive hand movements. Rett syndrome is usually recognized in children between 6 to 18 months as they begin to miss developmental milestones or lose abilities they had gained. Rett syndrome is caused by mutations on the X chromosome on a gene called MECP2. There are more than 900 different mutations found on the MECP2 gene. Most of these mutations are found in eight different “hot spots.” Rett syndrome is not a degenerative disorder with individuals living to middle age or beyond. Rett syndrome occurs worldwide in 1 of every 10,000 female births, and is even rarer in boys. Rett syndrome can present with a wide range of disability ranging from mild to severe. The course and severity of Rett syndrome is determined by the location, type and severity of the mutation and X-inactivation.

The main signs and symptoms of ret syndrome includes slowed growth loss of movement and coordination activities, loss of communication abilities and unusual hand movements, unusual eye movements, breathing problems, irritability and crying, other unusual behaviors, intellectual disabilities, seizures, sideways curvature of the spine (scoliosis), irregular heartbeat, sleep disturbances.

Diagnosing Rett syndrome involves careful observation of the child's growth and development and answering questions about medical and family history. The diagnosis is usually considered when slowing of head growth is noticed or loss of skills or developmental milestones occurs. Rett syndrome after evaluation, genetic testing (DNA analysis) may be needed to confirm the diagnosis other tests are CT scan, EEG (electroencephalogram), Genetic testing, MRI these all tests are involved in diagnosing the syndrome and helpful in the further treatment of the disease.

Although there is no cure for rett syndrome, treatments address symptoms and provide support. These may improve the potential for movement, communication and social participation. Treatments that can help children and adults with rett syndrome include, regular medical care, medications, physical therapy, occupational therapy, speech-language therapy, nutritional support, behavioral intervention, support services. Early intervention programs and school, social and job-training services may help with integration into school, work and social activities special adaptations may make participation possible.

'Rett Syndrome - Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Rett Syndrome pipeline landscape is provided which includes the disease overview and Rett Syndrome treatment guidelines. The assessment part of the report embraces, in depth Rett Syndrome commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Rett Syndrome collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

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.

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.

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 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 Players

• Blarcamesine
• TSHA-102
• NGN-401
• GXV 001
• Cannabidivarin
• AMO 04

Key Products

• Anavex Life Sciences Corp.

Taysha Gene Therapies, Inc.

Neurogene Inc.

GEXVal

• GW Pharmaceuticals
• AMO Pharma

This product will be delivered within 1-3 business days.