The Rett Syndrome Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.
“Rett Syndrome Pipeline Insight, 2024“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Rett Syndrome Market.
Some of the key takeaways from the Rett Syndrome Pipeline Report:
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Companies across the globe are diligently working toward developing novel Rett Syndrome treatment therapies with a considerable amount of success over the years.
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Rett Syndrome companies working in the treatment market are Prilenia Therapeutics, GEXVal, Neurogene Inc, Taysha Gene Therapies, Vyant Bio, Inc, Neuren Pharmaceuticals, Anavex Life Sciences, PTC Therapeutics, Novartis, Ultragenyx Pharmaceutical, GW Pharmaceuticals, StrideBio, AMO Pharma, Prilenia Therapeutics, Neurolixis, Biohaven Pharmaceuticals, and others, are developing therapies for the Rett Syndrome treatment
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Emerging Rett Syndrome therapies in the different phases of clinical trials are- Pridopidine, GXV-001, NGN-401, TSHA-102, VYNT-0126, Trofinetide, ANAVEX2-73 (blarcamesine), Vatiquinone, Fingolimod, Triheptanoin, Cannabidivarin, STRX 230, AMO 04, Pridopidine, NLX 101, BHV 5000, and others are expected to have a significant impact on the Rett Syndrome market in the coming years.
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In January 2024, Anavex Life Sciences Corp. announced topline results from the Phase II/III EXCELLENCE clinical trial, a randomized, double-blind, placebo-controlled study that assessed the clinical efficacy, safety, and tolerability of 30 mg ANAVEX®2-73 in 92 pediatric patients with Rett syndrome (RTT) aged 5 to 17 years.
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In March 2023, Melbourne-headquartered Neuren Pharmaceuticals reported that its North American collaborator, Acadia Pharmaceuticals, has obtained approval from the US Food and Drug Administration (FDA) for Neuren’s primary candidate, Daybue (trofinetide), designated for addressing Rett syndrome in both adults and children.
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In February 2023, Unravel Biosciences, Inc., a pharmaceutical firm specializing in developing treatments for intricate diseases through the utilization of a machine-learned model of human health, declared the successful conclusion of its Type B pre-IND meeting with the US Food and Drug Administration (FDA) concerning its RVL001 program targeting Rett Syndrome.
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In February 2023, PharmaTher Holdings Ltd., a prominent provider of specialized ketamine pharmaceuticals, revealed that the United States Food and Drug Administration (FDA) has granted orphan drug designation to KETARX (racemic ketamine) for addressing Rett Syndrome, a rare genetic neurological condition.
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In February 2023, Anavex Life Sciences Corp. declared the recruitment of 92 participants diagnosed with Rett syndrome in the ANAVEX®2-73 (blarcamesine) EXCELLENCE Phase 2/3 trial, encompassing Rett syndrome patients aged between 5 to 17 years.
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In January 2023, Neurogene Inc., a clinical-stage enterprise established to deliver transformative genetic therapies to individuals and families impacted by uncommon neurological conditions, disclosed that the FDA has endorsed the Company’s Investigational New Drug (IND) application for NGN-401, intended for addressing Rett syndrome.
Rett Syndrome Overview
Rett syndrome is a rare genetic neurological and developmental disorder that primarily affects girls. It leads to severe cognitive and physical impairments, often becoming apparent after a period of seemingly normal development. The disorder is caused by mutations in the MECP2 gene, which is essential for brain development.
