DelveInsight’s, “Batten Disease Pipeline Insight 2023” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in the Batten Disease pipeline landscape. It covers the Batten Disease pipeline drug profiles, including Batten Disease clinical trials and nonclinical stage products. It also covers the Batten Disease therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
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Key Takeaways from the Batten Disease Pipeline Report
- DelveInsight’s Batten Disease Pipeline report depicts a robust space with 10+ active players working to develop 10+ pipeline therapies for Batten Disease.
- The leading Batten Disease Companies include Recursion Pharmaceuticals, Polaryx Therapeutics, Neurogene Inc, Theranexus, and others
- Promising Batten Disease Pipeline Therapies include PLX-200, TSHA-118, LX1004, and others.
- In the Batten Disease Report, detailed description of the drug is given which includes mechanism of action of the drug, Batten Disease clinical trials studies, Batten Disease NDA approvals (if any), and product development activities comprising the technology, Batten Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
- The Batten Disease companies and academics are working to assess challenges and seek opportunities that could influence Batten Disease R&D. The Batten Disease therapies under development are focused on novel approaches to treat/improve Batten Disease.
For further information, refer to the detailed Batten Disease Unmet Needs, Batten Disease Market Drivers, and Batten Disease Market Barriers, click here for Batten Disease Ongoing Clinical Trial Analysis
Batten Disease Overview
Batten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system also known as neuronal ceroid lipofuscinoses, or NCLs. Most forms of Batten disease/NCLs usually begin during childhood. Children with the disease often appear healthy and develop normally before they begin to show symptoms.
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Batten Disease Emerging Drugs Profile
- PLX-200: Polaryx Therapeutics
PLX-200 is a repurposed drug that has been safely used to treat other diseases in both adults and children. It is a PPARα agonist that boosts lysosome biogenesis via TFEB upregulation. It has therapeutic and/or prophylactic potential for Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL or CLN2) and for other NCLs, such as Juvenile Infantile Neuronal Ceroid Lipofuscinosis (JNCL or CLN3). A neuroprotective effect has been demonstrated in murine LINCL and JNCL disease models. Fast track designation for JNCL with PLX-200 was granted from the FDA in August 2020. The drug is being currently evalauated in Phase III stage of development for the treatment of patients with Juvenile Infantile Neuronal Ceroid Lipofuscinosis.
- TSHA-118: Taysha Gene Therapies
TSHA-118 is being developed for the treatment of CLN1 disease, also known as infantile Batten disease, a rapidly progressing rare lysosomal storage disease with no approved treatment. TSHA-118 is a self-complementary AAV9 viral vector that expresses human codon-optimized CLN1 complementary deoxyribonucleic acid under control of the chicken ß-actin hybrid promoter.
- LX1004: Lexeo Therapeutics
LX1004 is an AAV mediated gene therapy treatment delivering CLN2 to the central nervous system. It is being evaluated for the treatment of Neuronal ceroid lipofuscinosis.
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Batten Disease Pipeline Therapeutics Assessment
There are approx. 10+ Batten Disease companies which are developing the Batten Disease therapies. The companies which have their Batten Disease drug candidates in the most advanced stage, i.e. Phase III include, Polaryx Therapeutics.
Scope of the Batten Disease Pipeline Report
- Coverage- Global
- Batten Disease Companies- Recursion Pharmaceuticals, Polaryx Therapeutics, Neurogene Inc, Theranexus, and others
- Batten Disease Pipeline Therapies- PLX-200, TSHA-118, LX1004, and others.
- Batten Disease Pipeline Segmentation: Product Type, Molecule Type, Route of Administration
Got Queries? Find out the related information on Batten Disease Mergers and acquisitions, Batten Disease Licensing Activities @ Batten Disease Emerging Drugs, and Recent Trends
Table of Content
- Introduction
- Executive Summary
- Batten Disease: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Late Stage Products (Phase III)
- PLX-200: Polaryx Therapeutics
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I/II)
- AT-GTX-501: Amicus Therapeutics
- Drug profiles in the detailed report…..
- Preclinical stage products
- CLN5: Neurogene
- Drug profiles in the detailed report…..
- Inactive Products
- Batten Disease Key Companies
- Batten Disease Key Products
- Batten Disease- Unmet Needs
- Batten Disease- Market Drivers and Barriers
- Batten Disease- Future Perspectives and Conclusion
- Batten Disease Analyst Views
- Batten Disease Key Companies
- Appendix
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