Rett Syndrome Pipeline Insight Report 2023 (Updated) | Neuren Pharmaceuticals, Anavex Life Sciences, Edison Pharmaceuticals, Novartis, and others

DelveInsight's, "Rett Syndrome Pipeline Insight 2023" report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in the Rett Syndrome pipeline landscape. It covers the Rett Syndrome pipeline drug profiles, including Rett Syndrome clinical trials and nonclinical stage products. It also covers the Rett Syndrome therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Recent Developmental Activities in the Rett Syndrome Treatment Landscape
• In March 2023, Neuren Pharmaceuticals, based in Melbourne, announced that its North American partner Acadia Pharmaceuticals had received US Food and Drug Administration (FDA) approval for Neuren's lead candidate Daybue (trofinetide) to treat Rett syndrome in adults and children.
• In February 2023, Unravel Biosciences, Inc., a therapeutics company that advances drugs for complex diseases by leveraging a machine-learned model of human health, announced the successful completion of its Type B pre-IND meeting with the US Food and Drug Administration (FDA) for its RVL001 program in Rett Syndrome.
• In February 2023, PharmaTher Holdings Ltd., a leader in specialty ketamine pharmaceuticals, announced that the United States Food and Drug Administration had designated KETARX (racemic ketamine) as an orphan drug for the treatment of Rett Syndrome, a rare genetic neurological disorder.
• In February 2023, Anavex Life Sciences Corp. announced the enrollment of 92 Rett syndrome patients in the ANAVEX®2-73 (blarcamesine) EXCELLENCE Phase 2/3 study in Rett syndrome patients aged 5 to 17 years (inclusive).
• In January 2023, Neurogene Inc., a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, announced that the FDA has approved the Company's Investigational New Drug (IND) application for NGN-401 to treat Rett syndrome.

Discover the recent breakthroughs happening in the Rett Syndrome Pipeline landscape @ Rett Syndrome Pipeline Outlook- https://www.delveinsight.com/report-store/rett-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Key Takeaways from the Rett Syndrome Pipeline Report
• DelveInsight's Rett Syndrome pipeline report depicts a robust space with 15+ active players working to develop 15+ pipeline therapies for Rett Syndrome treatment.
• The leading Rett Syndrome Companies include Neuren Pharmaceuticals, Anavex Life Sciences, Edison Pharmaceuticals, Novartis, Ultragenyx Pharmaceutical, GW Pharmaceuticals, Q-State Biosciences, AveXis, AMO Pharma, Neurolixis, Biohaven Pharmaceuticals, Echo Pharmaceuticals, Vyant Bio, Prilenia Therapeutics, StrideBio, Relmada Therapeutics, VICO Therapeutics, Taysha Gene Therapies, Sarepta therapeutics, and others
• Promising Rett Syndrome Pipeline Therapies include Blarcamesine, EPI-743, Fingolimod, REL 1017, Triheptanoin, Cannabidivarin, STRX 230, Research programme: Rett syndrome therapeutics, AVXS 201, AMO 04, Pridopidine, NLX 101, BHV 5000, and others.
• The Rett Syndrome companies and academics are working to assess challenges and seek opportunities that could influence Rett Syndrome R&D. The Rett Syndrome therapies under development are focused on novel approaches to treat/improve Rett Syndrome.

Rett Syndrome Emerging Drugs Profile

• ANAVEX2-73 (blarcamesine): Anavex Life Sciences
ANAVEX®2-73 (blarcamesine) activates the Sigma-1 receptor (S1R) protein, which serves as a molecular chaperone and functional modulator involved in restoring cellular homeostasis. ANAVEX®2-73 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. ANAVEX®2-73 (blarcamesine) had previously received Fast Track designation, Rare Pediatric Disease designation and Orphan Drug designation from the FDA for the treatment of Rett syndrome. ANAVEX®2-73 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 Segmentation: Phases
DelveInsight's 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
• Parenteral
• Intravenous
• Subcutaneous
• Topical.

Molecule Type
Products have been categorized under various Molecule types such as
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Get More Information on the Rett Syndrome Drugs and Companies of Report @ Rett Syndrome Ongoing Clinical Trials Analysis- https://www.delveinsight.com/sample-request/rett-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Scope of the Rett Syndrome Pipeline Report
• Coverage- Global
• Rett Syndrome Companies- Neuren Pharmaceuticals, Anavex Life Sciences, Edison Pharmaceuticals, Novartis, Ultragenyx Pharmaceutical, GW Pharmaceuticals, Q-State Biosciences, AveXis, AMO Pharma, Neurolixis, Biohaven Pharmaceuticals, Echo Pharmaceuticals, Vyant Bio, Prilenia Therapeutics, StrideBio, Relmada Therapeutics, VICO Therapeutics, Taysha Gene Therapies, Sarepta therapeutics, and others
• Rett Syndrome Pipeline Therapies- Blarcamesine, EPI-743, Fingolimod, REL 1017, Triheptanoin, Cannabidivarin, STRX 230, Research programme: Rett syndrome therapeutics, AVXS 201, AMO 04, Pridopidine, NLX 101, BHV 5000, and others.
• Rett Syndrome Pipeline Segmentation: Product Type, Molecule Type, Route of Administration

Find out more about the list of FDA-approved drugs for Rett Syndrome @ Rett Syndrome Treatment Landscape- https://www.delveinsight.com/sample-request/rett-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Table of Content
1. Introduction
2. Executive Summary
3. Rett Syndrome: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Rett Syndrome - DelveInsight's Analytical Perspective
7. Late Stage Products (Preregistration)
8. Trofinetide: Neuren Pharmaceuticals
9. Drug profiles in the detailed report…..
10. Late Stage Products (Phase III)
11. Blarcamesine: Anavex Life Sciences
12. Drug profiles in the detailed report…..
13. Mid Stage Products (Phase II)
14. Drug Name: Company Name
15. Drug profiles in the detailed report…..
16. Early Stage Products (Phase I)
17. BHV 5000: Biohaven Pharmaceuticals
18. Drug profiles in the detailed report…..
19. Preclinical and Discovery Stage Products
20. Drug Name: Company Name
21. Drug profiles in the detailed report…..
22. Inactive Products
23. Rett Syndrome Key Companies
24. Rett Syndrome Key Products
25. Rett Syndrome- Unmet Needs
26. Rett Syndrome- Market Drivers and Barriers
27. Rett Syndrome- Future Perspectives and Conclusion
28. Rett Syndrome Analyst Views
29. Rett Syndrome Key Companies
30. Appendix

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