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"Batten Disease Pipeline 2025"DelveInsight's,“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.

With Batten Disease increasingly impacting populations worldwide and contributing to comorbidities such as diabetes, cardiovascular conditions, and certain cancers, the demand for safer and more effective treatments is rising. DelveInsight reports that over 10 pharmaceutical and biotech companies are actively developing more than 12 therapeutic candidates for Batten Disease. These therapies are at various stages of clinical and preclinical development, highlighting significant innovation and dedication toward addressing this critical public health challenge.

DelveInsight's“Batten Disease Pipeline Insight 2025” offers a comprehensive analysis of the R&D landscape, including clinical trial progress, emerging therapies, mechanisms of action, competitive positioning, and key company initiatives. The report serves as an essential resource for stakeholders-such as researchers, healthcare investors, and decision-makers-providing insights into the evolving Batten Disease therapeutics market and the breakthroughs shaping its future.

Explore the Cutting-Edge Landscape of Batten Disease Drug Development

During the ERS Congress 2025 in September 2025, several biotechnology and pharmaceutical companies shared notable advancements in pulmonary and cardiopulmonary research:

• In September 2025, Diagonal Therapeutics presented new preclinical data highlighting the potential of its lead clustering antibody, DIAG723, as a disease-modifying therapy for pulmonary arterial hypertension (PAH).

• In September 2025, a study revealed that babies raised with dogs may have a reduced risk of developing childhood asthma.

• In September 2025, Savara shared data from the Phase 3 IMPALA-2 trial of Molgramostim Inhalation Solution for patients with autoimmune pulmonary alveolar proteinosis (aPAP).

• In September 2025, AllRock Bio, Inc. reported encouraging Phase 1 results for ROC-101, a first-in-class oral pan-ROCK inhibitor for cardiopulmonary and fibrotic diseases.

• In September 2025, Gossamer Bio, Inc., in collaboration with Chiesi Group, announced five scientific presentations on seralutinib for PAH and PH-ILD.

• In September 2025, Boehringer Ingelheim presented pooled analyses from the Phase III FIBRONEER program on nerandomilast, an investigational oral PDE4B inhibitor, showing a nominally significant reduction in mortality risk across IPF and PPF.

• In September 2025, Kaia Health launched a pilot program of Kaia Breathe, a digital pulmonary rehabilitation solution for COPD patients, in partnership with MedImprove.

• In September 2025, aTyr Pharma, Inc. shared additional findings from the Phase 3 EFZO-FIT study of efzofitimod in 268 patients with pulmonary sarcoidosis, a key form of interstitial lung disease.

Key Takeaways from the Batten Disease Pipeline Report

• DelveInsight's Batten Disease pipeline report highlights a dynamic landscape, featuring over 10 active companies developing more than 12 therapeutic candidates for Batten Disease.

• As of June 2025, the U.S. Food and Drug Administration (FDA) has approved Brineura (cerliponase alfa) as the first and only therapy for CLN2 disease, a subtype of Batten Disease. Originally approved in 2017 for children aged 3 and older, its indication was expanded in July 2024 to include patients of all ages, including those under 3, regardless of symptom onset. Brineura, an enzyme replacement therapy, works by supplementing the deficient TPP1 enzyme, slowing the loss of motor function in children with CLN2 disease.

• Leading companies in the Batten Disease space, such as Polaryx Therapeutics, Neurogene, Theranexus, M6P Therapeutics, Regenxbio, and others, are actively developing novel therapies to enhance treatment options. Promising pipeline candidates under investigation include PLX-200, NGN-101, among others, reflecting ongoing innovation in this therapeutic area.

Batten Disease Overview:

Batten disease encompasses a group of rare, inherited neurological disorders known as neuronal ceroid lipofuscinoses (NCLs). These conditions result from genetic mutations that impair lysosomal function, causing the accumulation of cellular waste, such as proteins and fats, particularly in the brain and retina. This buildup disrupts nerve cell function and leads to progressive neurological deterioration.

