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 Batten disease is a group of fatal genetic disorders. Providers also call this group of disorders neuronal ceroid lipofuscinosis (NCL). There are 13 known types of Batten disease. All types cause many of the same symptoms. These include seizures, vision loss and cognitive (thinking and reasoning) problems. Symptoms can appear in babies, children and teenagers. It’s passed down through families, and it results from a genetic mutation (gene change). The disorder affects the cells’ ability to break down and get rid of cellular waste. The body can’t dispose of proteins, sugars and lipids (fats), so they build up. This buildup causes problems with the nervous system that eventually leads to death. 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.

Batten disease, a group of rare, inherited neurological disorders, manifests due to mutations in genes responsible for producing proteins involved in lysosomal function. These mutations lead to impaired lysosomal enzyme activity, resulting in the accumulation of cellular waste materials, particularly lipids and proteins, within lysosomes throughout the body, including neurons in the brain and retina. This progressive accumulation causes neuronal dysfunction and degeneration, leading to a range of symptoms such as seizures, vision loss, cognitive decline, and motor impairment. Ultimately, Batten disease results in profound neurological disability and premature death, typically during late childhood or adolescence.

The diagnosis of Batten disease typically involves a combination of clinical evaluation, genetic testing, and various diagnostic tests. Clinical which include seizures, vision loss, cognitive decline, and motor impairment often prompt suspicion of the disease. Genetic testing, particularly DNA sequencing to identify mutations in known Batten disease-associated genes such as CLN3, CLN5, CLN6, and others, confirms the diagnosis. Additionally, diagnostic tests such as electroencephalogram (EEG), magnetic resonance imaging (MRI) of the brain, and ophthalmologic examination may be performed to assess neurological and ocular abnormalities characteristic of Batten disease. Analysis of cerebrospinal fluid may reveal elevated levels of specific substances, further supporting the diagnosis.

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. Treatment focuses on managing symptoms and improving quality of life. This often involves a multidisciplinary approach, including seizure control with antiepileptic medications, physical therapy to maintain mobility, and supportive care to address vision loss and cognitive decline.

'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 III, 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. Currently, the drug is in Phase III stage of its development for the treatment of NCL. In preclinical studies PLX-200 demonstrated neuroprotective activity in TPP1-deficient animals and prolonged their lifespan, delaying loss of mobility and reducing inflammation and cell death.

NGN-101: Neurogene

NGN-101 is being developed as a one-time treatment for both ocular and neurological manifestations of CLN5 Batten disease using AAV9 to deliver the gene encoding CLN5, which is deficient in children with the disease. It had received orphan drug designation from both the FDA and the European Medicines Agency. Currently, the drug is in Phase I/II stage of its clinical trial for the treatment of Batten disease.

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, Neurogene.

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 Development Activities

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

Pipeline 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

• Polaryx Therapeutics
• Neurogene
• Theranexus
• M6P Therapeutics
• Regenxbio

Key Products

• PLX 200
• NGN-101
• Batten-1
• M 092
• RGX 181

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