(MENAFN- GlobeNewsWire - Nasdaq) The Batten Disease pipeline presents opportunities with over 10 companies developing 12+ drugs in varied clinical stages. Novel approaches, including gene therapy and enzyme replacement, address unmet needs, with key players like Polaryx and Tern Therapies advancing treatments for this rare disorder.

Dublin, Oct. 22, 2025 (GLOBE NEWSWIRE) -- The "Batten Disease - Pipeline Insight, 2025" has been added to ResearchAndMarkets's offering.

The "Batten Disease - Pipeline Insight, 2025" report offers a detailed examination of the Batten Disease treatment landscape, featuring insights from over 10 companies and a pipeline of more than 12 drugs. This report covers the clinical and nonclinical drug profiles, assessing therapeutics by product type, molecular structure, and developmental stage. It also sheds light on inactive pipeline products, presenting a robust snapshot of the current and future drug development efforts for Batten Disease.

Batten Disease, or neuronal ceroid lipofuscinosis (NCL), is a group of inherited nervous system disorders characterized by lysosomal accumulation, glial reactivity, and neuronal loss. The disease often begins in childhood and is marked by progressive symptoms such as vision loss, seizures, and cognitive decline. A major breakthrough has been the approval of cerliponase alfa (Brineura) in 2017, a pivotal enzyme replacement therapy for CLN2 Batten Disease.

The report emphasizes promising therapeutic approaches to Batten Disease, detailing current FDA-approved clinical trials and prospective treatment avenues. The report provides an in-depth commercial and clinical assessment of the drugs under development, including their mechanisms of action, clinical studies, and product development activities like collaborations, funding, and mergers.

Key emerging drugs include PLX-200 by Polaryx Therapeutics and TTX-381 by Tern Therapeutics. PLX-200, a repurposed PPAR? agonist, has shown neuroprotective effects in disease models and is in Phase III development. Having obtained fast track designation, it holds potential for multiple NCL subtypes. TTX-381, a pioneering gene therapy, targets ocular manifestations of CLN2. Built on an AAV9 viral vector, it demonstrated positive interim results in its Phase I/II trial, leading to a Fast Track designation by the FDA in May 2025.

The report breaks down the Batten Disease drugs by stage of development: late-stage (Phase III), mid-stage (Phase II), early-stage (Phase I), and preclinical stages. Therapies are also categorized by route of administration-oral, intravenous, subcutaneous-and molecule types such as small molecules and gene therapies.

Companies like Amicus Therapeutics, REGENXBIO, and M6P Therapeutics are key players in advancing Batten Disease treatments. The report offers an analytical perspective on ongoing collaborations, licensing activities, and trends aimed at addressing the unmet needs of current therapies.

The comprehensive insights extend to pipeline product profiles, therapeutic assessments, and evaluations of inactive drugs, providing a strategic framework essential for stakeholders in the Batten Disease therapeutic landscape.

Report Highlights

• Key Players: Amicus Therapeutics, Polaryx Therapeutics, Tern Therapeutics, Theranexus, M6P Therapeutics
• Key Products: AT-GTX-502, RGX-381, PLX-200, TTX-381, BBDF-101, M-082

Key Topics Covered:

Introduction

Executive Summary

Batten Disease: Overview

• Causes
• Pathophysiology
• Signs and Symptoms
• Diagnosis
• Disease Management

Pipeline Therapeutics

• Comparative Analysis

Therapeutic Assessment

• Assessment by Product Type
• Assessment by Stage and Product Type
• Assessment by Route of Administration
• Assessment by Stage and Route of Administration
• Assessment by Molecule Type
• Assessment by Stage and Molecule Type

Batten Disease - Analytical Perspective

Late Stage Products (Phase III)

• Comparative Analysis

PLX-200: Polaryx Therapeutics

• Product Description
• Research and Development
• Product Development Activities

Early Stage Products (Phase I/II)

• Comparative Analysis

TTX381: Tern Therapeutics

• Product Description
• Research and Development
• Product Development Activities

Early Stage Products (Phase I)

• Comparative Analysis

Drug Name: Company Name

• Product Description
• Research and Development
• Product Development Activities

Preclinical and Discovery Stage Products

• Comparative Analysis

Drug Name: Company Name

• Product Description
• Research and Development
• Product Development Activities

Inactive Products

• Comparative Analysis

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

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

For more information about this drug pipelines report visit

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