Batten Disease Drug Pipeline Market Report 2022: Comprehensive Insights About 10+ Companies and 10+ Pipeline Drugs

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Jan 04, 2023, 19:30 ET

DUBLIN, Jan. 4, 2023 /PRNewswire/ -- The "Batten Disease - Pipeline Insight, 2022" drug pipelines has been added to ResearchAndMarkets.com's offering.

This "Batten Disease - Pipeline Insight, 2022" report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Batten Disease pipeline landscape.

It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

The assessment part of the report embraces, in depth Batten Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, 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.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Batten Disease R&D. The therapies under development are focused on novel approaches to treat/improve Batten Disease.

Batten Disease Emerging Drugs Chapters

This segment of the Batten Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Batten Disease Emerging Drugs

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 PPAR? 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 ?-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.

Batten Disease: Therapeutic Assessment

This segment of the report provides insights about the different Batten Disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Batten Disease

There are approx. 10+ key companies which are developing the therapies for Batten Disease. The companies which have their Batten Disease drug candidates in the most advanced stage, i.e. Phase III include, Polaryx Therapeutics.

Batten Disease Report Insights