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.
To explore more information on the latest breakthroughs in the Batten Disease Pipeline treatment landscape of the report, click here @ Batten Disease Pipeline Outlook
Key Takeaways from the Batten Disease Pipeline Report
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 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 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 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.
Dive deep into rich insights for drugs for Batten Disease Market Drivers and Batten Disease Market Barriers, click here @ Batten Disease Unmet Needs and Analyst Views
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
Got Queries? Find out the related information on Batten Disease Mergers and acquisitions, Batten Disease Licensing Activities @ Batten Disease Emerging Drugs, and Recent Trends
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