Leber's Hereditary Optic Neuropathy (LHON) Pipeline 2025: Pioneering Clinical Developments By 6+ Global Leaders Delveinsight Featuring Gensight Biologics, Neurophth Therapeutics, Mitotech, Stealth

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"Leber's Hereditary Optic Neuropathy (LHON) Pipeline 2025"DelveInsight's,“Leber's Hereditary Optic Neuropathy - Pipeline Insight, 2025,” report provides comprehensive insights about 6+ companies and 7+ pipeline drugs in Leber's Hereditary Optic Neuropathy 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.

As Leber's hereditary optic neuropathy becomes increasingly recognized worldwide and is linked to comorbidities like diabetes, cardiovascular disease, and certain cancers, the demand for safer and more effective treatments is rising. According to DelveInsight, the LHON pipeline involves 6+ pharmaceutical and biotech companies actively developing over 7 therapeutic candidates. These therapies are at various stages of clinical and preclinical development, highlighting significant innovation and dedication to addressing this critical public health challenge.

DelveInsight's“Leber's Hereditary Optic Neuropathy Pipeline Insight 2025” report offers a comprehensive and strategic analysis of the ongoing R&D landscape. It examines clinical trial progress, emerging therapies, mechanisms of action, competitive positioning, and key initiatives by companies. The report is an essential resource for stakeholders-including researchers, investors, and decision-makers-seeking insights into the evolving LHON therapeutics market and the breakthroughs shaping its future.

Explore the Cutting-Edge Landscape of Leber's hereditary optic neuropathy Drug Development

Key Takeaways from the Leber's hereditary optic neuropathy Pipeline Report

• DelveInsight's Leber's Hereditary Optic Neuropathy (LHON) pipeline report highlights a dynamic landscape with 6+ active companies developing over 7 therapeutic candidates for LHON treatment.

• As of May 2025, no LHON gene therapies have received full FDA approval. However, orphan drug designation for NFS-01 and ongoing Phase III trials for LUMEVOQ demonstrate meaningful progress toward potential regulatory approval. This gene therapy uses an AAV vector to deliver a functional ND4 gene to retinal cells. According to a study published in JAMA Ophthalmology in August 2023, patients experienced sustained vision improvements for up to five years post-treatment, with improvements observed in both treated and untreated eyes, suggesting a possible bilateral effect.

• Key LHON-focused companies-including GenSight Biologics, Neurophth Therapeutics, Mitotech, Stealth BioTherapeutics, Fortify Therapeutics, Neuroptika, and others-are actively developing new therapies to enhance the treatment landscape. Promising pipeline candidates include GS 010, NFS-02, and additional therapies in various stages of development.

Leber's hereditary optic neuropathy Overview:

Leber hereditary optic neuropathy (LHON) is a genetic disorder that leads to sudden, painless loss of central vision, usually beginning in one eye and spreading to the other within a few weeks, most commonly in individuals under 40. In rare cases, it can also cause neurological issues such as tremors or muscle weakness. The condition is caused by mutations in mitochondrial DNA and is maternally inherited.

Although some carriers-particularly mothers-may show no symptoms, family histories often reveal early vision loss among female relatives. Management primarily involves supportive care, including visual aids and rehabilitation. Limited studies suggest that treatments like ubiquinone and idebenone may offer some benefit. Patients are also advised to avoid smoking and excessive alcohol consumption, as these can increase oxidative stress and worsen mitochondrial damage.

Download the Leber's hereditary optic neuropathy sample report to know in detail about the Leber's hereditary optic neuropathy treatment market

Leber's hereditary optic neuropathy Pipeline Analysis

The Leber's hereditary optic neuropathy pipeline insights report 2025, provides insights into:

• Provides comprehensive insights into key companies developing therapies in the Leber's hereditary optic neuropathy Market.

• Categorizes Leber's hereditary optic neuropathy 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 Leber's hereditary optic neuropathy drugs under development based on:

  • Stage of development

  • Leber's hereditary optic neuropathy Route of administration

  • Target receptor

  • Monotherapy vs. combination therapy

  • Leber's hereditary optic neuropathy Mechanism of action

  • Molecular type

• Offers detailed analysis of:

  • Company-to-company and company-academia collaborations

  • Leber's hereditary optic neuropathy Licensing agreements

  • Funding and investment activities supporting future Leber's hereditary optic neuropathy market advancement.

Unlock key insights into emerging Leber's hereditary optic neuropathy therapies and market strategies here:

Leber's hereditary optic neuropathy Emerging Drugs

GS 010: GenSight Biologics

LUMEVOQ (GS010; lenadogene nolparvovec) is a gene therapy developed to treat Leber Hereditary Optic Neuropathy (LHON) using a proprietary mitochondrial targeting sequence (MTS) technology from the Institut de la Vision in Paris. This approach delivers the therapeutic gene into cells via an adeno-associated virus (AAV) vector, enabling the production of a functional protein that is directed to the mitochondria to restore their activity. The European Medicines Agency (EMA) designated "LUMEVOQ" as the official drug name in 2018. The therapy is currently under regulatory review in Europe and is in Phase III clinical trials in the U.S., progressing toward a Biologics License Application (BLA) submission.

NFS-02: Neurophth Therapeutics

Neurophth is advancing NFS-01 (NR082, rAAV-ND4), a gene therapy designed for LHON associated with the G11778A (ND4) and G3460A (ND1) mutations. The therapy employs a recombinant adeno-associated virus (rAAV) to deliver functional genes directly into affected optic ganglion cells through intravitreal injection, aiming to restore mitochondrial function and improve vision. In September 2020, the U.S. FDA granted NFS-01 orphan drug designation, facilitating its accelerated development worldwide.

Leber's hereditary optic neuropathy Pipeline Therapeutic Assessment

Leber's hereditary optic neuropathy Assessment by Product Type

. Mono

. Combination

. Mono/Combination

Leber's hereditary optic neuropathy 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

Leber's hereditary optic neuropathy Assessment by Route of Administration

. Oral

. Parenteral

. Intravenous

. Subcutaneous

. Topical

Leber's hereditary optic neuropathy Assessment by Molecule Type

. Recombinant fusion proteins

. Small molecule

. Monoclonal antibody

. Peptide

. Polymer

. Gene therapy

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Table of Contents

1. Report Introduction

2. Executive Summary

3. Leber's hereditary optic neuropathy Current Treatment Patterns

6. Leber's hereditary optic neuropathy - DelveInsight's Analytical Perspective

5. Therapeutic Assessment

6. Leber's hereditary optic neuropathy Late-Stage Products (Phase-III)

7. Leber's hereditary optic neuropathy 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. Leber's hereditary optic neuropathy Discontinued Products

13. Leber's hereditary optic neuropathy Product Profiles

16. Leber's hereditary optic neuropathy Key Companies

15. Leber's hereditary optic neuropathy Key Products

16. Dormant and Discontinued Products

17. Leber's hereditary optic neuropathy Unmet Needs

18. Leber's hereditary optic neuropathy Future Perspectives

19. Leber's hereditary optic neuropathy 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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