Batten Disease - Pipeline Insight, 2025

This “Batten Disease - Pipeline Insight, 2025” report provides comprehensive insights about 10+ companies and 12+ 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.

Batten Disease: Understanding

Batten Disease: Overview

Batten disease (also known as neuronal ceroid lipofuscinosis, NCL) is the name for a group of inherited nervous system disorders that most often begin in childhood. They interfere with a cell's ability to recycle a cellular residue called lipofuscin. Batten is the term commonly used to describe the many forms of the disease, which is formally called neuronal ceroid lipofuscinosis. The many forms of the disease are classified by the gene that causes the disorder, with each gene being called "ceroid lipofucinosis, neuronal" (CLN) and given a different number as its subtype. Because of the different gene mutations, signs and symptoms range in severity and progress at different rates. Symptoms generally include progressive vision loss leading to blindness, seizures, movement disorder, and dementia. Developmental skills such as standing, walking, and talking may not be achieved or are gradually lost. Other symptoms that continue to worsen over time include learning difficulties, poor concentration, and progressive loss of language skills and speech. Most children become bedridden and unable to communicate. Some children develop problems sleeping. Currently, most diagnosis of Batten disease are made by genetic testing.

Pathologically, Batten disease is characterized by lysosomal accumulation of autofluorescent storage material, glial reactivity and neuronal loss. Substantial progress has been made towards the development of effective therapies and treatments for the multiple forms of Batten disease. In 2017, cerliponase alfa (Brineura), a tripeptidyl peptidase enzyme replacement therapy, became the first globally approved treatment for CLN2 Batten disease. Here, we provide an overview of the promising therapeutic avenues for Batten disease, highlighting current FDA-approved clinical trials and prospective future treatments.

"Batten Disease - Pipeline Insight, 2025" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Batten Disease pipeline landscape is provided which includes the disease overview and Batten Disease treatment guidelines. 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 used to treat other diseases in both adults and children. It is a PPARα agonist that boosts lysosome biogenesis via TFEB upregulation. It may have 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. The treatment extended the life span of a murine LINCL disease model and reduced the level of storage materials (lipofuscin) in the brain. Orphan drug designation has been obtained from both the FDA and EMA for all subtypes of neuronal ceroid lipofuscinosis. INDs for LINCL and JNCL with PLX-200 were approved in January and April 2020 from the FDA, respectively. Fast track designation for JNCL with PLX-200 was also granted from the FDA in August 2020. Currently, the drug is in Phase III stage of its development for the treatment of NCL.

TTX-381: Tern Therapeutics

TTX-381 is an investigational, one-time gene therapy being developed by Tern Therapeutics to treat the ocular (vision) manifestations of CLN2 disease - a rare, pediatric-onset form of Batten disease characterized by deficiency of the TPP1 enzyme. Built on an AAV9 viral vector, it delivers a functional TPP1 gene directly into the retina via subretinal injection. In its ongoing Phase I/II, first-in-human dose-escalation trial (NCT05791864) at Great Ormond Street Hospital (London), interim data demonstrate a favorable safety profile, successful enzyme expression in treated eyes, and preservation or improvement of photoreceptor integrity and functional vision outcomes in all treated eyes. In February 2025, Tern reported both completion of enrollment  - including dose selection for an expansion cohort  - alongside positive safety and early efficacy signals such as stabilization of retinal structure and vision improvement. Recognizing its potential to fulfill a significant unmet need in Batten disease, the US FDA granted Fast Track designation to TTX-381 in May 2025 .

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.

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.

Phases
The report covers around 12+ products under different phases of clinical development, like:

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of:
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration
Batten Disease 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
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type
Products have been categorized under various Molecule types, such as:

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Batten Disease: Pipeline Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Batten Disease therapeutic drugs key players involved in developing key drugs.

Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Batten Disease drugs.

Batten Disease Report Insights

• Batten Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Batten Disease Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Batten Disease drugs?
• How many Batten Disease drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Batten Disease?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Batten Disease therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Batten Disease and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Amicus Therapeutics
• REGENXBIO
• Polaryx Therapeutics
• Tern Therapeutics
• Theranexus
• M6P Therapeutics

Key Products

• AT-GTX-502
• RGX-381
• PLX-200
• TTX-381
• BBDF-101
• M-082

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