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Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Market, Clinical Trials, Companies, Therapies, Therapies, Treatment, Pipeline Analysis | Pfizer, Spark Therapeutics, REGENXBIO

08-26-2025 02:22 AM CET | Associations & Organizations

Press release from: ABNewswire

Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Market

Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Market

DelveInsight's "Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Market Insights, Epidemiology, and Market Forecast-2032" report delivers an in-depth understanding of the Neuronal Ceroid-Lipofuscinoses (CLN1 disease), historical and forecasted epidemiology as well as the Neuronal Ceroid-Lipofuscinoses (CLN1 disease) market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.
DelveInsight reports that several leading companies are actively advancing therapeutic development efforts for Neuronal Ceroid-Lipofuscinoses (CLN1 disease).

Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Overview:

Neuronal Ceroid-Lipofuscinoses (NCLs), including CLN1 disease, are rare inherited neurodegenerative conditions. CLN1, also referred to as Infantile NCL (INCL) or the CLN1 subtype of Batten disease, arises from mutations in the PPT1 (palmitoyl-protein thioesterase 1) gene. These mutations cause the buildup of lipopigments (lipofuscin) within neurons and other cells, ultimately leading to progressive neurological deterioration.

Request for a detailed insights report on Neuronal Ceroid-Lipofuscinoses (CLN1 disease) pipeline insights [https://www.delveinsight.com/report-store/neuronal-ceroid-lipofuscinoses-cln1-disease-market?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr]

DelveInsight's "Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Market Insights, Epidemiology, and Market Forecast-2032" report delivers an in-depth understanding of the Neuronal Ceroid-Lipofuscinoses (CLN1 disease), historical and forecasted epidemiology as well as the Neuronal Ceroid-Lipofuscinoses (CLN1 disease) market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

Key Takeaways from the Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Pipeline Report

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DelveInsight's Neuronal Ceroid-Lipofuscinoses (CLN1 disease) pipeline report highlights a dynamic landscape with several key players actively pursuing novel therapies for CLN1 disease.

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Leading companies such as Pfizer, Spark Therapeutics, REGENXBIO, and others are investigating innovative drug candidates aimed at improving treatment outcomes.

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In May 2019, Abeona Therapeutics secured FDA approval for its IND application to launch a Phase 1/2 clinical trial of ABO-202, an AAV9-based gene therapy developed to deliver a functional PPT1 gene to the central nervous system and peripheral organs. Subsequently, in June 2019, the FDA granted Fast Track designation to ABO-202, acknowledging its potential to meet significant unmet needs in CLN1 disease management.

Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Pipeline Analysis

The report provides insights into:

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The report provides detailed insights into the key companies that are developing therapies in the Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Market.

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The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Neuronal Ceroid-Lipofuscinoses (CLN1 disease) treatment.

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It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

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It navigates the emerging drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.

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Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement, and financing details for future advancement of the Neuronal Ceroid-Lipofuscinoses (CLN1 disease) market.

Download our free sample page report on Neuronal Ceroid-Lipofuscinoses (CLN1 disease) pipeline insights [https://www.delveinsight.com/sample-request/neuronal-ceroid-lipofuscinoses-cln1-disease-market?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr]

Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Companies

Several leading companies, including Pfizer, Spark Therapeutics, REGENXBIO, and others, are actively engaged in developing therapies for Neuronal Ceroid-Lipofuscinoses (CLN1 disease) (CRPS).

DelveInsight's report covers around 3+ products under different phases of clinical development like

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Late stage products (Phase III)

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Mid-stage products (Phase II)

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Early-stage product (Phase I) along with the details of

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Pre-clinical and Discovery stage candidates

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Discontinued & Inactive candidates

Neuronal Ceroid-Lipofuscinoses (CLN1 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

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Intravenous

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Subcutaneous

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Oral

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Intramuscular

Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Products have been categorized under various Molecule types such as

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Monoclonal antibody

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Small molecule

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Peptide

Download Sample Pages to Get an in-depth Assessment of the Emerging Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Therapies and Key Companies: Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Clinical Trials and advancements [https://www.delveinsight.com/report-store/neuronal-ceroid-lipofuscinoses-cln1-disease-market?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr]

Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Pipeline Therapeutic Assessment

- Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Assessment by Product Type

- Neuronal Ceroid-Lipofuscinoses (CLN1 disease) By Stage

- Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Assessment by Route of Administration

- Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Assessment by Molecule Type

Download Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Sample report to know in detail about the Neuronal Ceroid-Lipofuscinoses (CLN1 disease) treatment market @ Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Therapeutic Assessment [https://www.delveinsight.com/sample-request/neuronal-ceroid-lipofuscinoses-cln1-disease-market?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr]

Table of Content

1. Report Introduction

2. Executive Summary

3. Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Current Treatment Patterns

4. Neuronal Ceroid-Lipofuscinoses (CLN1 disease) - DelveInsight's Analytical Perspective

5. Therapeutic Assessment

6. Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Late-Stage Products (Phase-III)

7. Neuronal Ceroid-Lipofuscinoses (CLN1 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. Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Discontinued Products

13. Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Product Profiles

14. Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Key Companies

15. Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Key Products

16. Dormant and Discontinued Products

17. Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Unmet Needs

18. Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Future Perspectives

19. Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Analyst Review

20. Appendix

21. Report Methodology

Request the Sample PDF to Get Detailed Insights About the Neuronal Ceroid-Lipofuscinoses (CLN1 disease) Pipeline Reports Offerings [https://www.delveinsight.com/report-store/neuronal-ceroid-lipofuscinoses-cln1-disease-market?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr]

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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