The Friedreich’s Ataxia 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.
“Friedreich’s Ataxia Pipeline Insight, 2024“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Friedreich’s Ataxia Market.
Some of the key takeaways from the Friedreich’s Ataxia Pipeline Report:
Companies across the globe are diligently working toward developing novel Friedreich’s Ataxia treatment therapies with a considerable amount of success over the years.
Friedreich’s Ataxia companies working in the treatment market are Astellas Gene Therapies, Solid Biosciences, Design Therapeutics, Larimar Therapeutics, Minoryx Therapeutics, Metro International Biotech, LLC, PTC Therapeutics, Retrotope, and others, are developing therapies for the Friedreich’s Ataxia treatment
Emerging Friedreich’s Ataxia therapies in the different phases of clinical trials are- ASP 2016, AVB-202, DT-216, CTI-1601, MIN-102, MIB-626, Vatiquinone, RT001, and others are expected to have a significant impact on the Friedreich’s Ataxia market in the coming years.
In December 2024, Larimar Therapeutics, Inc. (Nasdaq: LRMR), a clinical-stage biotechnology company dedicated to treating complex rare diseases, announced promising initial results from its ongoing long-term OLE study. The study evaluates daily subcutaneous 25 mg injections of nomlabofusp, either self-administered or given by a caregiver, in participants with Friedreich’s ataxia (FA). Additionally, the company provided an update on the nomlabofusp development program.
In September 2024, Stealth BioTherapeutics Inc. (“Stealth”), a clinical-stage biotechnology company specializing in therapies for mitochondrial dysfunction, and the Friedreich’s Ataxia Research Alliance (FARA) announced that Stealth has received the Kyle Bryant Translational Research Award from FARA. The award will support the evaluation of the mitochondria-targeted molecule SBT-589 for the treatment of Friedreich’s ataxia (FA).
In September 2024, Papillon Therapeutics Inc., a clinical-stage biotechnology company developing multi-systemic genetic medicines for inherited diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to PPL-001, its investigational treatment for Friedreich’s ataxia.
Friedreich’s Ataxia Overview
Friedreich’s ataxia (FA) is a rare genetic disorder characterized by progressive damage to the nervous system, leading to symptoms such as difficulty with balance and coordination (ataxia), muscle weakness, loss of sensation in the extremities, and impaired speech. It is caused by a mutation in the FXN gene, which leads to a deficiency of frataxin, a protein involved in mitochondrial function.
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Emerging Friedreich’s Ataxia Drugs Under Different Phases of Clinical Development Include:
ASP 2016: Astellas Gene Therapies
AVB-202: Solid Biosciences
DT-216: Design Therapeutics
CTI-1601: Larimar Therapeutics
MIN-102: Minoryx Therapeutics
MIB-626: Metro International Biotech, LLC
Vatiquinone: PTC Therapeutics
RT001: Retrotope
Friedreich’s Ataxia Route of Administration
Friedreich’s Ataxia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as
Infusion
Intradermal
Intramuscular
Intranasal
Intravaginal
Oral
Parenteral
Subcutaneous
Topical
Molecule Type
Friedreich’s Ataxia Molecule Type
Friedreich’s Ataxia Products have been categorized under various Molecule types, such as
Vaccines
Monoclonal Antibody
Peptides
Polymer
Small molecule
Product Type
Friedreich’s Ataxia Pipeline Therapeutics Assessment
Friedreich’s Ataxia Assessment by Product Type
Friedreich’s Ataxia By Stage and Product Type
Friedreich’s Ataxia Assessment by Route of Administration
Friedreich’s Ataxia By Stage and Route of Administration
Friedreich’s Ataxia Assessment by Molecule Type
Friedreich’s Ataxia by Stage and Molecule Type
DelveInsight’s Friedreich’s Ataxia Report covers around 10+ 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 Friedreich’s Ataxia product details are provided in the report. Download the Friedreich’s Ataxia pipeline report to learn more about the emerging Friedreich’s Ataxia therapies
Some of the key companies in the Friedreich’s Ataxia Therapeutics Market include:
Key companies developing therapies for Friedreich’s Ataxia are – AbbVie, Minoryx Therapeutics S.L., Takeda, Reata Pharmaceuticals Inc., Design Therapeutics Inc., ApoPharma, Metro International Biotech LLC, PTC Therapeutics, Neurocrine Biosciences, Larimar Therapeutics Inc., Veristat Inc., Retrotope Inc., and others.
Friedreich’s Ataxia Pipeline Analysis:
The Friedreich’s Ataxia pipeline report provides insights into
The report provides detailed insights about companies that are developing therapies for the treatment of Friedreich’s Ataxia 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 Friedreich’s Ataxia Treatment.
Friedreich’s Ataxia key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
Friedreich’s Ataxia 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 Friedreich’s Ataxia 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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Friedreich’s Ataxia Pipeline Market Drivers
Rapid research in the field of ataxia therapeutics, increasing collaborations and acquisitions among key players are some of the important factors that are fueling the Friedreich’s Ataxia Market.
Friedreich’s Ataxia Pipeline Market Barriers
However, there are currently no approved therapies for the treatment of patients with Friedreich’s ataxia and other factors are creating obstacles in the Friedreich’s Ataxia Market growth.
Scope of Friedreich’s Ataxia Pipeline Drug Insight
Coverage: Global
Key Friedreich’s Ataxia Companies: Astellas Gene Therapies, Solid Biosciences, Design Therapeutics, Larimar Therapeutics, Minoryx Therapeutics, Metro International Biotech, LLC, PTC Therapeutics, Retrotope, and others
Key Friedreich’s Ataxia Therapies: ASP 2016, AVB-202, DT-216, CTI-1601, MIN-102, MIB-626, Vatiquinone, RT001, and others
Friedreich’s Ataxia Therapeutic Assessment: Friedreich’s Ataxia current marketed and Friedreich’s Ataxia emerging therapies
Friedreich’s Ataxia Market Dynamics: Friedreich’s Ataxia market drivers and Friedreich’s Ataxia market barriers
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Table of Contents
1. Friedreich’s Ataxia Report Introduction
2. Friedreich’s Ataxia Executive Summary
3. Friedreich’s Ataxia Overview
4. Friedreich’s Ataxia- Analytical Perspective In-depth Commercial Assessment
5. Friedreich’s Ataxia Pipeline Therapeutics
6. Friedreich’s Ataxia Late Stage Products (Phase II/III)
7. Friedreich’s Ataxia Mid Stage Products (Phase II)
8. Friedreich’s Ataxia Early Stage Products (Phase I)
9. Friedreich’s Ataxia Preclinical Stage Products
10. Friedreich’s Ataxia Therapeutics Assessment
11. Friedreich’s Ataxia Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Friedreich’s Ataxia Key Companies
14. Friedreich’s Ataxia Key Products
15. Friedreich’s Ataxia Unmet Needs
16 . Friedreich’s Ataxia Market Drivers and Barriers
17. Friedreich’s Ataxia Future Perspectives and Conclusion
18. Friedreich’s Ataxia 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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