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Emerging Rett Syndrome Drugs Under Different Phases of Clinical Development Include:
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Pridopidine: Prilenia Therapeutics
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GXV-001: GEXVal
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NGN-401: Neurogene Inc
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TSHA-102: Taysha Gene Therapies
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VYNT-0126: Vyant Bio, Inc
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Trofinetide: Neuren Pharmaceuticals
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ANAVEX2-73 (blarcamesine): Anavex Life Sciences
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Vatiquinone: PTC Therapeutics
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Fingolimod: Novartis
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Triheptanoin: Ultragenyx Pharmaceutical
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Cannabidivarin: GW Pharmaceuticals
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STRX 230: StrideBio
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AMO 04: AMO Pharma
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Pridopidine: Prilenia Therapeutics
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NLX 101: Neurolixis
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BHV 5000: Biohaven Pharmaceuticals
Rett Syndrome 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
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Oral
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Parenteral
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Intravenous
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Subcutaneous
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Topical
Rett Syndrome Molecule Type
Rett Syndrome Products have been categorized under various Molecule types, such as
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Monoclonal Antibody
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Peptides
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Polymer
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Small molecule
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Gene therapy
Rett Syndrome Pipeline Therapeutics Assessment
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Rett Syndrome Assessment by Product Type
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Rett Syndrome By Stage and Product Type
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Rett Syndrome Assessment by Route of Administration
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Rett Syndrome By Stage and Route of Administration
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Rett Syndrome Assessment by Molecule Type
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Rett Syndrome by Stage and Molecule Type
DelveInsight’s Rett Syndrome Report covers around 15+ products under different phases of clinical development like
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Late-stage products (Phase III)
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Mid-stage products (Phase II)
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Early-stage product (Phase I)
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Pre-clinical and Discovery stage candidates
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Discontinued & Inactive candidates
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Route of Administration
Further Rett Syndrome product details are provided in the report. Download the Rett Syndrome pipeline report to learn more about the emerging Rett Syndrome therapies
Some of the key companies in the Rett Syndrome Therapeutics Market include:
Key companies developing therapies for Rett Syndrome are – Neuren Pharmaceuticals, Anavex Life Sciences, Edison Pharmaceuticals, Novartis, Ultragenyx Pharmaceutical, GW Pharmaceuticals, Q-State Biosciences, AveXis, AMO Pharma, Neurolixis, Biohaven Pharmaceuticals, Echo Pharmaceuticals, and others.
Rett Syndrome Pipeline Analysis:
The Rett Syndrome pipeline report provides insights into
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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.
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It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Rett Syndrome Treatment.
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Rett Syndrome key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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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.
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Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Rett Syndrome market.
The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.
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Rett Syndrome Pipeline Market Drivers
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Increasing Prevalence in Rett Syndrome, increasing investment for healthcare infrastructure are some of the important factors that are fueling the Rett Syndrome Market.
Rett Syndrome Pipeline Market Barriers
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However, side-effects associated with the treatment of Rett Syndrome, complications involved with Rett syndrome and other factors are creating obstacles in the Rett Syndrome Market growth.
Scope of Rett Syndrome Pipeline Drug Insight
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Coverage: Global
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Key Rett Syndrome Companies: Prilenia Therapeutics, GEXVal, Neurogene Inc, Taysha Gene Therapies, Vyant Bio, Inc, Neuren Pharmaceuticals, Anavex Life Sciences, PTC Therapeutics, Novartis, Ultragenyx Pharmaceutical, GW Pharmaceuticals, StrideBio, AMO Pharma, Prilenia Therapeutics, Neurolixis, Biohaven Pharmaceuticals, and others
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Key Rett Syndrome Therapies: Pridopidine, GXV-001, NGN-401, TSHA-102, VYNT-0126, Trofinetide, ANAVEX2-73 (blarcamesine), Vatiquinone, Fingolimod, Triheptanoin, Cannabidivarin, STRX 230, AMO 04, Pridopidine, NLX 101, BHV 5000, and others
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Rett Syndrome Therapeutic Assessment: Rett Syndrome current marketed and Rett Syndrome emerging therapies
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Rett Syndrome Market Dynamics: Rett Syndrome market drivers and Rett Syndrome market barriers
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Table of Contents
1. Rett Syndrome Report Introduction
2. Rett Syndrome Executive Summary
3. Rett Syndrome Overview
4. Rett Syndrome- Analytical Perspective In-depth Commercial Assessment
5. Rett Syndrome Pipeline Therapeutics
6. Rett Syndrome Late Stage Products (Phase II/III)
7. Rett Syndrome Mid Stage Products (Phase II)
8. Rett Syndrome Early Stage Products (Phase I)
9. Rett Syndrome Preclinical Stage Products
10. Rett Syndrome Therapeutics Assessment
11. Rett Syndrome Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Rett Syndrome Key Companies
14. Rett Syndrome Key Products
15. Rett Syndrome Unmet Needs
16 . Rett Syndrome Market Drivers and Barriers
17. Rett Syndrome Future Perspectives and Conclusion
18. Rett Syndrome Analyst Views
19. Appendix
20. About DelveInsight
About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.
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