Rett Syndrome Pipeline Analysis, 2024 Updates | Latest FDA, EMA, and PMDA Approvals | Neuren Pharma, Anavex Life Sciences, Edison Pharma, Novartis, Ultragenyx Pharma, GW Pharma, Q-State Biosciences, AveXis

(Las Vegas, Nevada, United States) As per DelveInsight's assessment, globally, Rett Syndrome pipeline constitutes 15+ key companies continuously working towards developing 15+ Rett Syndrome treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

"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.

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.

Some of the key takeaways from the Rett Syndrome Pipeline Report: https://www.delveinsight.com/sample-request/rett-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr
• Companies across the globe are diligently working toward developing novel Rett Syndrome treatment therapies with a considerable amount of success over the years.
• 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
• 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.
• 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.
• 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.
• 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.
• 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.
• 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 neurodevelopmental condition predominantly observed in girls. It initially displays typical early growth and development, which then transitions into a gradual decline in development. This phase is marked by the loss of intentional hand usage, unique hand movements, decreased rate of brain and head growth, challenges in walking, seizures, and intellectual disability.

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Emerging Rett Syndrome Drugs Under Different Phases of Clinical Development Include:
• Pridopidine: Prilenia Therapeutics
• GXV-001: GEXVal
• NGN-401: Neurogene Inc
• TSHA-102: Taysha Gene Therapies
• VYNT-0126: Vyant Bio, Inc
• Trofinetide: Neuren Pharmaceuticals
• ANAVEX2-73 (blarcamesine): Anavex Life Sciences
• Vatiquinone: PTC Therapeutics
• Fingolimod: Novartis
• Triheptanoin: Ultragenyx Pharmaceutical
• Cannabidivarin: GW Pharmaceuticals
• STRX 230: StrideBio
• AMO 04: AMO Pharma
• Pridopidine: Prilenia Therapeutics
• NLX 101: Neurolixis
• 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
• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical

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

Rett Syndrome Pipeline Therapeutics Assessment
• Rett Syndrome Assessment by Product Type
• Rett Syndrome By Stage and Product Type
• Rett Syndrome Assessment by Route of Administration
• Rett Syndrome By Stage and Route of Administration
• Rett Syndrome Assessment by Molecule Type
• Rett Syndrome by Stage and Molecule Type

DelveInsight's Rett Syndrome 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)
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• 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
https://www.delveinsight.com/sample-request/rett-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

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
• 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 key 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.
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.

Download Sample PDF Report to know more about Rett Syndrome drugs and therapies https://www.delveinsight.com/sample-request/rett-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Rett Syndrome Pipeline Market Drivers
• 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
• 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
• Coverage: Global
• 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
• 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
• Rett Syndrome Therapeutic Assessment: Rett Syndrome current marketed and Rett Syndrome emerging therapies
• Rett Syndrome Market Dynamics: Rett Syndrome market drivers and Rett Syndrome market barriers

Request for Sample PDF Report for Rett Syndrome Pipeline Assessment and clinical trials https://www.delveinsight.com/sample-request/rett-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

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

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