There are 13 identified types of Batten disease, all sharing common symptoms such as seizures, vision loss, cognitive decline, and motor impairment. Affected individuals-typically ranging from infants to teenagers-experience gradual worsening of symptoms, including difficulties with speech, learning, concentration, and movement. Many children lose previously acquired abilities, such as walking or talking, struggle with communication, and may develop sleep disorders. Batten disease is ultimately fatal, often resulting in death during late childhood or adolescence.

Download the Batten Disease sample report to know in detail about the Batten Disease treatment market

Batten Disease Pipeline Analysis

The Batten Disease pipeline insights report 2025, provides insights into:

• Provides comprehensive insights into key companies developing therapies in the Batten Disease Market.

• Categorizes Batten Disease therapeutic companies by development stage: early, mid, and late-stage.

• Highlights major companies involved in targeted therapy development, including both active and inactive (paused/discontinued) projects.

• Reviews emerging Batten Disease drugs under development based on:

  • Stage of development

  • Batten Disease Route of administration

  • Target receptor

  • Monotherapy vs. combination therapy

  • Batten Disease Mechanism of action

  • Molecular type

• Offers detailed analysis of:

  • Company-to-company and company-academia collaborations

  • Batten Disease Licensing agreements

  • Funding and investment activities supporting future Batten Disease market advancement.

Unlock key insights into emerging Batten Disease therapies and market strategies here:

Batten Disease Emerging Drugs

PLX-200 – Polaryx Therapeutics

PLX-200 is a repurposed drug originally used to treat various conditions in both adults and children. It functions as a PPARα agonist and enhances lysosome biogenesis by upregulating TFEB, indicating its potential for treating Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL/CLN2) and other forms like Juvenile Neuronal Ceroid Lipofuscinosis (JNCL/CLN3). In mouse models of LINCL and JNCL, PLX-200 demonstrated neuroprotective benefits, including prolonged survival, decreased accumulation of lipofuscin in the brain, and reduced inflammation and cell death. The drug has received orphan drug designation from both the FDA and EMA for all NCL subtypes and is currently undergoing Phase III clinical trials for NCL treatment.

NGN-101 – Neurogene Inc.

NGN-101 is an experimental single-dose gene therapy designed to address both neurological and ocular manifestations of CLN5 Batten disease. It utilizes an AAV9 viral vector to deliver a functional copy of the mutated CLN5 gene in affected children. The therapy has been granted orphan drug status by both the FDA and EMA and is currently being evaluated in Phase I/II clinical trials for Batten disease treatment.

Batten Disease Pipeline Therapeutic Assessment

Batten Disease Assessment by Product Type

. Mono

. Combination

. Mono/Combination

Batten Disease By Stage

. 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

Batten Disease Assessment by Route of Administration

. Oral

. Parenteral

. Intravenous

. Subcutaneous

. Topical

Batten Disease Assessment by Molecule Type

. Recombinant fusion proteins

. Small molecule

. Monoclonal antibody

. Peptide

. Polymer

. Gene therapy

Download sample pages to get an in-depth assessment of the emerging Batten Disease therapies and key Batten Disease companies

Table of Contents

1. Report Introduction

2. Executive Summary

3. Batten Disease Current Treatment Patterns

4. Batten Disease - DelveInsight's Analytical Perspective

5. Therapeutic Assessment

6. Batten Disease Late-Stage Products (Phase-III)

7. Batten Disease Mid-Stage Products (Phase-II)

8. Early Stage Products (Phase-I)

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Batten Disease Discontinued Products

13. Batten Disease Product Profiles

14. Batten Disease Key Companies

15. Batten Disease Key Products

16. Dormant and Discontinued Products

17. Batten Disease Unmet Needs

18. Batten Disease Future Perspectives

19. Batten Disease Analyst Review

20. Appendix

21. Report Methodology

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About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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